Publications and Presentations

Medici, G., Tassinari, M., Galvani, G., Bastianini, S., Gennaccaro, L., Loi, M., Mottolese, M., Alvente, S., Berteotti, C., Sagona, G., Lupori, L., Baggett, H. R., Zoccoli, G., Giustetto, M., Muotri, A., Pizzorusso, T., Nakai, H., Trazzi, S., Ciani, E. (2021) Expression of a secretable, cell-penetrating cdkl5 protein enhances the efficacy of AAV vector-mediated gene therapy for cdkl5 deficiency disorder.
Sinnamon, J.R., Kim, S.Y., Fisk, J.R., Song, Z., Nakai, H., Jeng, S., McWeeney, S.K., Mandel, G. (2020) In vivo repair of a protein underlying a neurological disorder by programmable RNA editing. Cell Reports (accepted).
Adachi, K., Dissen, G.A., Lomniczi, A., Xie, Q., Ojeda, S.R., Nakai, H. (2020) Adeno-associated virus-binding antibodies detected in cats living in the Northeastern United States lack neutralizing activity. Sci. Rep. (accepted).
Cabanes-Creus, M., Ginn, S.L., Amaya, A.K.,  Liao, S.H.Y., Westhaus, A., Hallwirth, C.V., Ward, J., Dilworth, K.L., Santilli, G., Rybicki, A., Nakai, H., Thrasher, A.J., Filip, A.C., Alexander, I.E., Lisowski1, L. (2018) Novel codon-optimization of wild-type adeno-associated virus capsid sequences enhances DNA family shuffling while conserving functionality. Mol. Ther. Methods Clin.  12:71-84.
Powers, J.M., Chang X.L., Song, Z., Nakai, H. (2018) Assessment of the ability for adeno-associated virus assembly-activating protein to promote capsid assembly by a quantitative dot blot assay. Journal of Visualized Experiments 136: e56766.
Sinnamon, J.R., Kim, S.Y., Corson, G.M., Song, Z., Nakai, H., Adelman, J.P., Mandel, G. (2017) Site-directed RNA repair of endogenous Mecp2 RNA in neurons. Proc. Natl. Acad. Sci. U. S. A. 114:E9353-E9402.
Earley, L.F., Powers, J.M, Adachi, K., Baumgart, J., Meyer, N.L., Xie, Q., Chapman, M.S., Nakai, H. (2017) Adeno-associated virus assembly-activating protein is not an essential requirement for capsid assembly of AAV serotypes 4, 5 and 11. J. Virol. 91:e01980-16.
Dissen, G.A., Lomniczi, A., Adachi, K., Chatkupt, T., Davidson, B.L., Nakai, H., Ojeda, S.R. (2017) Engineering a Gene Silencing Viral Construct that Targets the Cat Hypothalamus to Induce Permanent Sterility: An Update. Reproduction in Domestic Animals Suppl 2:354-358.
Earley, L.F., Kawano, Y., Adachi, K., Sun, X.-X., Dai, M., Nakai, H. (2015) Identification and characterization of nuclear and nucleolar localization signals in the adeno-associated virus serotype 2 assembly-activating protein. J. Virol. 89:3038-3048.
Adachi, K., Enoki, T., Kawano, Y., Veraz, M. G., Nakai, H. (2014) Drawing a high resolution functional map of adeno-associated virus capsid by massively parallel sequencing. Nat. Commun. 5:3075.
Kawano, Y., Neeley, S., 1, Adachi, K., Nakai, H. (2013) An experimental and computational evolution-based method to study a mode of co-evolution of overlapping open reading frames in the AAV2 viral genome.  PLoS One 8, e66211.
Yu, H., Fischer, G., Ferhatovic, L., Fan, F., Light, A. R., Weihrauch, D., Sapunar, D., Nakai, H.,  Park, F., Hogan, Q. H. (2013) Intraganglionic AAV6 results in efficient and long-term gene transfer to peripheral sensory nervous system in adult rats. PLoS One 8:e61266.
Charan, R.A., Niizawa, G., Nakai, H., Clemens, P.R. (2013) Adeno-associated virus serotype 8 (AAV8) delivery of recombinant A20 to skeletal muscle reduces pathological activation of nuclear factor (NF)-κB in muscle of mdx mice.  Mol. Med. 18, 1527-35.
Wang, Z., Lisowski, L., Finegold, M. J., Nakai, H., Kay, M. A. & Grompe, M. (2012) AAV vectors containing rDNA homology display increased chromosomal integration and transgene persistence. Mol. Ther. 20,  1902-1911.
Fischer, G. J, Kostic, S., Nakai, H., Park, F., Sapunar, D., Yu, H., Hogan, Q. (2011) Direct injection into the dorsal root ganglion: Technical, behavioral, and histological observations. J. Neurosci. Methods 199, 43-55.
Kotchey, N., Adachi, K., Zahid, M., Inagaki, K., Charan, R., Parker, P., Nakai, H. (2011) A potential role of distinctively delayed blood clearance of recombinant adeno-associated virus serotype 9 in robust cardiac transduction. Mol. Ther. 19, 1079-1089.
Adachi, K., Nakai, H. (2010) A new recombinant adeno-associated virus (AAV)-based random peptide display library system: infection-defective AAV1.9-3 as a novel detargeted platform for vector evolution. Gene Ther. Regulation 5, 31-55.
Eghtesad, S., Zheng H., Nakai, H., Michael W Epperly, M. W., Clemens, P. R. (2010) Irradiation of adult mdx mice prior to full-length dystrophin cDNA transfer results in a delayed and diminished anti-dystrophin immune response. Gene Ther. 17, 1181-1190.
Dong, B., Nakai, H. and Xiao, W. (2010) Characterization of genome integrity for oversized recombinant AAV vector. Mol. Ther. 18, 87-92.
Nishiyama, A., Ampong, B.N., Ohshima, S., Shin, J.H., Nakai, H., Imamura, M., Miyagoe-Suzuki, Y., Okada, T. and Takeda, S. (2008) Recombinant adeno-associated virus type 8-mediated extensive therapeutic gene delivery into skeletal muscle of alpha-sarcoglycan-deficient mice. Hum. Gene Ther., 19, 719-730.
Miyagi, N., Rao, V.P., Ricci, D., Du, Z., Byrne, G.W., Bailey, K.R., Nakai, H., Russell, S.J. and McGregor, C.G. (2008) Efficient and durable gene transfer to transplanted heart using adeno-associated virus 9 vector. J. Heart Lung Transplant., 27, 554-560.
McCaffrey, A.P., Fawcett, P., Nakai, H., McCaffrey, R.L., Ehrhardt, A., Pham, T.T., Pandey, K., Xu, H., Feuss, S., Storm, T.A. and Kay, M.A. (2008) The host response to adenovirus, helper-dependent adenovirus, and adeno-associated virus in mouse liver. Mol. Ther., 16, 931-941.
Inagaki, K., Piao, C., Kotchey, N., Wu, X. and Nakai, H. (2008) Frequency and spectrum of genomic integration of recombinant adeno-associated virus serotype 8 vector in neonatal mouse liver. J. Virol., 82, 9513-9524 (highlighted in Spotlight).
Inagaki, K., Ma, C., Storm, T.A., Kay, M.A. and Nakai, H. (2007) The Role of DNA-PKcs and Artemis in opening viral DNA hairpin termini in various tissues in mice. J. Virol., 81, 11304-11321.
Inagaki, K., Lewis, S.M., Wu, X., Ma, C., Munroe, D.J., Fuess, S., Storm, T.A., Kay, M.A. and Nakai, H. (2007) DNA palindromes with a modest arm length of  > ~20 base pairs are a significant target for rAAV vector integration in the liver, muscles and heart in mice. J. Virol., 81, 11290-11303 (highlighted in Spotlight).
Manno, C.S., Pierce, G.F., Arruda, V.R., Glader, B., Ragni, M., Rasko, J.J., Ozelo, M.C., Hoots, K., Blatt, P., Konkle, B., Dake, M., Kaye, R., Razavi, M., Zajko, A., Zehnder, J., Rustagi, P.K., Nakai, H., Chew, A., Leonard, D., Wright, J.F., Lessard, R.R., Sommer, J.M., Tigges, M., Sabatino, D., Luk, A., Jiang, H., Mingozzi, F., Couto, L., Ertl, H.C., High, K.A. and Kay, M.A. (2006) Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response. Nat. Med., 12, 342-347.
Inagaki, K., Fuess, S., Storm, T.A., Gibson, G.A., McTiernan, C.F., Kay, M.A. and Nakai, H. (2006) Robust systemic transduction with AAV9 vectors in mice: efficient global cardiac gene transfer superior to that of AAV8. Mol. Ther., 14, 45-53.
Grimm, D., Pandey, K., Nakai, H., Storm, T.A. and Kay, M.A. (2006) Liver transduction with recombinant adeno-associated virus is primarily restricted by capsid serotype not vector genotype. J. Virol., 80, 426-439.
Ohashi, K., Nakai, H., Couto, L.B. and Kay, M.A. (2005) Modified infusion procedures affect recombinant adeno-associated virus vector type 2 transduction in the liver. Hum. Gene Ther., 16, 299-306.
Nakai, H., Wu, X., Fuess, S., Storm, T.A., Munroe, D., Montini, E., Burgess, S.M., Grompe, M. and Kay, M.A. (2005) Large-scale molecular characterization of adeno-associated virus vector integration in mouse liver. J. Virol., 79, 3606-3614.
Nakai, H., Fuess, S., Storm, T.A., Muramatsu, S., Nara, Y. and Kay, M.A. (2005) Unrestricted hepatocyte transduction with adeno-associated virus serotype 8 vectors in mice. J. Virol., 79, 214-224.
Nakai, H., Storm, T.A., Fuess, S. and Kay, M.A. (2003) Pathways of removal of free DNA vector ends in normal and DNA-PKcs-deficient SCID mouse hepatocytes transduced with rAAV vectors. Hum. Gene Ther., 14, 871-881.
Nakai, H., Montini, E., Fuess, S., Storm, T.A., Meuse, L., Finegold, M., Grompe, M. and Kay, M.A. (2003) Helper-independent and AAV-ITR-independent chromosomal integration of double-stranded linear DNA vectors in mice. Mol. Ther., 7, 101-111.
Nakai, H., Montini, E., Fuess, S., Storm, T.A., Grompe, M. and Kay, M.A. (2003) AAV serotype 2 vectors preferentially integrate into active genes in mice. Nat. Genet., 34, 297-302.
Nakai, H., Fuess, S., Storm, T.A., Meuse, L.A. and Kay, M.A. (2003) Free DNA ends are essential for concatemerization of synthetic double-stranded adeno-associated virus vector genomes transfected into mouse hepatocytes in vivo. Mol. Ther., 7, 112-121.
Kay, M.A. and Nakai, H. (2003) Looking into the safety of AAV vectors. Nature, 424, 251
McCaffrey, A.P., Nakai, H., Pandey, K., Huang, Z., Salazar, F.H., Xu, H., Wieland, S.F., Marion, P.L. and Kay, M.A. (2003) Inhibition of hepatitis B virus in mice by RNA interference. Nat. Biotechnol., 21, 639-644.
Grimm, D., Zhou, S., Nakai, H., Thomas, C.E., Storm, T.A., Fuess, S., Matsushita, T., Allen, J., Surosky, R., Lochrie, M., Meuse, L., McClelland, A., Colosi, P. and Kay, M.A. (2003) Preclinical in vivo evaluation of pseudotyped adeno-associated virus vectors for liver gene therapy. Blood, 102, 2412-2419.
Nakai, H., Thomas, C.E., Storm, T.A., Fuess, S., Powell, S., Wright, J.F. and Kay, M.A. (2002) A limited number of transducible hepatocytes restricts a wide-range linear vector dose response in recombinant adeno-associated virus-mediated liver transduction. J. Virol., 76, 11343-11349.
Nakai, H., Yant, S.R., Storm, T.A., Fuess, S., Meuse, L. and Kay, M.A. (2001) Extrachromosomal recombinant adeno-associated virus vector genomes are primarily responsible for stable liver transduction in vivo. J. Virol., 75, 6969-6976.
Ohashi, K., Marion, P.L., Nakai, H., Meuse, L., Cullen, J.M., Bordier, B.B., Schwall, R., Greenberg, H.B., Glenn, J.S. and Kay, M.A. (2000) Sustained survival of human hepatocytes in mice: A model for in vivo infection with human hepatitis B and hepatitis delta viruses. Nat. Med., 6, 327-331.
Nakai, H., Storm, T.A. and Kay, M.A. (2000) Increasing the size of rAAV-mediated expression cassettes in vivo by intermolecular joining of two complementary vectors. Nat. Biotechnol., 18, 527-532.
Nakai, H., Storm, T.A. and Kay, M.A. (2000) Recruitment of single-stranded recombinant adeno-associated virus vector genomes and intermolecular recombination are responsible for stable transduction of liver in vivo. J. Virol., 74, 9451-9463.
Nakai, H., Miao, C.H., Thompson, A.R., Storm, T.A., Chiu, W., Snyder, R.O. and Kay, M.A. (2000) Nonrandom transduction of recombinant adeno-associated virus vectors in mouse hepatocytes in vivo: cell cycling does not influence hepatocyte transduction. J. Virol., 74, 3793-3803.
Arai, Y., Kubo, T., Fushiki, S., Mazda, O., Nakai, H., Iwaki, Y., Imanishi, J. and Hirasawa, Y. (2000) Gene delivery to human chondrocytes by an adeno associated virus vector. J. Rheumatol., 27, 979-982.
Nakai, H., Iwaki, Y., Kay, M.A. and Couto, L.B. (1999) Isolation of recombinant adeno-associated virus vector-cellular DNA junctions from mouse liver. J. Virol., 73, 5438-5447.
Burton, M., Nakai, H., Colosi, P., Cunningham, J., Mitchell, R. and Couto, L. (1999) Coexpression of factor VIII heavy and light chain adeno-associated viral vectors produces biologically active protein. Proc. Natl. Acad. Sci. U. S. A., 96, 12725-12730.
Nakai, H., Herzog, R.W., Hagstrom, J.N., Walter, J., Kung, S.H., Yang, E.Y., Tai, S.J., Iwaki, Y., Kurtzman, G.J., Fisher, K.J., Colosi, P., Couto, L.B. and High, K.A. (1998) Adeno-associated viral vector-mediated gene transfer of human blood coagulation factor IX into mouse liver. Blood, 91, 4600-4607.
Medici, G., Tassinari, M., Galvani, G., Bastianini, S., Gennaccaro, L., Loi, M., Mottolese, M., Alvente, S., Berteotti, C., Sagona, G., Lupori, L., Baggett, H. R., Zoccoli, G., Giustetto, M., Muotri, A., Pizzorusso, T., Nakai, H., Trazzi, S., Ciani, E. (2021) Expression of a secretable, cell-penetrating cdkl5 protein enhances the efficacy of AAV vector-mediated gene therapy for cdkl5 deficiency disorder. bioRxiv (doi: https://doi.org/10.1101/2021.07.26.453746)
Pincus, A. B., 1, Huang, S. J., 1, Lebold, K.M., de la Torre, U., Proskocil, B. J., Drake, M. J., Nakai, H., Fryer, A. D., Jacoby, D. B. (2022) Multicolor labeling of airway neurons and analysis of parasympathetic heterogeneity. Sci. Rep. (accepted)
Chang, S., Nakai, H. The Role of the Degron, a Protein-Degradation Motif, Essential for Capsid Assembly of Adeno-Associated Virus (AAV). Saturday Academy Apprenticeships in Science and Engineering (ASE) Program Symposium. University of Portland, Portland, OR, August 16, 2019.
Wieland, J., Nakai, H. Increasing Genetic Manipulation Efficiency by Identifying Enhancer Regions on Adeno-Associated Virus (AAV) Vectors. Saturday Academy Apprenticeships in Science and Engineering (ASE) Program Symposium. University of Portland, Portland, OR, August 16, 2019.
Neferkara, A., Nakai, H. Investigating the Mechanism of AAP Independendent Viral Capsid Assembly of the Adeno-Associated Virus, a Promising Gene Therapy Vector. Saturday Academy Apprenticeships in Science and Engineering (ASE) Program Symposium. University of Portland, Portland, OR, August 16, 2019.
Adachi, K., Nakai, H. Impaired production of an AAV VP1 unique region (VP1u) mutant provides new insight into the AAV vector biology. The 26th Annual Meeting of the Japanese Society of Gene and Cell Therapy. Tokyo, Japan, July 20-23, 2019.
Kollu, S., Song, Z., Sairavi, A., David, L., Nakai, H. Identification of VP-interacting cellular proteins in AAV capsid assembly by proximity proteomics. The 26th Annual Meeting of the Japanese Society of Gene and Cell Therapy. Tokyo, Japan, July 20-23, 2019.
Huang, S. Adachi, K., Baggett, H. R., Song, Z., Dissen, D. A., Ojeda, S. R., Nakai, H. A transcription-dependent approach to directed evolution of the AAV capsid identifies novel liver detargeted variants capable of enhanced neuronal transduction in mouse and non-human primate. The 26th Annual Meeting of the Japanese Society of Gene and Cell Therapy. Tokyo, Japan, July 20-23, 2019 (Received Anges Travel Award).
Takahama, K., Kimura, M., Adachi, K., Dissen, D. A., Nakai, H. Identification of novel AAV1 / AAV9 chimeric mutants exhibiting enhanced gene delivery in the central nervous system. The 26th Annual Meeting of the Japanese Society of Gene and Cell Therapy. Tokyo, Japan, July 20-23, 2019.
Huang, S. Adachi, K., Baggett, H. R., Chai, S., Grompe, M., Nakai, H. A novel approach to developing enhanced β cell-targeting AAV capsids by TRAnscription-dependent Directed Evolution (TRADE). The 5th HIRN (Human Islet Research Network) Annual Investigator Meeting, Washington, D.C., MD, April 28 - May 1, 2019.
Adachi, K., Nakai, H. A single amino acid residue in the VP1 unique region plays an essential role in AAV capsid formation. The 22th Annual Meeting of the American Society of Gene and Cell Therapy, Washington D.C., April 29 - May 2, 2019.
Baggett, H. R., Chang, X. L., Adachi, K., Nakai, H. Human polyclonal anti-AAV neutralizing antibody epitope mapping by NGS identifies common epitopes and enables the design of stealth mutants. The 22th Annual Meeting of the American Society of Gene and Cell Therapy, Washington D.C., April 29 - May 2, 2019.
Kollu, S., Song, Z., Sairavi, A., David, L., Nakai, H. Identification of VP-interacting cellular proteins in AAV capsid assembly by proximity proteomics. The 22th Annual Meeting of the American Society of Gene and Cell Therapy, Washington D.C., April 29 - May 2, 2019 (Received the ASGCT Meritorious Abstract Travel Award).
Huang, S. Adachi, K., Baggett, H. R., Song, Z., Dissen, D. A., Ojeda, S. R., Nakai, H. Cell Type-Specific TRAnscription-Dependent Directed Evolution (TRADE) Identifies Novel AAV Capsids Capable of Enhanced Neuronal Transduction in Mice and Non-Human Primates. The 22th Annual Meeting of the American Society of Gene and Cell Therapy, Washington D.C., April 29 - May 2, 2019 (Received the ASGCT Excellence in Research Award and Meritorious Abstract Travel Award).
Takahama, K., Kimura, M., Adachi, K., Dissen, D. A., Nakai, H. Novel AAV1 mutants for robust transduction of the central nervous system following intravenous injection. The 22th Annual Meeting of the American Society of Gene and Cell Therapy, Washington D.C., April 29 - May 2, 2019 (Received the ASGCT Meritorious Abstract Travel Award).
Nakai, H. A mysterious disappearance of a partner in AAV viral capsid assembly. OHSU Gene Therapy Symposium, Portland, October 18, 2018.
Tanaka, Y., Kollu, S., Sairavi, A., Nakai, H. Separation, interchange and chimerization of the N- and C-terminal halves of the AAV AAP proteins reveals their functional domains. OHSU Gene Therapy Symposium, Portland, October 18, 2018.
Tanaka, Y., Takahama, K., Sairavi, A., Nakai, H. Production and characterization of infectious Snake AAV vectors with a cross-packaged AAV2-ITR genome. OHSU Gene Therapy Symposium, Portland, October 18, 2018.
Kollu, S., Song, Z., Chang, X., Earley, L.F., Kulik, V., Nakai, H. A proximity-based proteomics approach to identify cellular proteins interacting with AAV assembly activating protein (AAP). OHSU Gene Therapy Symposium, Portland, October 18, 2018.
Ferrick-Kiddie, E., Gupta, A., Song, Z., Galivo, F., Grompe, M., Nakai, H. A high-throughput method of constructing and screening short synthetic gene regulatory elements. OHSU Gene Therapy Symposium, Portland, October 18, 2018.
Huang, S.J., Ryals, R., Pennesi, M.E., Nakai, H. Intravitreal administration of an AAV6 triple mutant vector fails to transduce retinal microglia. OHSU Gene Therapy Symposium, Portland, October 18, 2018.
Adachi, K., Liu, Z., Kroenke, C.D., Dissen, D.A., Ojeda, S.R., Hou, J., Nakai, H. Distribution and transduction of multiple rAAV serotype/mutant vectors in the non-human primate brain after intracisternal injection by an AAV DNA/RNA barcoding library. OHSU Gene Therapy Symposium, Portland, October 18, 2018.
Nakai, H. AAV vectors and gene therapy. Markus Grompe 60th Anniversary Symposium, Portland, March 16, 2018
Nugent, N., Nakai, H. Deciphering a puzzle of overlapping protein coding sequences: a deeper understanding of a virus used for human gene therapy. Saturday Academy Apprenticeships in Science and Engineering (ASE) Program Symposium. Portland, OR, August 17, 2018.
Morakinyo, E., Nakai, H. The disappearance act: the mystery behind the capsid formation of adeno-associated viruses, a gene therapy vector. Saturday Academy Apprenticeships in Science and Engineering (ASE) Program Symposium. Portland, OR, August 17, 2018.
Ferrick-Kiddie, E., Gupta, A., Song, Z., Galivo, F., Grompe, M., Nakai, H. A high-throughput method of constructing and screening short synthetic gene regulatory elements. The 25th Annual Meeting of the Japanese Society of Gene and Cell Therapy. Tokyo, Japan, July 26-28, 2018.
Kollu, S., Song, Z., Chang, X., Earley, L.F., Kulik, V., Nakai, H. A proximity-based proteomics approach to identify cellular proteins interacting with AAV assembly activating protein (AAP). The 25th Annual Meeting of the Japanese Society of Gene and Cell Therapy. Tokyo, Japan, July 26-28, 2018.
Tanaka, Y., Kollu, S., Sairavi, A., Nakai, H. Separation, interchange and chimerization of the N- and C-terminal halves of the AAV AAP proteins reveals their functional domains. The 21th Annual Meeting of the American Society of Gene and Cell Therapy, Chicago, IL, May 16-19, 2018.
Tanaka, Y., Takahama, K., Sairavi, A., Nakai, H. Production and characterization of infectious Snake AAV vectors with a cross-packaged AAV2-ITR genome. The 21th Annual Meeting of the American Society of Gene and Cell Therapy, Chicago, IL, May 16-19, 2018.
Kollu, S., Song, Z., Chang, X., Earley, L.F., Kulik, V., Nakai, H. A proximity-based proteomics approach to identify cellular proteins interacting with AAV assembly activating protein (AAP). The 21th Annual Meeting of the American Society of Gene and Cell Therapy, Chicago, IL, May 16-19, 2018. (Awarded ASGCT Travel Grant).
Ferrick-Kiddie, E., Gupta, A., Song, Z., Galivo, F., Grompe, M., Nakai, H. A high-throughput method of constructing and screening short synthetic gene regulatory elements. The 21th Annual Meeting of the American Society of Gene and Cell Therapy, Chicago, IL, May 16-19, 2018.
Huang, S.J., Ryals, R., Pennesi, M.E., Nakai, H. Intravitreal administration of an AAV6 triple mutant vector fails to transduce retinal microglia. The 21th Annual Meeting of the American Society of Gene and Cell Therapy, Chicago, IL, May 16-19, 2018.
Adachi, K., Liu, Z., Kroenke, C.D., Dissen, D.A., Ojeda, S.R., Hou, J., Nakai, H. Distribution and transduction of multiple rAAV serotype/mutant vectors in the non-human primate brain after intracisternal injection by an AAV DNA/RNA barcoding library. The 21th Annual Meeting of the American Society of Gene and Cell Therapy, Chicago, IL, May 16-19, 2018.
Ferrick-Kiddie, E., Gupta, A., Song, Z., Galivo, F., Grompe, M., Nakai, H. A high-throughput method of constructing and screening short synthetic gene regulatory elements. The 4th HIRN (Human Islet Research Network) Annual Investigator Meeting, Bethesda, MD, May 9-11, 2018.
Nakai, H. Liverpool hypothesis. OHSU Gene Therapy Symposium, Portland, October 11, 2017.
Li, L., Nakai, H. Identification of amino acids regulating AAP requirement for AAV capsid assembly. OHSU Gene Therapy Symposium, Portland, October 11, 2017.
Chang X.L., Adachi, Nakai, H. Adeno-associated virus 2 (AAV2) antibody epitope mapping. OHSU Gene Therapy Symposium, Portland, October 11, 2017.
Kollu, S., Nakai, H. Identification of cellular proteins interacting with assembly-activating protein (AAP). OHSU Gene Therapy Symposium, Portland, October 11, 2017.
Tanaka, Y., Adachi, K., Hanebury, E., Banerjee H., Nakai, H. Biology of capsid and assembly-activating protein of reptile adeno-associated viruses isolated from royal python and bearded dragon. OHSU Gene Therapy Symposium, Portland, October 11, 2017.
Galivo, F.H., Adachi, K., Song, Z., Ackermann A., Sander, M., Kaestner, K., Nakai, H., Grompe, M. Transcriptional targeting of human alpha and beta cells using recombinant AAV. OHSU Gene Therapy Symposium, Portland, October 11, 2017.
Song, Z., Galivo, F.H., Adachi, K., Grompe, M., Nakai, H. Strong alpha cell preference of the AAV strains that best transduce human pancreatic islets in vitro. OHSU Gene Therapy Symposium, Portland, October 11, 2017.
Chen, Y., Song, Z., Adachi, K., Barber, J., Dissen, G.A., Ojeda, S.O., Lomniczi, A., Nakai, H. Unique biological properties of anti-AAV antibodies found in cat sera. OHSU Gene Therapy Symposium, Portland, October 11, 2017.
Gupta, A., Nakai, H. A novel method of constructing short synthetic gene regulatory elements. OHSU Gene Therapy Symposium, Portland, October 11, 2017.
Li, L., Nakai, H. Identification of amino acids regulating AAP requirement for AAV capsid assembly. Saturday Academy Apprenticeships in Science and Engineering (ASE) Program Symposium. Portland, OR, August 18, 2017.
Dissen, G., Lomniczi, A., Adachi, K., Chatkupt, T., Nakai, H., Ojeda, S.R. Contraception today and tomorrow. The 20th International Congress of European Veterinary Society for Small Animal Reproduction. Vienna, Austria, June 29-July 1, 2017.
Kollu, S., Earley, L.F., Powers, J.M., Song, Z., Adachi, K., Kelley, H., Guo, C., Cheng, G., Nakai, H. A single amino acid in AAV capsids regulate the requirement of the assembly activating protein (AAP) for assembly. The 23th Annual Meeting of the Japanese Society of Gene and Cell Therapy. Okayama, Japan, July 20-22, 2017.
Song, Z., Galivo, F.H., Adachi, K., Grompe, M., Nakai, H. Strong alpha cell preference of the AAV strains that best transduce human pancreatic islets in vitro. The 23th Annual Meeting of the Japanese Society of Gene and Cell Therapy. Okayama, Japan, July 20-22, 2017.
Chang X.L., Adachi, Nakai, H. High-throughput mapping of conformational epitopes of monoclonal and polyclonal antibodies against AAV capsids by Immunoprecipitation (IP)-Seq. The 23th Annual Meeting of the Japanese Society of Gene and Cell Therapy. Okayama, Japan, July 20-22, 2017.
Huang S.J., Song, Z., Adachi, K., Dissen, G., Ojeda, S.R., Nakai, H. A novel single amino acid mutation to AAV-PHP.B detargets the liver while retaining highly efficient brain neuron transduction. The 23th Annual Meeting of the Japanese Society of Gene and Cell Therapy. Okayama, Japan, July 20-22, 2017 (Received Anges Travel Award).
Galivo, F.H., Adachi, K., Song, Z., Ackermann A., Sander, M., Kaestner, K., Nakai, H., Grompe, M. Transcriptional targeting of human alpha and beta cells using recombinant AAV. The 20th Annual Meeting of the American Society of Gene and Cell Therapy, Washington D.C., May 10-13, 2017.
Song, Z., Galivo, F.H., Adachi, K., Grompe, M., Nakai, H. Strong alpha cell preference of the AAV strains that best transduce human pancreatic islets in vitro. The 20th Annual Meeting of the American Society of Gene and Cell Therapy, Washington D.C., May 10-13, 2017.
Huang S.J., Song, Z., Adachi, K., Dissen, G., Ojeda, S.R., Nakai, H. Highly efficient brain neuron targeting and hepatocyte detargeting with a novel AAV9-PHP.B capsid harboring a liver-detargeting mutation. The 20th Annual Meeting of the American Society of Gene and Cell Therapy, Washington D.C., May 10-13, 2017.
Chang X.L., Adachi, K., Keck, I., Nakai, H. Mapping of conformational epitopes of monoclonal and polyclonal antibodies against AAV capsids by an Immunoprecipitation-Seq (IP-Seq) technology. The 20th Annual Meeting of the American Society of Gene and Cell Therapy, Washington D.C., May 10-13, 2017.
Tanaka, Y., Adachi, K., Banerjee H., Nakai, H. Biology of capsid and assembly-activating protein of reptile adeno-associated viruses isolated from royal python and bearded dragon. The 20th Annual Meeting of the American Society of Gene and Cell Therapy, Washington D.C., May 10-13, 2017.
Powers, J.M., Adachi, K., Sivyer B., Taylor, R.R., Pennesi, M.E., Nakai, H. Assessment and optimization of DNA/RNA barcode sequencing for comparing transduction capabilities of numerous adeno-associated virus (AAV) strains following intravitreal injection. The 20th Annual Meeting of the American Society of Gene and Cell Therapy, Washington D.C., May 10-13, 2017.
Kollu, S., Earley, L.F., Powers, J.M., Song, Z., Adachi, K., Kelley, H., Guo, C., Cheng, G., Nakai, H. A single amino acid in AAV capsids regulate the requirement of the assembly activating protein (AAP) for assembly. The 20th Annual Meeting of the American Society of Gene and Cell Therapy, Washington D.C., May 10-13, 2017. (Selected as the top 6 abstracts and received Excellence in Research Award and ASGCT Travel Grant).
Galivo, F.H., Adachi, K., Song, Z., Ackermann A., Kaestner, K., Nakai, H., Grompe, M. Transcriptional targeting of human alpha and beta cells using recombinant AAV. The 3nd HIRN (Human Islet Research Network) Annual Investigator Meeting, Bethesda, MD, March 8-10, 2017.
Song, Z., Galivo, F.H., Adachi, K., Grompe, M., Nakai, H. The best AAV transducer of human pancreatic islets in vitro has a strong preference for alpha cells. The 3nd HIRN (Human Islet Research Network) Annual Investigator Meeting, Bethesda, MD, March 8-10, 2017.
Nakai, H., Adachi, K., Galivo, F.H., Grompe, M. Development of liver-detargeting AAV mutants important for in vivo islet transduction by comprehensive alanine scanning of multiple AAV serotype capsids. The 3nd HIRN (Human Islet Research Network) Annual Investigator Meeting, Bethesda, MD, March 8-10, 2017.
Chang X.L., Adachi, Nakai, H. Adeno-associated virus 2 (AAV2) antibody epitope mapping. The 5th Annual OHSU Gene Therapy Symposium, Portland, OR, October 27, 2016. (Second best poster award)
Song, Z., Dissen, G.A., Chen, Y., Lomniczi, A., Ojeda, S.R., Nakai, H. rAAV: A delivery agent that reliably and efficiently targets neurons of the cat hypothalamus and induce permanent sterility. The 5th Annual OHSU Gene Therapy Symposium, Portland, OR, October 27, 2016. (Best poster award)
Keeley, H., Guo, C. (equal contribution), Nakai, H. AAP-independent capsid assembly in adeno-associated virus. Saturday Academy Apprenticeships in Science and Engineering (ASE) Program Symposium. Portland, OR, August 12, 2016.
Kulik, V., Nakai, H. BioID: Identifying mystery proteins. Saturday Academy Apprenticeships in Science and Engineering (ASE) Program Symposium. Portland, OR, August 12, 2016.
Earley, L.F., Powers, J., Adachi, K., Meyer, N.L., Xie, Q., Chapman, M.S., Nakai, H. Assembly-activating protein is not required for the production of infectious AAV5 virions. The 22th Annual Meeting of the Japanese Society of Gene and Cell Therapy. Tokyo, Japan, July 28-30, 2016.
Powers, J., Adachi, K., Sivyer, B., Taylor, W.R., Pennesi, M.E., Nakai, H. An approach to compare multiple adeno-associated virus (AAV) capsids side-by-side in a single eye following intravitreal injection. The 22th Annual Meeting of the Japanese Society of Gene and Cell Therapy. Tokyo, Japan, July 28-30, 2016.
Adachi, K., Nakai, H. The 16 amino acid stretch of N-terminal region in AAV9 capsid is essential for persistence in blood circulation. The 22th Annual Meeting of the Japanese Society of Gene and Cell Therapy. Tokyo, Japan, July 28-30, 2016.
Dissen, G.A., Lomniczi, A., Adachi, K., Chatkupt, T., Davidson, B.L., Nakai, H., Ojeda, S.R. Engineering a gene silencing viral construct that targets the cat hypothalamus to induce permanent sterility. The 8th International Symposium on Canine and Feline Reproduction with XIX EVSSAR Congress. Paris, France, June 22-25, 2016.
Adachi, K., Galivo, F.H., Grompe, M., Nakai, H. In vitro comparison of 40 AAV serotypes and mutants in human islet transduction using an RNA barcoding technology. The 2nd HIRN (Human Islet Research Network) Annual Investigator Meeting, Bethesda, MD, May 25-27, 2016.
Galivo, F., Adachi, K., Sander, M., Ackermann A., Kaestner, K., Nakai, H., Grompe M. RNA-barcoded rAAV-mediated cell specific targeting of pancreatic endocrine-specific transcription modules. The 2nd HIRN (Human Islet Research Network) Annual Investigator Meeting, Bethesda, MD, May 25-27, 2016
Kimura, M., Nakai, H. Identification of a 16 amino acid-long motif in the N-terminal half of the AAV9 capsid essential for persistence in the blood for an extended period of up to 3 days. The 19th Annual Meeting of the American Society of Gene and Cell Therapy, Washington D.C., May 4-7, 2016.
Kimura, M., Adachi, K., Nakai, H. A method for developing more potent AAV capsids by prolonging the vector half-lives in the blood circulation. The 19th Annual Meeting of the American Society of Gene and Cell Therapy, Washington D.C., May 4-7, 2016. (Awarded ASGCT Travel Grant).
Powers, J., Adachi, K., Sivyer, B., Taylor, W., Pennesi, M., Nakai, H. An approach to compare multiple adeno-associated virus (AAV) capsids side-by-side in a single eye following intravitreal injection. The 19th Annual Meeting of the American Society of Gene and Cell Therapy, Washington D.C., May 4-7, 2016.
Earley, L.F., Powers, J., Adachi, K., Meyer, N., Xie, Q., Chapman, M.S., Nakai, H. Assembly-activating protein is not an essential requirement for capsid assembly of adeno-associated virus serotypes 4, 5, and 11. The 19th Annual Meeting of the American Society of Gene and Cell Therapy, Washington D.C., May 4-7, 2016.
Powers, J., Adachi, K., Sivyer, B., Taylor, W., Pennesi, M., Nakai, H. An approach to identify novel adeno-associated virus (AAV) capsids that provide enhanced retinal cell transduction following intravitreal injections. The Annual Meeting of the Association for Research in Vision and Ophthalmology (ARVO) 2016, Seattle, WA, May 2-5, 2016.
Richards, D.Y., Adachi, K., Chang, X.L., Nakai, H. Approach to design knowledge-based neutralizing antibody-escaping adeno-associated virus vectors. OHSU Gene Therapy Symposium, Portland, OR, November 4, 2015. (Best poster award)
Powers, J.M., Adachi, K., Sivyer, B., Taylor, R., Pennesi, M., Nakai, H. Identification of novel adeno-associated virus (AAV) capsids that provide enhanced retinal cell transduction following intravitreal injection. OHSU Gene Therapy Symposium, Portland, OR, November 4, 2015.
Earley, L.F., Powers, J.M., Meyer, N., Xie, Q., Chapman M., Nakai, H. Assembly-activating protein is not an absolute requirement of capsid assembly of a subset of adeno-associated virus serotypes. OHSU Gene Therapy Symposium, Portland, OR, November 4, 2015.
Nakai, H. Pharmacokinetics of AAV vectors. OHSU Gene Therapy Symposium, Portland, OR, November 4, 2015.
Banerjee, H., Nakai, H. Creation and characterization of a novel snake adeno-associated virus vector for gene therapy. Saturday Academy Apprenticeships in Science and Engineering (ASE) Program Symposium. Portland, OR, August 21, 2015.
Shiheido H., Adachi, K., Galivo, F., Nakai, H., Kay, M.A., Grompe, M. Development of novel cell-type specific AAV vectors and cis-regulatory elements toward gene therapy for type I diabetes and islet research. The 1st HIRN Annual Meeting, Los Angeles, CA, May 14, 2015.
Earley, L.F., Powers, J., Adachi, K., Meyer, N., Xie, Q., Chapman, M., Nakai, H. Striking differences in the mode of capsid assembly between adeno-associated virus serotypes 2 and 5. The 21th Annual Meeting of the Japanese Society of Gene Therapy. Osaka, Japan, August 23-25, 2015.
Adachi, K., Liu Z., Kroenke C.D., Dissen, G.A., Ojeda, S.R., Nakai, H. Three-dimensional imaging of multiple AAV vector distributions in the non-human primate brain. The 21th Annual Meeting of the Japanese Society of Gene Therapy. Osaka, Japan, August 23-25, 2015.
Adachi, K., Liu Z., Kroenke C.D., Dissen, G.A., Ojeda, S.R., Nakai, H. Three-dimensional imaging of multiple AAV serotype/mutant vector transductions in the non-human primate brain by massively parallel sequencing. The 18th Annual Meeting of the American Society of Gene and Cell Therapy, New Orleans, LO, May 13-16, 2015. (Selected as the top 3 abstracts for presentation at the Presidential Symposium, awarded ASGCT Travel Grant).
Earley, L., Powers, J., Adachi, K., Nakai, H. Unique properties of AAV5 assembly-activating protein (AAP) and its role in capsid assembly. The 18th Annual Meeting of the American Society of Gene and Cell Therapy, New Orleans, LO, May 13-16, 2015.
Earley, L., Adachi, K., Nakai, H. The joint nuclear-nucleolar signal in the AAV2 assembly-activating protein (AAP) contains a secondary signal important for yielding high AAV titer. The 18th Annual Meeting of the American Society of Gene and Cell Therapy, New Orleans, LO, May 13-16, 2015. (Awarded ASGCT Travel Grant).
Powers, J., Earley, L., Adachi, K., Nakai, H. Distinct intracellular localization of AAPs derived from AAV serotypes 1-11 and their ability to cross-complementation in heterologous capsid assembly. The 18th Annual Meeting of the American Society of Gene and Cell Therapy, New Orleans, LO, May 13-16, 2015.
Adachi, K., Klocke, K., Roy, P., Rodricks, D., Chapman, M.S., Nakai, H. Capacity of viral genome packaging and internal volumes of AAV viral particles. The 18th Annual Meeting of the American Society of Gene and Cell Therapy, New Orleans, LO, May 13-16, 2015.
Adachi, K., Yoshida, K., Dissen, G.A., Lomniczi, A., Ojeda, S.R., Nakai, H. Very low prevalence of circulating antibodies against common AAV serotypes in a population of cats. The 18th Annual Meeting of the American Society of Gene and Cell Therapy, New Orleans, LO, May 13-16, 2015.
Fuchs, C., Viggiano, R., Trazzi, S., De Franceschi, M., Bartesaghi, R., Nakai, H., Ciani E. GSK3β-targeted therapy in CDKL5 disorder. Rare Disorders Research Consortium Conference, Oregon Health and Science University, Portland OR, April 7, 2015
Rodricks, D., Klocke, K., Adachi, K., Ido, H., Earley, L., Nakai, H. A study of adeno-associated virus (AAV)-based gene therapy vector using the CRISPR-Cas9 system. Saturday Academy Apprenticeships in Science and Engineering (ASE) Program Symposium. Portland, OR, August 15, 2014.
Baumgart, J., Nakai, H. Functional characterization of adeno-associated virus (AAV) assembly-activating proteins (AAPs) derived from various serotypes. OHSU Equity and CURE Scientific Research Poster Presentations, Portland, OR, August 8, 2014.
Adachi, K., Ido, H., Holman, T.W., Nakai, H. A universal AAV Barcode-Seq system expressing RNA barcodes from a recombinant AAV viral genome. The 20th Annual Meeting of the Japanese Society of Gene Therapy. Tokyo, Japan, August 6-8, 2014.
Earley, L., Nakai, H. Expression of the adeno-associated virus rep proteins decreases assembly-activating protein levels. The 20th Annual Meeting of the Japanese Society of Gene Therapy. Tokyo, Japan, August 6-8, 2014.
Ido, H., Adachi, K., Holman, T.W., Nakai, H. A novel immunoprecipitation (IP)-Seq-based method for anti-AAV capsid antibody epitope mapping. The 20th Annual Meeting of the Japanese Society of Gene Therapy. Tokyo, Japan, August 6-8, 2014.
Winn, S.R., Koeberl, D.D., Nakai, H., Harding, C.O. Neonatal rAAV2/8 vector administration does not prevent therapeutic response to repeat vector injection in adult Pahenu2/enu2 mice. The 17th Annual Meeting of the American Society of Gene and Cell Therapy, Washington D.C., May 21-24, 2014.
Ido, H., Adachi, K., Holman, T.W., Nakai, H. A novel immunoprecipitation (IP)-Seq-based method for anti-AAV capsid antibody epitope mapping. The 17th Annual Meeting of the American Society of Gene and Cell Therapy, Washington D.C., May 21-24, 2014.
Adachi, K., Ido, H., Holman, T.W., Nakai, H. Development of a universal AAV Barcode-Seq system expressing RNA barcodes. The 17th Annual Meeting of the American Society of Gene and Cell Therapy, Washington D.C., May 21-24, 2014. (Received a Free Poster Print Award).
Earley, L.F., Nakai, H. An intriguing Rep and VP-dependent fate of assembly-activating protein during adeno-associated virus capsid formation. The 17th Annual Meeting of the American Society of Gene and Cell Therapy, Washington D.C., May 21-24, 2014. (Awarded ASGCT Travel Grant).
Adachi, K., Ido, H., Holman, T.W., Nakai, H. Development of a universal AAV Barcode-Seq system expressing RNA barcodes for AAV vector research. Oregon Health and Science University Research Week, Portland, OR, May 5-9, 2014.
Earley, L.F., Nakai, H. Expression of the adeno-associated virus rep gene causes decreased assembly-activating protein levels. Oregon Health and Science University Research Week, Portland, OR, May 5-9, 2014.
Klocke, K., Adachi, K., Nakai, H. Recombinant AAV (rAAV) vectors with greater internal volume: can they package longer genomes? Oregon Health and Science University Research Week, Portland, OR, May 5-9, 2014.
Ido, H., Adachi, K., Holman, T.W., Nakai, H. A novel immunoprecipitation (IP)-Seq-based method for anti-AAV capsid antibody epitope mapping. Oregon Health and Science University Research Week, Portland, OR, May 5-9, 2014.
Holman, T.W., Nakai, H. Genetically engineering phosphorylation and aggregation-resistant tau proteins toward establishing a new approach to treat Alzheimer’s disease. Oregon Health and Science University Research Week, Portland, OR, May 5-9, 2014.
Nakai, H. Beauty of relativity in phenotypic quantification by deep barcode sequencing. Oregon Health and Science University Research Week, Portland, OR, May 5-9, 2014.
Earley, L.F, Nakai, H. Characterization of nucleolar localization signals in assembly activating protein. OHSU Department of Molecular Microbiology and Immunology Retreat, World Forestry Center, Portland, OR, April 18, 2014 (Received Best Poster Award).
Adachi, K., Gesuette R., Dissen, G., Stenzel-Poore, M., Ojeda, S. R., Nakai, H. A high-throughput analysis of the BBB and BCSFB penetrability of >100 adeno-associated virus (AAV) serotype and mutant vectors by massively parallel sequencing. The 20th Annual Blood-Brain Barrier Consortium Meeting in collaboration with International Brain Barriers Society. Sunriver, OR, March 20-22, 2014.
Nakai, H. A high-resolution functional map of adeno-associated virus serotype 9 capsid. OHSU Gene Therapy Symposium, Portland, OR, November 21, 2013.
Earley, L.F., Sun X-X., Dai, M., Nakai, H. Time course of adeno-associated virus capsid formation reveals novel dynamics between viral and host cell proteins. OHSU Gene Therapy Symposium, Portland, OR, November 21, 2013. (Best poster award)
Adachi, K., Ido, H., Holman, T.W., Nakai, H. Comprehensive understanding of functional AAV capsid amino acids by a next generation sequencing-based high-throughput approach: AAV Barcode-Seq. OHSU Gene Therapy Symposium, Portland, OR, November 21, 2013.
Earley, L.F., Sun X-X., Adachi, K., Dai, M., Nakai, H. Characterization of nucleolar localization signals in assembly activating protein. OHSU Department of Molecular Microbiology and Immunology Retreat, OHSU, Portland, OR. April 2013.
Klocke, K., Holman, T.W., Adachi, K., Ido, H., Earley, L., Nakai, H. Development of novel AAV vectors with integrin binding motifs. Saturday Academy Apprenticeships in Science and Engineering (ASE) Program Symposium. Portland, OR, August 16, 2013.
Garcia-Lopez J.M., Adachi, K., Holman, T.W., Earley, L.F., Ido, H., Nakai, H. Genetically engineering a phosphorylation-resistant tau protein. OHSU Equity and CURE Scientific Research Poster Presentations, Portland, OR, August 9, 2013.
Earley, L.F., Sun X-X., Adachi, K., Dai, M., Nakai, H. Identification and characterization of nucleolar localization signals of Assembly Activating Protein of AAV2. The 19th Annual Meeting of the Japanese Society of Gene Therapy. Okayama, Japan, July 4-6, 2013.
Adachi K., Kawano, Y., Dissen, G., Ojeda, S., Nakai, H. Pharmacokinetic analysis of 11 AAV serotypes and 134 AAV mutants in one rhesus macaque by deep sequencing. The 19th Annual Meeting of the Japanese Society of Gene Therapy. Okayama, Japan, July 4-6, 2013.
Adachi, K., Nakai, H. Understanding AAV biology in non-human primates using a DNA-barcoded AAV library/Illumina sequencing-based approach. Oregon Health and Science University Research Week, Portland, OR, May 20-24, 2013.
Earley, L., Nakai, H. Characterization of nucleolar localization mutants of assembly-activating protein. Oregon Health and Science University Research Week, Portland, OR, May 20-24, 2013.
Adachi, K., Kawano, Y., Dissen, G., Ojeda, S., Nakai, H. Simultaneous pharmacokinetic profiling of multiple AAV serotypes and mutants in a non-human primate by AAV barcode-seq. Identification of nucleolar localization sequences in the c-terminal region of assembly-activating protein. The 16th Annual Meeting of the American Society of Gene and Cell Therapy, Salt Lake City, UT, May 15-18, 2013. (Awarded ASGCT Travel Grant).
Earley, L., Sun, X.-X., Adachi, K. Dai, M., Nakai, H. Identification of nucleolar localization sequences in the c-terminal region of assembly-activating protein. The 16th Annual Meeting of the American Society of Gene and Cell Therapy, Salt Lake City, UT, May 15-18, 2013. (Awarded ASGCT Travel Grant).
Earley, L., Adachi, K., Nakai, H. Assembly-activating protein is highly tolerant to point mutations without loss of function: a high-throughput mutagenesis screen utilizing DNA barcoding. The 16th Annual Meeting of the American Society of Gene and Cell Therapy, Salt Lake City, UT, May 15-18, 2013.
Adachi, K., Verga, M., Nakai, H. Creation of liver-detargeting AAV2-derived mutants based on the knowledge of AAV9 capsid functions. The 16th Annual Meeting of the American Society of Gene and Cell Therapy, Salt Lake City, UT, May 15-18, 2013.
Adachi, K. Summers, A., Nakai, H. A unique biological property of an AAV9 mutant showing increased cell attachment in the presence and absence of AAV5. The 16th Annual Meeting of the American Society of Gene and Cell Therapy, Salt Lake City, UT, May 15-18, 2013.
Earley, L., Adachi, K., Nakai, H. Engineering adeno-associated virus assembly-activating protein (AAP) by a directed evolution approach. OHSU Gene Therapy Symposium, Portland, OR, November 29, 2012.
Adachi, K., Enoki, T., Kawano, Y., Nakai, H. Rational design of novel AAV vectors based on a next generation sequencing-based high-throughput approach. OHSU Gene Therapy Symposium, Portland, OR, November 29, 2012.
Neeley, S., Kawano, Y., Adachi, K., Nakai, H. Computational Evolution of Adeno-Associated Virus Supported by Experimental Directed Evolution. OHSU Gene Therapy Symposium, Portland, OR, November 29, 2012.
Fischer, G., Fan, F., Nakai, H., Park, F., Hogan, Q., Yu, H. Differential properties of AAV6- and AAV8-mediated gene transfer to primary sensory neurons in adult rat. Neuroscience 2012, New Orleans, LA, October 13-17, 2012
Summers, A., Neeley, S., Adachi, K., Nakai, H. Creation and characterization of a novel AAV vector that binds to two primary cell surface receptors. Saturday Academy Apprenticeships in Science and Engineering (ASE) Program Symposium. Portland, OR, August 17, 2012.
Knapper, A., Earley, L., Adachi, K., Sanford, L., Hayflick, S., Nakai, H. Establishment of a novel mouse model for PKAN (pantothene kinase-associated neurodegeneration). OHSU Equity and CURE Scientific Research Poster Presentations, Portland, OR, August 10, 2012.
Kawano, Y., Adachi, K., Sun, X., Dai, M., Nakai, H. An arginine/lysine-rich nucleolar localization signal within the AAV2 Assembly-Activating Protein constrains the structural diversity of the AAV2 capsid. The 18th Annual Meeting of the Japanese Society of Gene Therapy. Kumamoto, Japan, June 28-30, 2012.
Adachi, K., Enoki, T., Kawano, Y., Nakai, H. Identification of a galactose binding motif in the AAV9 capsid and its exploitation to create AAV9-like AAV2 vectors. The 18th Annual Meeting of the Japanese Society of Gene Therapy. Kumamoto, Japan, June 28-30, 2012.
Adachi, K., Enoki, T., Kawano, Y., Nakai, H. Tissue-detargeting AAV9 mutants identified by comprehensive alanine scanning of the capsid protein. The 18th Annual Meeting of the Japanese Society of Gene Therapy. Kumamoto, Japan, June 28-30, 2012. (Awarded AnGes Award)
Adachi, K., Enoki, T., Kawano, Y., Nakai, H. Creation of a novel AAV2 vector showing AAV9-like transduction properties by displaying a galactose binding motif on the capsid. The 15th Annual Meeting of the American Society of Gene and Cell Therapy, Philadelphia, PA, May 16-19, 2012
Adachi, K., Enoki, T., Kawano, Y., Nakai, H. A comprehensive analysis of structural and functional significance of the 381 amino acids in the C-terminal half of AAV9 capsid. The 15th Annual Meeting of the American Society of Gene and Cell Therapy, Philadelphia, PA, May 16-19, 2012 (Awarded ASGCT Travel Grant).
Kawano, Y., Adachi, K., Neeley, S., Sun, X., Dai, M., Nakai, H. A potential functional domain rich in basic amino acids within the AAV2 Assembly-Activating Protein (AAP2) constrains the structural diversity of the AAV2 capsid. The 15th Annual Meeting of the American Society of Gene and Cell Therapy, Philadelphia, PA, May 16-19, 2012
Kawano, Y., Adachi, K. Nakai, H. A basic amino acid-rich nucleolar localization signal within the AAV2 assembly-activating protein constrains the structural diversity of the AAV2 capsid. Oregon Health and Science University Research Week, Portland, OR, May 7-10, 2012
Adachi, K., Enoki, T., Kawano, Y., Nakai, H. Rational design of novel adeno-associated virus vectors by a next generation sequencing-based approach (Barcode-Seq) to study virus biology. Oregon Health and Science University Research Week, Portland, OR, May 7-10, 2012
Adachi, K., Enoki, T., Kawano, Y., Nakai, H. A high-throughput approach to develop novel AAV vectors with the most desirable biological properties toward clinical translation. Rare Disorders Research Consortium Conference, Oregon Health and Science University, Portland OR, February 29, 2012
Tatsumi, K., Ohashi, K., Tateno, C., Nakai, H., Ehrhardt, A., Utoh, R., Yoshizato, K., Okano, T. Propagation and genetic modification of autologous hepatocytes toward gene and cell therapy for hemophilia B. XXIII Congress of The International Society on Thrombosis and Haematostasis. Kyoto, Japan, July 23-29, 2011.
Enoki, T., Adachi, K., Naitza, C. S. and Nakai, H. Assessment of AAV Assembly-Activating Protein (AAP) Function by Using A Simple and Serotype-Independent Method. The 17th Annual Meeting of the Japanese Society of Gene Therapy. Fukuoka, Japan, July 14-17, 2011.
Adachi, K. and Nakai, H. A New Insight into The Dose-Response in rAAV Vector Integration Frequency in The Liver. The 16th Annual Meeting of the Japanese Society of Gene Therapy. Fukuoka, Japan, July 14-17, 2011.
Adachi, K., Enoki, T., Naitza, C. S. and Nakai, H. A Novel High-Throughput Approach to Analyze Adeno-Associated Virus Capsid Functions Using Barcoded Virus Libraries and A Deep Sequencing Technology. The 17th Annual Meeting of the Japanese Society of Gene Therapy. Fukuoka, Japan, July 14-17, 2011. (Awarded Journal of Gene Medicine Young Investigator Award)
Adachi, K., Naitza, C. S., Huang, X. N., Ahmad, F. and Nakai, H. Development of an Effective AAV Vector-Based Gene Therapy To Treat the PRKAG2-Tg Mice, an Animal Model of Autosomal Dominant Progressive Cardiomyopathy. The 14th Annual Meeting of the American Society of Gene and Cell Therapy, Seattle, WA, May 18-21, 2011
Charan, R., Niizawa, G. A., Nakai, H., and Clemens, P. R. Exploring the Role of Deubiquitinating Enzyme A20 for Treatment of Muscular Dystrophy in mdx Mice. The 14th Annual Meeting of the American Society of Gene and Cell Therapy, Seattle, WA, May 18-21, 2011
Niizawa, G. A., Nakai, H., Wang, B. and Clemens, P. R. Molecular Treatment of Muscle Atrophy Caused by Focal Nerve Injury. The 14th Annual Meeting of the American Society of Gene and Cell Therapy, Seattle, WA, May 18-21, 2011
Enoki, T., Adachi, K., Naitza, C. S. and Nakai, H. Development of a Simple and Serotype-Independent System To Assess the AAV Assembly-Activating Protein (AAP) Function. The 14th Annual Meeting of the American Society of Gene and Cell Therapy, Seattle, WA, May 18-21, 2011.
Adachi, K., Enoki, T., Naitza, C. S. and Nakai, H. A Potential Breakthrough in the AAV Biology: A Novel High-Throughput Capsid Functional Analysis Using Barcoded AAV Libraries and a Deep Sequencing Technology. The 14th Annual Meeting of the American Society of Gene and Cell Therapy, Seattle, WA, May 18-21, 2011.
Wang, Z., Nakai, H. and Grompe, M. Comprehensive Integration Site Analysis of One New Gene Therapy Vector AAV-rDNA. The 14th Annual Meeting of the American Society of Gene and Cell Therapy, Seattle, WA, May 18-21, 2011.
Huang, B. and Nakai, H. Establishment and Use of a Configuration-Specific Method To Determine the Structure of AAV-ITRs Recombined by DNA Repair Pathways. The 14th Annual Meeting of the American Society of Gene and Cell Therapy, Seattle, WA, May 18-21, 2011.
Adachi, K. and Nakai, H. A Distinctive Fate of rAAV Vector Genomes in the Liver Transduced with Very Low Doses of rAAV8 in Mice. The 14th Annual Meeting of the American Society of Gene and Cell Therapy, Seattle, WA, May 18-21, 2011.
Adachi, K., Enoki, T., Naitza, C.S., Allen, R. and Nakai, H. A potential breakthrough in the adeno-associated virus capsid functional analysis using barcoded virus libraries and a deep sequencing technology. MVM Retreat, University of Pittsburgh, Pittsburgh, PA, April 29, 2011.
Adachi, K. and Nakai, H. AAV1.9-3 exhibiting significantly impaired infectivity is a novel detargeted platform of AAV random peptide display libraries. Science2010-Transformations, University of Pittsburgh, Pittsburgh, PA, October 8, 2010.
Adachi, K., Kotchey, N.M., Inagaki, K., Zahid, M., Charan, R., Watkins, S.C., Parker, S. and Nakai, H. Transvascular transport of recombinant AAV9 vector is a capacity-limited caveolin--independent slow process that limits cardiac transduction. The 16th Annual Meeting of the Japanese Society of Gene Therapy. Tochigi, Japan, July 1-3, 2010.
Ohashi, K., Tatsumi, K., Tateno, C., Nakai, H., Ehrhardt, A., Utoh, R., Yoshizato, K., and Okano, T. Hepatocyte engineering and genetic modification approaches of hemophilia hepatocytes within a living mouse. The 13th Annual Meeting of the American Society of Gene Therapy, Washington, DC, May 19-22, 2010.
Eghtesad, S., Zheng., H, Nakai, H., Epperly, M.W., and Clemens, P.R. Irradiation of adult mdx mice prior to full-length murine dystrophin cDNA transfer results in a delayed and diminished anti-dystrophin host immune response. The 13th Annual Meeting of the American Society of Gene Therapy, Washington, DC, May 19-22, 2010.
Adachi, K, Kotchey, N, Zahid, M, and Nakai, H. Intravenously administered rAAV9 vector efficiently transduces tissues in vivo via caveolin-1-independent transport across the capillary endothelial cell barrier. The 13th Annual Meeting of the American Society of Gene Therapy, Washington, DC, May 19-22, 2010.
Adachi, K, Kotchey, N, Tobita, K, and Nakai, H. AAV1.9-3 exhibiting significantly impaired infectivity and substantially delayed blood clearance, is a novel detargeted platform of AAV random peptide display libraries. The 13th Annual Meeting of the American Society of Gene Therapy, Washington, DC, May 19-22, 2010.
Kang, Y., Kotchey, N., Zahid, M., and Nakai, H. A mechanism of robust heart transduction with recombinant adeno-associated virus type 9 (AAV9), the cardiotropic serotype. The 15th Annual Meeting of the Japanese Society of Gene Therapy. Osaka, Japan, July 9-11, 2009.
Kang, Y., Kotchey, N., Zahid, M., and Nakai, H. Insights into the mechanism of robust heart transduction with rAAV serotype 9 vectors. The 12th Annual Meeting of the American Society of Gene Therapy, San Diego, CA, May 27-31, 2009.
Piao, C., Inagaki, K., Kotchey, N., Ma, C. and Nakai, H. Similar and differential involvement of DNA-PKcs and Artemis in single-stranded and double-stranded rAAV vector genome processing in mice. The 11th Annual Meeting of the American Society of Gene Therapy. Boston, MA, May 28-June 1, 2008 (Travel Grant Awarded).
Piao, C., Inagaki, K., Kotchey, N., Ma, C., Charan, R., Parker, R.S. and Nakai, H. Pharmacokinetic studies of various serotype and variant rAAV vectors in mice reveal distinct features of rAAV serotype 9. The 11th Annual Meeting of the American Society of Gene Therapy. Boston, MA, May 28-June 1, 2008.
Shin, J.-H., Ohshima, S., Nishiyama, A., Yuasa, K., Nakai, H., Okada, T. and Takeda, S. Efficient systemic delivery of rAAV8 into dystrophic subcutaneous injection. The 13th Annual Meeting of the Japanese Society of Gene Therapy. Nagoya, Japan, June 28-26, 2007.
Ohshima, S., Shin, J.H., Nishiyama, A., Yuasa, K., Nakai, H., Okada, T. and Takeda, S. Distinct transduction profiles in the dystrophic dogs with rAAV serotype 8. The 10th Annual Meeting of the American Society of Gene Therapy. Seattle, WA, May 30-June 3, 2007.
Nishiyama, A., Ampong, B.N., Shin, J.H., Nakai, H., Okada, T. and Takeda, S. rAAV type 8-mediated extensive therapeutic gene delivery into skeletal muscle of α-sarcoglycan deficient mice. The 10th Annual Meeting of the American Society of Gene Therapy. Seattle, WA, May 30-June 3, 2007.
Nishiyama, A., Ampong, B.N., Shin, J.-H., Nakai, H., Okada, T. and Takeda, S. Safe and extensive rAAV8-mediated α -SG transduction of the limb-girdle muscular dystrophy type 2D model mice. The 13th Annual Meeting of the Japanese Society of Gene Therapy. Nagoya, Japan, June 28-26, 2007.
Inagaki, K., Ma, C., Storm, T.A., Kay, M.A. and Nakai, H. Sequencing of rAAV genome termini in wild type and SCID mouse tissues unravels a role of artemis/DNA-PKcs in opening AAV-ITR hairpins in vivo. The 10th Annual Meeting of the American Society of Gene Therapy. Seattle, WA, May 30-June 3, 2007.
Inagaki, K., Ma, C. and Nakai, H. rAAV vectors with a prolonged half-life: implications for the improvement of rAAV gene therapy. The 13th Annual Meeting of the Japanese Society of Gene Therapy. Nagoya, Japan, June 28-26, 2007.
Inagaki, K., Lewis, S.M., Wu, X., Munroe, D., Fuess, S., Storm, T.A., Kay, M.A. and Nakai, H. The prevalence and characterization of break-prone DNA palindromes in a mammalian genome. Keystone Symposia, Genome Instability and Repair, Breckenridge, CO, January 17-22, 2007 (Scholarship Awarded).
Inagaki, K., Lewis, S.M., Wu, X., Ma, C., Munroe, D.J., Fuess, S., Storm, T.A., Kay, M.A. and Nakai, H. AAV labeling of break-prone palindromes in mammalian genomes: Exploitation of the in vivo rAAV biology for studies of genomic instability in living animals. The 10th Annual Meeting of the American Society of Gene Therapy. Seattle, WA, May 30-June 3, 2007.
Inagaki, K., Lewis, S.M., Wu, X., Ma, C., Munroe, D., Fuess, S., Storm, T.A., Kay, M.A. and Nakai, H. DNA palindromes are a prevalent and significant source of DNA breaks in the mammalian genome. American Association for Cancer Research Annual Meeting, Los Angeles, CA, April 14-18, 2007.
Fawcett, P., Nakai, H., Ehrhardt, A., Kay, M.A. and McCaffrey, A.P. Host response to adenovirus, helper-dependent adenovirus and adeno-associated virus in mouse liver. The 10th Annual Meeting of the American Society of Gene Therapy. Seattle, WA, May 30-June 3, 2007.
Ohshima, S., Nishiyama, A., Yuasa, K., Nakamura, A., M., Y., Miyagoe-Suzuki, Y., Nakai, H. and Takeda, S. A Recombinant AAV-Mediated Gene Transfer Into Canine Skeletal Muscle. The 9th Annual Meeting of the American Society of Gene Therapy. Baltimore, MA, May 31-June 4, 2006.
Nishiyama, A., N., B.A., Yuasa, K., Nakai, H. and Takeda, S. Efficacy Of Adeno-Associated Virus Serotype 8 In Alpha-Sarcoglycan Deficient Mice. The 9th Annual Meeting of the American Society of Gene Therapy. Baltimore, MA, May 31-June 4, 2006.
Inagaki, K., Storm, T.A., Kay, M.A. and Nakai, H. Revisiting rAAV Vector Integration in scid Mice: DNA-PKcs Deficiency Does Not Substantially Increase Integration Frequency in Hepatic and Non-Hepatic Tissues In Vivo. The 9th Annual Meeting of the American Society of Gene Therapy. Baltimore, MA, May 31-June 4, 2006.
Inagaki, K., Storm, T.A., Kay, M.A. and Nakai, H. Mechanisms of Hairpin Loop Opening of “Closed” AAV-ITRs by Specific Cellular Endonuclease Activities, a Prerequisite for rAAV Vector Genome Recombinations In Vivo. The 9th Annual Meeting of the American Society of Gene Therapy. Baltimore, MA, May 31-June 4, 2006 (Travel Grant Awarded).
Inagaki, K., Monga, S.P.S., Storm, T.A., Fuess, S., Kay, M.A. and Nakai, H. AAV8 Vector Injection At Birth Significantly Augments the Progression Of Liver Tumors In HBV Transgenic Mice Predisposed To Hepatocellular Carcinoma. The 12th Annual Meeting of the Japanese Society of Gene Therapy. Tokyo, Japan, August 24-26, 2006.
Inagaki, K., Fuess, S., Storm, T.A., Kay, M.A. and Nakai, H. Biology of rAAV8 in Mouse Liver Following Vector Administration at Birth. The 9th Annual Meeting of the American Society of Gene Therapy. Baltimore, MA, May 31-June 4, 2006.
Inagaki, K., Wu, X., Fuess, S., Storm, T.A., Kay, M.A. and Nakai, H. Analysis of Vector Genome Integration Sites in Various Tissues Following Systemic Administration of AAV Serotype 8 Vector in Mice. The 11th Annual Meeting of the Japanese Society of Gene Therapy. Tokyo, Japan, July 28-30, 2005.
Inagaki, K., Wu, X., Fuess, S., Storm, T.A., Kay, M.A. and Nakai, H. Analysis of AAV Serotype 8 Vector Integration in Normal and DNA-PKcs-Deficient Scid Mice by a Novel Strategy. The 8th Annual Meeting of the American Society of Gene Therapy. St. Louis, MO, June 1-5, 2005.
Inagaki, K., Storm, T.A., Fuess, S., Kay, M.A. and Nakai, H. In Vivo Characterization Of AAV Serotype 9 Vectors In Mice. The 8th Annual Meeting of the American Society of Gene Therapy. St. Louis, MO, June 1-5, 2005.
Nakai, H., Wu, X., Fuess, S., Storm, T.A., Burgess, S., Grompe, M. and Kay, M.A. Hot Spots for rAAV2 Vector Integration In Mice. The 7th Annual Meeting of the American Society of Gene Therapy, Minneapolis, MN, June 2-6, 2004.
Nakai, H., Storm, T.A., Fuess, S. and Kay, M.A. Unrestricted Liver Transduction with AAV Serotype 8 Vector in Mice. The 7th Annual Meeting of the American Society of Gene Therapy, Minneapolis, MN, June 2-6, 2004.
Nakai, H., Muramatsu, S., Nara, Y., Fuess, S., Storm, T.A. and Kay, M.A. Unrestricted liver transduction with AAV serotype 8 vectors in mice. The 10th Annual Meeting of the Japanese Society of Gene Therapy. Tokyo, Japan, August 5-7, 2004.
Grimm, D., Streets, K.L., Storm, T.A., Nakai, H., McCaffrey, A.P., Huang, Z., Salazar, F.H., Marion, P.L. and Kay, M.A. Complete Inhibition of Hepatitis B Virus Gene Expression In Vivo with Short Hairpin RNA Expressed from a Novel Double-Stranded, Bi-Cistronic Adeno-Associated Virus Pseudotype 8 Vector. The 7th Annual Meeting of the American Society of Gene Therapy, Minneapolis, MN, June 2-6, 2004.
Grimm, D., Nakai, H., Storm, T.A., Riu, E.P. and Kay, M.A. Transgene Expression from Recombinant AAV8 Vectors in the Liver Occurs Independently of the Serotype Origin of the Viral Inverted Terminal Repeats. The 7th Annual Meeting of the American Society of Gene Therapy, Minneapolis, MN, June 2-6, 2004.
Ohashi, K., Nakai, H., Couto, L.B., Nakajima, Y. and Kay, M.A. Delivery parameters affecting liver-directed AAV transduction. The 6th Annual Meeting of the American Society of Gene Therapy, Washington, DC, June 4-8, 2003.
Nakai, H., Storm, T.A., Fuess, S. and Kay, M.A. Pathways of removal of free DNA vector ends in mouse hepatocytes transduced with rAAV vectors. The 9th Annual Meeting of the Japanese Society of Gene Therapy, Tokyo, Japan, July 18-20, 2003.
Nakai, H., Montini, E., Fuess, S., Storm, T.A., Grompe, M. and Kay, M.A. AAV serotype 2 vectors preferentially integrate into active genes in mice. The 9th Annual Meeting of the Japanese Society of Gene Therapy, Tokyo, Japan, July 18-20, 2003.
Nakai, H., Montini, E., Fuess, S., Storm, T.A., Grompe, M. and Kay, M.A. AAV serotype 2 vectors preferentially integrate into active genes in mice. The 6th Annual Meeting of the American Society of Gene Therapy, Washington, DC, June 4-8, 2003 (Received Travel Award).
McCaffrey, A.P., Nakai, H., Pandey, K., Huang, Z., Salazar, F.H., Marion, P.L. and Kay, M.A. Inhibition of Hepatitis B virus by RNA interference in mice. The 6th Annual Meeting of the American Society of Gene Therapy, Washington, DC, June 4-8, 2003.
Grimm, D., Zhou, S., Nakai, H., Thomas, C.E., Storm, T.A., Fuess, S., Matsushita, T., Surosky, R., Lochrie, M., Meuse, L., McClelland, A., Colosi, P. and A., K.M. Preclinical in vivo evaluation of pseudotyped AAV vectors for liver gene therapy. The 6th Annual Meeting of the American Society of Gene Therapy, Washington, DC, June 4-8, 2003.
Zhou, S., Christie, B., Wellman, J., Allen, J., Akeefe, H., Surosky, R., Lochrie, M., Grimm, D., Thomas, C.E., Nakai, H., Kay, M.A., McClelland, A. and Colosi, P. A simple, efficient, and general method for the production of AAV type 1-6 vectors. The 5th Annual Meeting of the American Society of Gene Therapy, Boston, MA, June 5-9, 2002.
Thomas, C.E., Nakai, H., Storm, T.A., Meuse, L., Powell, S., Wright, F. and Kay, M.A. Linear increase in hFIX expression, proportional to vector dose are obtained from rAAV-hFIX mediated gene transfer to mouse liver. The 5th Annual Meeting of the American Society of Gene Therapy, Boston, MA, June 5-9, 2002.
Parker, A., Nagy, D., Powell, S.K., Qu, G., Wright, J.F., Dake, M., Razavi, M., Nakai, H., Kay, M.A., McClelland, A. and Couto, L.B. Preclinical safety testing of AAV-hFIX vectors purified by CsCl centrifugation or column chromatography. The 5th Annual Meeting of the American Society of Gene Therapy, Boston, MA, June 5-9, 2002.
Nakai, H., Thomas, C.E., Storm, T.A., Fuess, S., Powell, S., Wright, F. and Kay, M.A. High concentrations of rAAV administered to liver result in a non-linear dose response. The 8th Annual Meeting of the Japanese Society of Gene Therapy, Tokyo, Japan, July 18-20, 2002.
Nakai, H., Thomas, C.E., Storm, T.A., Fuess, S., Powell, S., Wright, F. and Kay, M.A. High concentration of rAAV administrated to liver results in a non-linear dose response. The 5th Annual Meeting of the American Society of Gene Therapy, Boston, MA, June 5-9, 2002.
Nakai, H., Storm, T.A., Fuess, S. and Kay, M.A. A mechanism for free DNA vector end removal of recombinant adeno-associated virus vector DNA in vivo. The 44th Annual Meeting of the American Society of Hematology, Philadelphia, PA, December 6-10, 2002.
Nakai, H., Montini, E., Fuess, S., Storm, T.A., Meuse, L., Finegold, M., Grompe, M. and Kay, M.A. Host chromosomal effects of naked plasmid DNA vector integration in mice. The 8th Annual Meeting of the Japanese Society of Gene Therapy, Tokyo, Japan, July 18-20, 2002.
Nakai, H., Montini, E., Fuess, S., Storm, T.A., Meuse, L., Finegold, M., Grompe, M. and Kay, M.A. Helper-independent chromosomal integration of naked double-stranded linear DNA vectors in mice. The 8th Annual Meeting of the Japanese Society of Gene Therapy, Tokyo, Japan, July 18-20, 2002.
Nakai, H., Montini, E., Fuess, S., Storm, T.A., Meuse, L., Finegold, M., Grompe, M. and Kay, M.A. Helper-independent chromosomal integration of naked double-stranded linear DNA vectors in mouse liver in vivo. The 5th Annual Meeting of the American Society of Gene Therapy, Boston, MA, June 5-9, 2002.
Nakai, H., Montini, E., Fuess, S., Storm, T.A., Grompe, M. and Kay, M.A. Host chromosomal effects of recombinant adeno-associated virus vector integration in vivo. The 44th Annual Meeting of the American Society of Hematology, Philadelphia, PA, December 6-10, 2002.
Nakai, H., Storm, T.A., Yant, S.R., Fuess, S., Meuse, L. and Kay, M.A. Episomal recombinant adeno-associated virus vector genomes are primarily responsible for stable liver transduction in vivo. The 7th Annual Meeting of the Japanese Society of Gene Therapy, Tokyo, Japan, July 5-7, 2001.
Nakai, H., Storm, T.A., Yant, S.R., Fuess, S., Meuse, L. and Kay, M.A. Episomal Recombinant Adeno-Associated Virus Vector Genomes are Primarily Responsible for Stable Liver Transduction in vivo. The 4th Annual Meeting of the American Society of Gene Therapy, Seattle, WA, May 30-June 3, 2001 (Received Travel Award).
Nakai, H., Storm, T.A. and Kay, M.A. Mismatch repair of heteroduplex rAAV genomes in hepatocytes: additional support for annealing of complementary ss vector genomes as a mechanism for the formation of stable ds genomes in vivo. Keystone Symposia, Gene Therapy 2001: A Gene Odyssey, Snowbird, UT, January 6 - 11, 2001.
Nakai, H., Ohashi, K., Arruda, V., McClelland, A., Couto, L.B., Meuse, L., Storm, T.A., Dake, M.D., Razavi, M., Manno, C.S., Glader, B., High, K.A. and Kay, M.A. A proposed rAAV-liver directed clinical trial for hemophilia B. The 7th Annual Meeting of the Japanese Society of Gene Therapy, Tokyo, Japan, July 5-7, 2001.
Nakai, H., Fuess, S., Storm, T.A., Meuse, L. and Kay, M.A. Identification of intermediates for rAAV vector genome concatemerization and integration in hepatocytes in vivo: evidence for double-stranded linear monomer genomes not circles as reactive intermediates. The 7th Annual Meeting of the Japanese Society of Gene Therapy, Tokyo, Japan, July 5-7, 2001.
Nakai, H., Fuess, S., Storm, T.A., Meuse, L. and Kay, M.A. Identification of Intermediates for Recombinant Adeno-associated Virus Vector Genome Concatemerization and Integration in Hepatocytes in vivo: Evidence for Double-Stranded Linear Monomer Genomes not Circles as Reactive Intermediates. The 4th Annual Meeting of the American Society of Gene Therapy, Seattle, WA, May 30-June 3, 2001.
Kay, M.A., Nakai, H., Couto, L.B., Nichols, T., Mount, J., Lothrop, C.D.J., Glader, B., Manno, C.S., Arruda, V., Herzog, R.W. and High, K.A. Safety and efficacy studies of AAV-mediated, liver-directed gene transfer for hemophilia B. The XVIII Congress of the International Society on Thrombosis and Haemostasis, Paris, France, July 6-12, 2001.
Kay, M.A., Miao, C.H., Ohashi, K., Arruda, V., McClelland, A., Couto, L.B., Meuse, L., Storm, T.A., Dake, M.D., Razavi, M., Manno, C.S., Glader, B., High, K.A. and Nakai, H. A proposed rAAV-liver-directed clinical trial for hemophilia B. The 4th Annual Meeting of the American Society of Gene Therapy, Seattle, WA, May 30-June 3, 2001.
Nakai, H., Storm, T.A. and Kay, M.A. Recruitment of single-stranded recombinant adeno-associated viral vector genomes and intermolecular recombination are responsible for stable in vivo liver transduction. The 6th Annual Meeting of the Japanese Society of Gene Therapy, Tokyo, Japan, July 27-29, 2000.
Nakai, H., Storm, T.A. and Kay, M.A. Increasing the size of rAAV-mediated expression cassettes in vivo by intermolecular joining of two complementary vectors. The 3rd Annual Meeting of the American Society of Gene Therapy. Denver, CO, May 31 - June 4, 2000.
Nakai, H., Storm, T.A. and Kay, M.A. Annealing of complimentary single-stranded genomes and subsequent intermolecular joining is the mechanism of stable in vivo liver transduction by recombinant adeno-associated virus vectors. The 3rd Annual Meeting of the American Society of Gene Therapy. Denver, CO, May 31 - June 4, 2000 (Received Young Investigator Award and Travel Award).
Nakai, H., Ohashi, K., Arruda, V., McClelland, A., Couto, L.B., Meuse, L., Storm, T.A., Dake, M.D., Manno, C.S., Glader, B., High, K.A. and Kay, M.A. A proposed rAAV-liver directed clinical trial for hemophilia B. The 42th Annual Meeting of the American Society of Hematology, San Francisco, December 1-5, 2000.
Nakai, H., Fuess, S., Meuse, L., Storm, T.A. and Kay, M.A. Persistent hFIX expression in mouse hepatocytes from episomal rAAV circular intermediates does not rely on the presence of AAV-ITR but the structure of expression cassette itself. The 42th Annual Meeting of the American Society of Hematology, San Francisco, CA, December 1-5, 2000.
Nakai, H., Couto, L.B., Storm, T.A., Burton, M., Colosi, P. and Kay, M.A. Complementary recombinant adeno-associated virus (rAAV) vectors: two novel strategies to overcome the limited packaging size of current rAAV vectors. The 6th Annual Meeting of the Japanese Society of Gene Therapy, Tokyo, Japan, July 27-29, 2000.
Nakai, H., Iwaki, Y. and Couto, L.B. Recombinant AAV Vectors Integrate into the Host Genomes in Mouse Liver: Isolation of rAAV Vector-Cellular DNA Junctions. Keytone Symposia, Molecular and Cellular Biology of Gene Therapy, Salt Lake City, UT, January 14-20, 1999.
Couto, L.B., Burton, M., Colosi, P., Cunningham, J. and Nakai, H. Co-expression of factor VIII heavy and light chains using AAV vectors leads to biologically active protein. Keytone Symposia, Molecular and Cellular Biology of Gene Therapy, Salt Lake City, UT, January 14-20, 1999.
Nakai, H., Podsakoff, G.M., Colosi, P., Iwaki, Y., Kurtzman, G.J. and Couto, L.B. Persistent expression of circulating human coagulation factor IX in mice carrying inhibitors following intramuscular expression of an AAV vector. Keystone Symposia on Molecular and Cellular Biology of Gene Therapy, Keystone, CO, January 19-25, 1998.
Nakai, H., Kurtzman, G., Iwaki, Y., Colosi, P. and Couto, L.B. AAV-hFIX Delivery to Mouse Liver: A Comparison of Vectors and Routes of Administration. The 4th Annual Meeting of the Japanese Society of Gene Therapy, Tokyo, Japan, July 4-5, 1998.
Nakai, H., Iwaki, Y. and Couto, L.B. Recombinant AAV Vector Integrates into Host Genome in Mouse Liver. Cold Spring Harbor Gene Therapy Meeting, Cold Spring Harbor, NY, September, 1998.
Nakai, H., Couto, L.B., Herzog, R.W., Hagstrom, J.N., Walter, J., Kung, S.H., Yang, E.Y., Tai, S.J., Iwaki, Y., Kurtzman, G.J., Colosi, P. and High, K.A. AAV-Mediated Gene Transfer of Human Blood Coagulation Factor IX into Mouse Liver. The 1st Annual Meeting of the American Society of Gene Therapy. Seattle, WA, May 28-30, 1998.
Nakai, H., Kurtzman, G., Iwaki, Y., Colosi, P. and Couto, L.B. Secretion of human FIX following administration of AAV vectors to muscle and liver. The 3rd Annual Meeting of the Japan Society of Gene Therapy, Tokyo, Japan, May, 1997.
Nakai, H., Herzog, R.W., Iwaki, Y., Colosi, P., Eisensmith, R., High, K.A., Kurtzman, G. and Couto, L.B. Persistent expression of human coagulation factor IX following administration of AAV vectors to mouse muscle and liver. The 39th Annual Meeting of the American Society of Hematology, San Diego, CA, December 5-10, 1997.
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