Cell and Gene Therapies

Stem cells: harnessing the regenerative power of the body.


WebImage_neurons from stem cellsThe devastation of Alzheimer's, Parkinson's, and Lou Gehrig's disease spreads far beyond the individual who suffers from these and other neurodegenerative human conditions. As of yet, these disorders are incurable—rendering families and victims helpless to endure the inevitable degeneration of the brain and nervous system. But there is hope. With the regenerative power of stem cells, we have the potential to reverse this fate.

Despite controversies and funding limitations, embryonic stem cell research has significantly enhanced our understanding of systems at the cellular level. These "master" cells of the human body are capable of becoming an unlimited source of cell and tissue types, providing a possible cure for many conditions. 

The greatest challenge in stem cell replacement therapy has been immune-response rejection of transplanted cells. Because stem cells can only be extracted from early embryos, sometimes the human body will attack the genetically dissimilar transplants. That's why researchers at the ONPRC are creating innovative ways of allowing introduced stem cells to avoid rejection. 

Nearly every cell in the human body contains the complete set of genes needed to create a fully functional adult. Using a method called somatic cell nuclear transfer, ONPRC scientists have managed to coax a skin cell to revert back to its embryonic state. This breakthrough means that scientists may be able to one day develop nerve, heart, liver, and other cells that originate from the patient—and avoid the risk of immune system rejection. 

Mitochondrial disease caused by pathogenic germline mitochondrial gene (mtDNA) mutations is maternally transmitted through oocyte. The resulting loss of cellular respiration leads to severe and progressive disorders, involving several different organs with prominent involvement of the central nervous system, cardiac and other vital organs. ONPRC scientists developed mitochondrial replacement therapies (MRT) that could prevent transmission of mtDNA defects from mothers to their children. Preclinical and clinical studies demonstrated feasibility, efficacy and long-term safety of MRT approaches. Clinical applications of MRT have been recently approved in the UK and currently under consideration in USA.

The advances at the ONPRC are accelerating research in this field, taking us closer to treatments and cures for some of the most common human diseases.