Gene therapy

Gene therapy research program

Ocular gene therapy is a promising new investigational procedure that uses genes to treat vision loss. Doctors at the Casey Eye Institute have been participating in gene therapy clinical trials since 2009. We currently offer more ocular gene therapy trials than any other center in the United States.

Ongoing gene therapy trials open to enrollment

These studies are actively seeking new participants. If you are interested in participating or would like moreinformaiton, contact 503 494-0020 or email the ORDC.

Achromatopsia (CNGB3) Gene Therapy Trial 

The purpose of this study is to learn about a new gene therapy that may help patients with Achromatopsia. This is the first study that aims to treat Achromatopsia disease by gene therapy. The study investigators want to find out whether it is safe for use in humans. The gene therapy is given by a surgical injection into the retina (the lining of the back of the eye that detects light) of one eye. The eye with worse vision will receive the gene therapy. 

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Achromatopsia (CNGA3) Gene Therapy Trial 

The purpose of this study is to learn about a new gene therapy that may help patients with Achromatopsia. The study investigators want to find out whether it is safe for use in humans. The gene therapy is given by a surgical injection into the retina (the lining of the back of the eye that detects light) of one eye. The eye with worse vision will receive the gene therapy. 

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Choroideremia (CHM) Natural History Study

The purpose of this study is to learn more about Choroideremia; how this condition affects a person's eyes and vision and add to the knowledge base for this rare disease.

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Stargardt's Macular Degeneration (ABCA4) gene therapy trial

The purpose of this study is to learn about a new gene therapy that may help patients with Stargardt's Macular Degeneration (SMD). This is the first study that aims to treat Stargardt's disease by gene therapy. The study investigators want to find out whether it is safe for use in humans. The gene therapy is given by a surgical injection underneath the retina of one eye. The eye with worse vision will receive the gene therapy. 

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Usher Syndrome Type 1B (MYO7A) gene therapy trial

The purpose of this study is to learn about a new gene therapy being studied in patients with Retinitis Pigmentosa (RP) as a result of Usher Syndrome.This is the first study that aims to treat RP due to Usher Syndrome by gene therapy.The study investigators want to find out if UshStat is safe for use in humans.The gene therapy is given by surgical injection underneath the retina of one eye.The eye with worse vision will receive the gene therapy

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Usher Syndrome Type 2A (USH2A) Natural History Study

The purpose of this research study is to learn more abotu Usher Syndrome type 2A and Retinitis Pigmentosa caused by specific changes in the USH2A gene and how these conditions affect a person's eyes and vision.

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X-Linked Retinitis Pigmentosa (RPGR) Gene Therapy Trial

The purpose of this study is to learn about a new investigational gene therapy that may help patients with XLRP. The study investigators want to find out whether it is safe and to see if it can improve your vision and other symptoms of XLRP. The gene therapy is given by a surgical injection into the retina (the lining of the back of the eye that detects light) of one eye. The eye with the worse vision will receive the gene therapy. The study is sponsored by AGTC.

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X-Linked Retinitis Pigmentosa (RPGR) Natural History Study

The purpose of this study is to learn about a condition called X-Linked Retinitis Pigmentosa (XLRP) caused by changes in the RPGR gene. The study investigators want to find out how changes in the RPGR gene affect vision, and what tests are more helpful for tracking changes in a person's eyes and vision. This study is sponsored by AGTC.

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X-Linked Retinitis Pigmentosa (RPGR) Natural History Study

The purpose of this study is to learn more about a condition called X-Linked Retinitis Pigmentosa (XLRP). The study investigators want to gain a better understanding of disease progression over time in subjects with SLRP. The study is sponsored by Nightstar. 

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X-linked Retinoschisis (RS1) Gene Therapy Trial

The purpose of this study is to learn about a new gene therapy that may help patients with X-Linked Retinoschisis (XLRS). This is the first study that aims to treat XLRS disease by gene therapy. The study investigators want to find out whether it is safe for use in humans. The gene therapy is given by a surgical injection into the vitreous (a thick, gel-like transparent substance that fills the center of the eye) of one eye. The eye with worse vision will receive the gene therapy.

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