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.

X-linked Retinoschisis (RS1)

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.

Contact 503 494-0020 or email the ORDC.

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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

Contact 503 494-0020 or email the ORDC.

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. 

Contact 503 494-0020 or email the ORDC.

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Gene therapy for wet AMD (GEM Study)

Purpose: To evaluate the safety and dosing levels of a gene-based treatment, RetinoStat®, for wet AMD. In this study, two helpful genes are delivered directly to the retina, where they "turn on" proteins that block abnormal blood vessel growth in a sustained fashion. Enrollment is completed and study patients are being followed.

Contact: Ann Lundquist, 503 494-6364.