Gene Therapy
UshStat gene replacement therapy clinical trial
A participant who has Usher Syndrome – Type I talks about her involvement in the UshStat clinical trial at Casey Eye Institute and what gene replacement therapy means to her.
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Gene Therapy Research Program
Abnormal growth of new blood vessels in the eye, called ocular neovascularization, is the most common cause of permanent blindness in developed countries. Just three diseases are associated with this condition: diabetes, retinopathy of prematurity and age-related macular degeneration (AMD). While they affect different groups of patients, these diseases share one process: uncontrolled cell division, leading to the growth of new blood vessels. These blood vessels damage the retina, causing vision loss.
Trials Currently Recruiting Participants
Trials listed in this section are currently open for enrollment.
X-linked Retinoschisis (XLRS)
The purpose of this study is to learn more about how to follow changes in vision and other symptoms associated with XLRS. This information is needed for the development of future gene therapy trials.
Subretinally Injected Ushstat
A Phase I/IIa Dose Escalation Safety Study of Subretinally Injected UshStat®, Administered to Patients with Retinitis Pigmentosa Associated with Usher Syndrome Type 1B.
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
Stargardt's Macular Degeneration
A Phase I/IIa Dose Escalation Safety Study of Subretinally Injected StarGen, Administered to Patients with Stargardt's Macular Degeneration.
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.
Leber Congenital Amaurosis
This study is investigating an experimental gene therapy in patients with LCA caused by mutations (changes) in the RPE65 gene. Study is currently enrolling patients age 6 years and older.
