Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional
Official Title: A Phase I/IIa Study of RS1 Ocular Gene Transfer for X-linked Retinoschisis
Brief Summary:
Background:
- X-linked juvenile retinoschisis (XLRS) is caused by changes in the RS1 gene. These changes cause abnormal function of the eye protein retinoschisin. Without normal retinoschisin, the layers of the retina split and vision is lost. Researchers want to try to introduce a healthy RS1 gene into eye cells, to see if this helps retinal cells make healthy retinoschisin. They will put the gene in a virus. The gene and virus package is known as a gene transfer vector (AAV-RS1 vector).
Objectives:
- To see if the AAV-RS1 vector is safe to use in people.
Eligibility:
- Adults 18 and older with a mutation of the RS1 gene, 20/63 vision or worse in one eye, and XLRS.
Design: