Experimental gene therapy aims to restore sight in rare childhood blindness
NCT ID NCT05878860
First seen May 01, 2026 · Last updated May 01, 2026
Summary
This study tests a new gene therapy called ATSN-201 for people with X-linked retinoschisis, a rare genetic eye disease that causes vision loss. The trial includes children and adults aged 6 and older, and will check if the treatment is safe and can improve vision. The therapy works by delivering a working copy of the RS1 gene to the retina.
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Bascom Palmer Eye Institute
RECRUITINGMiami, Florida, 33136, United States
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Children's Hospital of Los Angeles
RECRUITINGLos Angeles, California, 90027, United States
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Children's Hospital of Philadelphia
RECRUITINGPhiladelphia, Pennsylvania, 19104, United States
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Oregon Health Sciences University
RECRUITINGPortland, Oregon, 97239, United States
Contact
Conditions
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