One-of-a-kind gene therapy targets rare blindness
NCT ID NCT07177196
First seen Nov 01, 2025 · Last updated May 22, 2026 · Updated 26 times
Summary
This study tests a personalized medicine designed for a single person with retinal dystrophy caused by a specific PRPH2 gene mutation. The treatment is an antisense oligonucleotide (ASO) given to try to slow or stop vision loss. The main goals are to check safety and see if the drug affects vision and eye structure.
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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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Locations
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University of California San Diego
San Diego, California, 92093, United States
Conditions
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