Gene therapy fails to improve vision in advanced retinitis pigmentosa trial
NCT ID NCT02556736
First seen Jan 12, 2026 · Last updated May 12, 2026 · Updated 17 times
Summary
This early-stage study tested a gene therapy called RST-001 in 14 people with advanced retinitis pigmentosa, a genetic eye disease that causes severe vision loss. All participants had very poor vision at the start (could only see hand motions or less). The main goal was to check safety, and the therapy did not improve vision. The study was completed, but no benefit was shown.
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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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Cincinnati Eye Institute- Edgewood /ID# 236713
Edgewood, Kentucky, 41017-3415, United States
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Duke Eye Center /ID# 235715
Durham, North Carolina, 27705, United States
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Retina Foundation of the Southwest /ID# 235199
Dallas, Texas, 75231, United States
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University of California San Francisco - Mission Bay /ID# 235717
San Francisco, California, 94158, United States
Conditions
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