Gene therapy trial offers new hope for cystinosis patients
NCT ID NCT03897361
First seen Jan 19, 2026 · Last updated May 15, 2026 · Updated 18 times
Summary
This study tested a new gene therapy for cystinosis, a rare genetic disease that causes harmful crystals to build up in cells. Researchers took stem cells from 6 patients, corrected the faulty gene in a lab, and gave the cells back. The goal was to see if this approach is safe and can help control the disease long-term.
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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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Locations
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University of California San Diego
La Jolla, California, 92093, United States
Conditions
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