Gene-Fixed stem cells show promise against rare kidney disease
NCT ID NCT03897361
First seen Jan 19, 2026 · Last updated May 20, 2026 · Updated 21 times
Summary
This study tested a gene therapy for cystinosis, a rare disease that causes cystine crystals to build up and damage organs. Researchers took blood stem cells from 6 patients, fixed the faulty gene in a lab, and put the cells back. The goal was to see if it's safe and if it can lower cystine levels. This is an early-phase trial, so it focuses on safety and finding the right dose.
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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
La Jolla, California, 92093, United States
Conditions
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