Scientists test One-Time gene fix for devastating rare disease
NCT ID NCT03897361
Summary
This early-stage study tested a one-time gene therapy for cystinosis, a rare and serious genetic disorder that causes cystine to build up and damage organs. Researchers collected stem cells from 6 patients, used a virus to insert a corrected gene into those cells, and then infused them back into the patients after chemotherapy. The main goals were to see if this process was safe and if it could reduce harmful cystine levels in the body.
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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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