First gene therapy attempt for rare brain disorder begins
NCT ID NCT07173153
First seen Nov 01, 2025 · Last updated May 25, 2026 · Updated 25 times
Summary
This study tests a new gene therapy for people with SLC6A1 neurodevelopmental disorder, a rare genetic condition that affects brain function. One participant will receive a single injection of a harmless virus carrying a working copy of the SLC6A1 gene into the spinal fluid. The main goal is to check safety, and researchers will also track changes in behavior and development over three years.
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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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Genom att skicka in godkänner du våra Användarvillkor
Contacts and locations
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Genom att skicka in godkänner du våra Användarvillkor
Locations
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Nationwide Children's Hospital
Columbus, Ohio, 43205, United States
Conditions
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