First gene therapy injection targets rare brain disorder
NCT ID NCT07173153
First seen Jun 26, 2026 · Last updated Jun 26, 2026
Summary
This early-phase trial tests a one-time gene therapy injection into the spinal fluid for people with SLC6A1 neurodevelopmental disorder, a rare genetic condition. The study involves just 1 participant and will monitor safety and changes in behavior and development over 3 years. The goal is to see if replacing the faulty gene can improve symptoms.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- AAV9.SLC6A1 gene therapy
- What this could lead to
- If successful, this could point toward a treatment that improves symptoms of SLC6A1 neurodevelopmental disorder.
- What could go wrong
- This is a very early, small trial with only 1 participant, so results may not apply to others. Gene therapy also carries risks like immune reactions or toxicity.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Locations
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Nationwide Children's Hospital
Columbus, Ohio, 43205, United States
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