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SLC6A1

Clinical trials for SLC6A1 explained in plain language.

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  • First gene therapy attempt for rare brain disorder begins

    Disease control ENROLLING_BY_INVITATION

    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 …

    Matched conditions: SLC6A1

    Phase: PHASE1, PHASE2 • Sponsor: Emily de los Reyes • Aim: Disease control

    Last updated May 26, 2026 11:09 UTC

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