Emily de los Reyes
This is gene therapy study of an AAV9 vector carrying the SLCA1 gene for SLC6A1 neurodevelopmental disorder.
Inclusion Criteria: * Confirmation of pathogenic mutation S295L in the SLC6A1 gene * Ability to cooperate and complete assessments per determination of the physician and therapists Exclusion Criteria: * Serological evidence of HIV infection, or Hepatitis B or C infection * Diagnosis of (or ongoing treatment) for an autoimmune disease * Presence of a medical condition or extenuating circumstance that, in the opinion of the Sponsor-Investigator, might compromise the participant's ability to comply with the protocol required testing or procedures or compromise the participant's wellbeing, safe…
This is an open-label, single injection study of an AAV9 vector carrying the SLCA1 coding sequence delivered one time through an intrathecal injection.
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