Gene therapy study for SLC6A1 genetic disorder
Part of Brain & nervous system, Genetic & congenital clinical trials.
This trial tests a gene therapy for people with a specific mutation (S295L) in the SLC6A1 gene, which causes a neurodevelopmental disorder. The therapy aims to correct the genetic issue, and participants will be monitored closely for safety and effectiveness.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must have a confirmed S295L mutation in the SLC6A1 gene
- You must be able to cooperate with tests and procedures as determined by the study team
- You cannot have HIV, hepatitis B or C, or an active autoimmune disease
- You must have adequate blood cell counts and liver/kidney function
- You cannot have had a prior reaction to the AAV9 virus antibody test
- You must be willing to tell your primary doctor about being in the study
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
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