Gene editing for children with Rett syndrome
Part of Brain & nervous system, Genetic & congenital clinical trials.
This trial tests a gene-editing approach for Rett syndrome in children who have a confirmed MECP2 gene mutation. It may help by targeting specific “hotspot” genetic changes that cause the condition.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must be female (the study includes girls only).
- You must be more than 6 months old.
- Your Rett syndrome diagnosis must be confirmed by genetic testing (NGS).
- Your MECP2 gene test must show one of these specific repeated mutations: T158M, R168X, R255X, or R306C.
- A parent or legal guardian must be willing and able to give informed consent.
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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