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MIT Researcher Tests CRISPR Gene Therapy for Rare Childhood Epilepsy
MIT PhD candidate Shannon Knight is in the early stages of developing a CRISPR-based gene therapy for SYNGAP1 haploinsufficiency, a rare genetic disorder linked to childhood-onset epilepsy and neurodevelopmental challenges. Preliminary testing in mice with a version of the disorder has shown improvements in seizures and behavioral traits, but the approach has not yet entered human trials.
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