In the long human effort to meet suffering with understanding, a young MIT researcher named Shannon Knight has turned a childhood encounter with a human brain into a doctoral pursuit that may one day spare infants from a life of unrelenting seizures. Working at the intersection of CRISPR gene editing and rare neurological disease, Knight is developing a therapy for SYNGAP1 haploinsufficiency — a genetic disorder that begins dismantling a child's neurological life as early as four months old. Her early results in mice suggest that targeting the root cause of the disorder, rather than its sympto
MIT Researcher Develops CRISPR Gene Therapy for Rare Childhood Epilepsy
SYNGAP1 disorder affects children starting at 4 months old, causing seizures, intellectual disabilities, and movement difficulties that worsen with age as medication resistance develops.