Gene-based therapy restores cellular development and function in brain cells from people with Timothy syndrome

Gene-based therapy restores cellular development and function in brain cells from people with Timothy syndrome

In a proof-of-concept study, researchers demonstrated the effectiveness of a potential new therapy for Timothy syndrome, an often life-threatening and rare genetic disorder that affects a wide range of bodily systems, leading to severe cardiac, neurological, and psychiatric symptoms as well as physical differences such as webbed fingers and toes.

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