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.

from Women's Health News -- ScienceDaily https://ift.tt/vSs0Ftj