As RTT patients are mosaic with respect to MeCP2 expression owing to random X-chromosome inactivation, gene therapy-based strategies for treatment of RTT that involve increasing MeCP2 expression ...

Rett syndrome is caused by mutations of the MECP2 gene located on the X chromosome. Children with Rett initially show typical ...

TSHA-102 is a self-complementary intrathecally delivered AAV9 investigational gene transfer therapy in clinical evaluation for Rett syndrome. Designed as a one-time treatment, TSHA-102 aims to address ...