Study shows that Rett syndrome in females is not just less severe, but different
Rett syndrome is caused by mutations of the MECP2 gene located on the X chromosome. Children with Rett initially show typical ...
Nature2mon
Modifying MeCP2
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 ...
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Taysha Gene Therapies Reports Full-Year 2024 Financial Results and Provides Corporate Update
About Rett Syndrome Rett syndrome is a rare neurodevelopmental disorder caused by mutations in the X-linked MECP2 gene encoding methyl CpG-binding protein 2 (MeCP2), which is essential for regulating ...
Taysha Gene Therapies, Inc.: Taysha Gene Therapies Reports Full-Year 2024 Financial Results and Provides Corporate Update
High dose and low dose of TSHA-102 continue to be generally well tolerated with no treatment-related SAEs or DLTs in all pediatric, adolescent and adult patients treated (high dose, n=6; low dose, n=4 ...