In vitro data demonstrated self-complementary AAV9 (scAAV9) enabled ~30-fold higher MeCP2 protein expression compared to single-stranded AAV9 (ssAAV9), supporting the ability to effectively deliver ...

NEW YORK and TRUMBULL, Conn., April 30, 2025 /PRNewswire/ -- Apertura Gene Therapy, a biotechnology company focused on innovative gene therapy solutions, and the Rett Syndrome Research Trust (RSRT), ...

Findings bolster the idea that the functions of this protein -- MeCP2 -- are more centered on nucleosomes, rather than other forms of DNA. One particular protein lies at the heart of brain development ...

HOUSTON (March 4, 2026) – A team of researchers at Texas Children’s Duncan Neurological Research Institute (NRI) and Baylor College of Medicine report in Science Translational Medicine a potential new ...

Rett syndrome is a devastating rare genetic childhood disorder primarily affecting girls. Merely 1 out of 10,000 girls are born with it and much fewer boys. It is caused by mutations in the MeCP2 gene ...

Rett syndrome is a rare neurodevelopment disorder that leads to developmental regression, usually after a period of normal growth, and causes impairments in communication, motor skills, and speech. It ...

Although many studies approach the developmental disorder Rett syndrome as a single condition arising from general loss of ...