Rett syndrome is a rare devastating neurological disorder that primarily affects young girls and manifests as an impaired ability to walk and talk, along with characteristic 'hand-wringing' movements, ...

Gene therapy may offer hope for people with a rare childhood disease called Rett syndrome. Around one in 10,000 girls are born each year with Rett syndrome, a rare genetic condition that impacts brain ...

Researchers have gained new insights into the molecular changes leading to Rett syndrome, a severe neurological disorder caused by mutations in the MeCP2 gene encoding methyl-CpG binding protein 2 ...

SAN FRANCISCO--(BUSINESS WIRE)--Herophilus announced today that in vivo studies underway of its lead candidate HRP-12975 are funded in part by the Rett Syndrome Research Trust (RSRT). HRP-12975 is the ...

In vitro data demonstrated the miRARE control element downregulates MECP2 transgene and protein expression in response to cellular levels of MeCP2 in cell culture models Data recapitulate in vivo ...

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 ...

Most autism spectrum disorders have a complex, multifactorial genetic component, making it difficult to find specific treatments. Rett syndrome is an exception. Babies born with this form of the ...

Rett syndrome is a rare devastating neurological disorder that primarily affects young girls and manifests as an impaired ability to walk and talk, along with characteristic ‘hand-wringing’ movements, ...

New preclinical data after neonatal administration in wild-type mice showed no detectable impact on survival, neurobehavioral functions and overall health, suggesting TSHA-102, engineered with novel ...

The neurodevelopmental disorder MECP2 duplication syndrome (MDS) is caused by duplications spanning the methyl-CpG binding protein 2 gene (MECP2) locus, and researchers have shown that the MDS-like ...