AAVHSC15 Mecp2 gene editing vector

AAVHSC15 Mecp2 gene editing vector is an experimental in vivo genome editing therapeutic designed for the treatment of Rett syndrome, a severe neurodevelopmental disorder caused by mutations in the X-linked MECP2 gene. Developed by researchers at the City of Hope, the vector utilizes the AAVHSC15 serotype, which is derived from human hematopoietic stem cells and is capable of crossing the blood-brain barrier to transduce both the central nervous system and peripheral tissues. The therapy employs a nuclease-free, transcription-coupled, BRCA2-dependent homologous recombination strategy to precisely correct mutations within the endogenous Mecp2 locus. By targeting the native genomic sequence, the approach preserves all endogenous regulatory elements, ensuring physiological expression levels and avoiding the toxicity associated with MeCP2 overexpression. Preclinical studies in the Mecp2-/Y mouse model have demonstrated that systemic administration of the vector significantly extends lifespan and improves Rett-associated symptoms, including motor function, tremors, and respiratory deficiencies.

Other names

AAVHSC15 Mecp2 editing vectorAAVHSC-15 Mecp2 editing vectorAAVHSC 15 Mecp2 editing vector