AAV-MECP2

AAV-MECP2 is an investigational gene therapy designed for the treatment of Rett syndrome, a severe neurodevelopmental disorder caused by mutations in the methyl-CpG-binding protein 2 (MECP2) gene. The therapy utilizes a recombinant adeno-associated virus (AAV) vector to deliver a functional human MECP2 gene into neurons. It employs a neuron-specific promoter (CAG) to drive expression of the MECP2 protein, aiming to restore the epigenetic regulatory functions of MeCP2 in the central nervous system. Typically administered via a single intrathecal injection, the product is intended to improve symptoms such as cognitive regression, loss of motor skills, and respiratory abnormalities by compensating for the endogenous protein deficiency.