AAV-Mecp2-CTRG gene therapy

AAV-Mecp2-CTRG is an experimental gene therapy developed by researchers at Ospedale San Raffaele and Reverta for the treatment of Rett syndrome. It utilizes a novel regulatory cassette (CTRG) designed to address the strict dosage sensitivity of the MECP2 protein. The platform combines the *Mecp2* coding sequence with two control elements: an shRNA that targets endogenous *Mecp2* to prevent toxic overexpression in transduced cells, and a miRNA-based detargeting strategy to achieve cell-type-specific expression across neuronal and glial populations. The therapy is being evaluated using advanced brain-penetrant AAV vectors, such as AAV9 variants targeting the human transferrin receptor (hTfR1), to ensure widespread delivery across the blood-brain barrier.

Other names

Cell-type regulated Mecp2 gene therapy