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AAV-Mecp2-CTRG gene therapy
- Development stage
- Preclinical
- Lead developer
- IRCCS Ospedale San Raffaele
- Modality
- Viral-delivered RNAi → In Vivo RNAi → Gene Silencing → Gene Therapies, AAV Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies
- Administration
- Intravenous
01
Overview
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
02
Targets
MECP2 (Methyl-CpG-binding protein 2)TLR9 (Toll-like receptor 9)
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