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AAV-shRNA targeting Msh3

Development stage: Preclinical
Lead developer: Brigham and Women's Hospital
Modality: Viral-delivered RNAi → In Vivo RNAi → Gene Silencing → Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics
Administration: Intravenous

Overview

AAV-shRNA targeting Msh3 is an experimental gene therapy designed to treat Huntington's Disease (HD) by inhibiting somatic repeat expansion. HD is caused by a CAG trinucleotide repeat expansion in the HTT gene, and somatic expansion of these repeats is driven by the DNA mismatch repair pathway, specifically the MSH3 protein. This therapy utilizes adeno-associated virus (AAV) vectors—including scAAV9 and AAV-CPP16 capsids—to deliver short hairpin RNA (shRNA) that knocks down Msh3 expression in the striatum. To overcome the blood-brain barrier (BBB), the therapy is being investigated in combination with focused ultrasound (FUS) and microbubbles for non-invasive delivery.

Other names: AAV-shRNA-Msh3AAV-shRNA-Msh-3AAV-shRNA-Msh 3scAAV9-shRNA-Msh3scAAV-9-shRNA-Msh3scAAV 9-shRNA-Msh3AAV-CPP16-shRNA-Msh3AAV-CPP-16-shRNA-Msh3AAV-CPP 16-shRNA-Msh3

Targets

MSH3 (DNA mismatch repair protein Msh3)

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