AAV6-MBNL1-RNAi

**AAV6-MBNL1-RNAi** is an investigational, muscle-targeted adeno-associated virus serotype 6 gene-therapy approach for **myotonic dystrophy type 1**. The vector is designed to co-express muscleblind-like protein 1 to restore deficient MBNL1 activity and an RNA-interference cassette intended to reduce toxic expanded dystrophia myotonica protein kinase messenger RNA. In the HSALR mouse model, co-delivery of MBNL1 and RNAi produced an additive improvement in aberrant Atp2a1 splicing, a molecular disease readout. The program remains preclinical and is being developed to restrict transgene expression to skeletal muscle and reduce potential cardiac toxicity.