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AAV-MBNL1-RNAi
- Development stage
- Preclinical
- Lead developer
- University of Washington
- Modality
- Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intramuscular, Intravenous
01
Overview
AAV-MBNL1-RNAi is a dual-action adeno-associated virus (AAV)-mediated gene therapy designed for the treatment of myotonic dystrophy type 1 (DM1). Developed by researchers at the University of Washington, the vector combines the expression of the Muscleblind-like protein 1 (MBNL1) splicing factor with an RNA interference (RNAi) sequence targeting the dystrophia myotonica protein kinase (DMPK) mRNA. In DM1, expanded CUG repeats in the DMPK transcript sequester MBNL1, leading to widespread splicing defects (spliceopathy). By simultaneously knocking down the toxic DMPK mRNA via RNAi and supplementing functional MBNL1 protein, AAV-MBNL1-RNAi aims to restore normal splicing and ameliorate the disease phenotype at lower viral doses, potentially reducing the risk of cardiotoxicity associated with high-dose AAV or MBNL1 overexpression alone.
- Other names
- AAV-MBNL1-RNAi vectorAAV-MBNL-1-RNAi vectorAAV-MBNL 1-RNAi vectordual AAV-mediated RNAi and protein expression therapy
02
Targets
AGO2 (Argonaute RISC catalytic component 2)CELF1 (CUGBP Elav-like family member 1)Pathogenic DMPK RNA repeat expansionMBNL1 (Muscleblind-like splicing regulator 1)MBNL2 (Muscleblind-like protein 2)
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