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myoAAV-gRNA (Mstn, Acvr2a, Acvr2b)
- Development stage
- Preclinical
- Lead developer
- University of Arkansas
- Modality
- Gene Silencing → Gene Therapies, Gene Addition/Replacement → Gene Therapies, CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies
- Administration
- Intramuscular, Intravenous
01
Overview
myoAAV-gRNA (Mstn, Acvr2a, Acvr2b) is an experimental gene therapy candidate developed by researchers at the University of Arkansas for the treatment of muscle wasting diseases, including Duchenne Muscular Dystrophy (DMD). The therapy utilizes a muscle-tropic adeno-associated virus (myoAAV) to deliver guide RNAs (gRNAs) that target the myostatin signaling pathway. This system works in conjunction with a skeletal muscle-specific dCas9-KRAB transcriptional repressor to achieve multiplexed downregulation of three key genes: Mstn (myostatin), Acvr2a (activin receptor type-2A), and Acvr2b (activin receptor type-2B). By inhibiting these negative regulators of muscle growth, the therapy aims to increase muscle fiber size and restore muscle function. Preclinical studies have demonstrated significant downregulation of Mstn and Acvr2a following both intramuscular and systemic administration.
- Other names
- myoAAV-gRNA (Mstn/Acvr2a/Acvr2b)
02
Targets
MSTN (Myostatin)ACVR2A (Activin receptor type 2A)
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