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AAV9-miniCRISPRa
- Development stage
- Preclinical
- Lead developer
- University of Pittsburgh
- Modality
- CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies
- Administration
- Intramuscular, Parenteral
01
Overview
AAV9-miniCRISPRa (also known as mini-CRISPRa 2.0) is an investigational gene therapy candidate developed by the University of Pittsburgh for the treatment of LAMA2-related muscular dystrophy (also known as merosin-deficient congenital muscular dystrophy type 1A, or MDC1A). The therapy utilizes a single adeno-associated virus serotype 9 (AAV9) vector to deliver a miniaturized CRISPR activation (CRISPRa) system. This system consists of a Staphylococcus aureus dead Cas9 (SadCas9) driven by a short synthetic 4xNRF1 promoter, fused to a miniaturized tripartite transcriptional activator (miniVPR: VP64, P65, and RTA), and a single guide RNA (sgRNA) targeting the promoter of the LAMA1 gene. By upregulating the expression of the compensatory LAMA1 gene (which encodes laminin-α1), the therapy aims to functionally compensate for the deficiency of laminin-α2 (encoded by the mutated LAMA2 gene) in skeletal muscle and Schwann cells, thereby improving muscle pathology and neuromuscular function.
- Other names
- AAV9-mini-CRISPRaAAV-9-mini-CRISPRaAAV 9-mini-CRISPRamini-CRISPRamini-CRISPRa 2.0mini-CRISPRa2.0mini-CRISPRa-2.0
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