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adeno-associated virus CRISPR Streptococcus pyogenes Cas9
- Development stage
- Preclinical
- Lead developer
- CRISPR Therapeutics
- Modality
- CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies
- Administration
- Intramuscular
01
Overview
Adeno-associated virus CRISPR Streptococcus pyogenes Cas9 refers to a gene-editing therapeutic platform that utilizes an adeno-associated viral (AAV) vector to deliver the CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) machinery, specifically the Cas9 nuclease derived from *Streptococcus pyogenes* (SpCas9). This system is engineered to induce site-specific double-strand breaks in genomic DNA, guided by a single-guide RNA (sgRNA), to facilitate gene disruption, correction, or transgene integration. Due to the large size of the SpCas9 gene, it is frequently delivered using a dual-vector system or a single vector with truncated regulatory elements. In preclinical research, such as studies for Duchenne Muscular Dystrophy (DMD), the platform is employed to target and edit mutations in the DMD gene to restore the production of functional dystrophin protein in muscle and cardiac tissues.
- Other names
- AAV-SpCas9AAV-SpCas-9AAV-SpCas 9AAV-CRISPR/Cas9AAV-delivered SpCas9
02
Targets
gDNA (Genomic DNA (CRISPR-Cas9 target site))
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