CRISPR-Cas Systems Delivered via AAV Vectors: Unlocking Precision Gene Editing
CRISPR-Cas systems have revolutionized the field of gene editing, offering a precise, efficient, and versatile method for modifying DNA. However, delivering these powerful tools into living organisms poses challenges, particularly due to size constraints associated with viral vectors like Adeno-Associated Virus (AAV) . Fortunately, the development of miniature CRISPR systems has enabled effective delivery through AAV vectors, broadening the horizons of gene therapy and biomedical research. Miniature CRISPR Systems for AAV Delivery The standard CRISPR-Cas9 system, derived from Streptococcus pyogenes (SpCas9), is too large to fit into AAV vectors, which have a packaging capacity of about 4.7 kilobases (kb) . To overcome this, researchers have identified smaller Cas proteins that retain the DNA-cutting ability while fitting within AAV’s limits. 1. SaCas9 ( Staphylococcus aureus Cas9) Size Advantage: About 1 kb smaller than SpCas9. Functionality: SaCas9 offers similar gene-editing...