Cas9 protein

Cas9 protein is a specialized RNA-guided DNA endonuclease derived from the CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) bacterial immune system. It serves as the catalytic component of the CRISPR/Cas9 genome editing platform, where it is complexed with a guide RNA (gRNA) to identify and cleave specific double-stranded DNA sequences. In the clinical trial context (ChiCTR1800018955) associated with the South University of Science and Technology of China, the Cas9 protein was utilized to attempt targeted gene disruption in human embryos, specifically targeting the CCR5 gene to confer HIV resistance and the PCSK9 gene for cholesterol management. The protein induces double-strand breaks that are subsequently repaired by cellular mechanisms, leading to gene knockout or precise genomic alterations.

Other names

CRISPR-associated protein 9Cas9Cas-9Cas 9