Tag: Crispr cas9

In the widely used *Streptococcus pyogenes* CRISPR-Cas9 system, the Cas9 enzyme relies on a specific protospacer adjacent motif (PAM) sequence, typically 5′-NGG-3′, to correctly identify and modify target DNA. This short DNA sequence is essential for the CRISPR machinery to differentiate between the host's own genetic material and foreign, invading DNA, thereby enabling precise gene editing while preventing a self-inflicted attack.

In December 2023, the U.S. Food and Drug Administration (FDA) approved the world's first CRISPR-based therapy, Casgevy, marking a historic shift in treating genetic blood disorders. This groundbreaking approach directly modifies a patient's own cells, which is central to understanding what is the active ingredient in Casgevy.

In a landmark decision in late 2023, the U.S. FDA approved Casgevy, the first treatment utilizing CRISPR technology to receive authorization. Understanding **how does casgevy gene therapy work** involves a look at its process of gene editing to enable a patient's body to produce healthy blood cells.