CRISPR-CAS9 GENE EDITING THERAPY: FROM BENCH TO CLINICAL APPROVAL

Abstract

CRISPR-Cas9 has transformed the landscape of genetic medicine by enabling precise, programmable editing of the human genome. The system relies on a guide RNA that directs the Cas9 endonuclease to a specific DNA sequence, where it introduces a double-strand break that the cell repairs either by non-homologous end joining, which typically disrupts gene function, or by homology-directed repair, which allows the insertion of a corrected sequence. This dual capability makes the platform equally suited to gene knockout and gene correction strategies, and it has rapidly moved from a laboratory tool into a clinically validated therapeutic modality.