CRISPR gene editing in humans has moved from laboratory promise to bedside reality. The technology now treats blood disorders and HIV in active clinical trials, with regulatory frameworks in place across North America to govern how these therapies reach patients. Unlike earlier gene therapies that added new DNA, CRISPR-based treatments precisely cut and repair faulty genetic sequences at their source, using a guide RNA to direct the Cas9 enzyme to target mutations responsible for disease.
The clinical landscape has matured considerably. Excision Biotherapeutics completed phase I/II trials using CRISPR molecules to excise integrated …