With First CRISPR Trials, Gene Editing Moves Toward the Clinic

May 4, 2020

(JAMA) – In March, a patient with an inherited form of blindness called Leber congenital amaurosis became the first person to undergo gene editing inside the body using clustered regularly interspaced short palindromic repeats (CRISPR) and associated protein 9 (Cas9). The procedure, part of a phase 1 and 2 trial that’s expected to enroll about 18 patients, comes less than a decade after scientists first demonstrated how CRISPR-Cas9, a bacterial defense system, could be programmed to edit the human genome. Compared with older gene-editing technologies, the technique can be readily and cheaply designed to target almost any genome sequence, and at multiple sites at the same time, with exceptional efficiency. This has opened up the potential to safely and affordably correct an array of disease-causing genetic alterations.