Study Will Test CRISPR Gene Editing Inside the Body for the First Time

Notes from NCER:

In hopes of receiving sight, adults and children with an inherited form of blindness will have their genes edited in the first ever treatment that will alter patients’ native DNA using CRISPR. The specific condition, Leber congenital amaurosis, is rare (3 out of 100,000), and occurs when a child inherits two flawed genes, one from each parent. This is a stellar example of the ethical use of gene editing in humans in …