Gene Editing Inside the Body Using CRISPR

CRISPR-Cas9 technology is used to edit DNA to correct genetic abnormalities, but until now has required cells be removed for gene editing outside the body. In March 2020 researchers announced a test of the first in-the-body (in vivo) CRISPR-Cas9 treatment for Leber congenital amaurosis (LCA) type 10, a condition that causes severe, early-onset vision loss. This video explains the principles of DNA editing with #CRISPR-Cas9 and the rationale for its use and testing as a therapy for LCA10 in the BRILLIANCE clinical trial. Click https://ja.ma/35adPDf to read a #JAMAMedNews review of the range of clinical trials testing CRISPR as a treatment for genetic blindness and other human diseases in 2020.