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CRISPR-Cas9 technology is used to edit DNA to correct genetic abnormalities, but until now has required that tissue samples be removed for gene editing outside the body. In March 2020 researchers announced a test of the first 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 the related article link to read a review of the range of clinical trials testing CRISPR as a treatment for human disease in 2020.
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