New form of gene editing efficient for treatment of AATD, liver and lung disease
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14 July 2021New form of gene editing efficient for treatment of AATD, liver and lung disease
New research results show that a new form of gene editing is efficient in correcting a mutation in patient cells with the monogenic disease Alpha-1 antitrypsin deficiency (AATD), a common inherited disease that affects the liver and the lungs. This new approach, called base editing, is different from other forms of editing including CRISPR, because the base editors do not induce a break in the DNA, which helps prevent double strand breaks, potential off-target editing, and unwanted mutations during cell repair.
