CRISPR 2.0: Base Editing in the Groove

Base editing has made rapid strides through preclinical studies and is poised to revolutionize medicine. This precise technology holds the key for a new class of genetic medicines developed through direct correction of disease-causing mutations and other genetic manipulations—inserting protective genetic variants, activating or silencing regulatory elements, knocking out genes through stop codon or splice site mutations, and potentially realizing a multiplexed approach.