CRISPR 2.0: Base Editing in the Groove A series of exciting preclinical and animal model results show that base editing is making rapid strides toward the clinic March 4, 2021 Researchers from the Broad Institute, the NIH, and Vanderbilt University have used an adenine base editor (ABE) to treat progeria in mice. Correcting the mutation that causes progeria led to strong symptom reduction and longer lifespan. An ABE is shown here bound to a guide RNA and a targeted piece of DNA. [Aditya Raguram, Liu Laboratory, Broad Institute] Share A reasonable idea, suggests Sekar Kathiresan, MD, co-founder and CEO of Verve Therapeutics, is the development of a one-time treatment—a single spelling change in the DNA of a liver gene in an adult person—that would “turn off the gene and lower blood cholesterol levels for the rest of the person’s life.” Although this idea would not have been taken very seriously 10 years ago, it is now being investigated. It is a potent illustration of the magnificent promise of genome editing in general, and of base editing in particular.