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Base editing successfully treats progeria in mice

A team led by researchers from the Broad Institute of MIT and Harvard, the National Institutes of Health, and Vanderbilt University Medical Center has used base editing, a recently developed form of gene editing, to rescue disease symptoms and lifespan in mice that have Hutchinson-Gilford progeria syndrome.

Co-senior authors on the study include David Liu, director of the Merkin Institute of Transformative Technologies in Healthcare at the Broad Institute, professor at Harvard University, and investigator with the Howard Hughes Medical Institute, Francis Collins, director of the National Institutes of Health and senior investigator at the National Human Genome Research Institute, and Jonathan Brown, assistant professor of medicine in the Division of Cardiovascular Medicine at Vanderbilt University Medical Center.

Learn more at broad.io/progeria.

Paper cited:
Koblan LW, Erdos MR, et al. In vivo adenine base editing rescues Hutchinson-Gilford progeria syndrome. Nature. Online January 6, 2021. DOI: 10.1038/s41586-020-03086-7