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Base Editing of Mice With Progeria Extends Lifespan

Researchers have used base editing to extend the lives of mice with a premature aging syndrome, highlighting the approach as a potential treatment for people with the condition.

As they report in Nature this week, researchers led by the Broad Institute's David Liu used an adenine base editor to correct the pathogenic mutation that occurs in Hutchinson-Gilford progeria syndrome. That syndrome is marked by a mutation in the gene that encodes nuclear lamin A that leads to mis-splicing and the production of the toxic progerin protein. 

Mice with progeria treated with the base editor lived longer than those not treated. Untreated mice with progeria had a median life span of 214 days, while treated mice had a median lifespan of 510 days, which, according to the researchers, is close to what is considered old age in the mice. In addition, the treated mice exhibited improved aortic pathology and vitality. 

"The extension of lifespan in the mice is a stark and compelling result that underscores the potential for these types of technologies to be transformative," Charles Gersbach, director of the Duke University Center for Advanced Genomic Technologies, who wasn't involved in the study, tells the Wall Street Journal.

At his blog, study author Francis Collins, the director of the US National Institutes of Health, writes that "[w]e are hopeful this gene editing work might eventually lead to a cure for progeria. But mice certainly aren't humans, and there are still important steps that need to be completed before such a gene-editing treatment could be tried safely in people."

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