In DepthBiomedicine

Gene therapy beats premature-aging syndrome in mice

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Science  08 Jan 2021:
Vol. 371, Issue 6525, pp. 114
DOI: 10.1126/science.371.6525.114

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Summary

A new study is inspiring hope for treating children born with progeria, a rare, fatal genetic disease that causes symptoms much like early aging. In mice with a progeria-causing mutation, a cousin of the celebrated genome editor known as CRISPR corrected the DNA mistake, preventing the heart damage typical of the disease, a research team reports this week in Nature. Treated mice lived about 500 days, more than twice as long as untreated animals. Although developers of the progeria therapy aim to improve it, they are also taking steps toward testing the current version in affected kids and some other scientists endorse a rush.

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