Base editor treats progeria in mice.
Nature(2021)
Abstract
Study design: Preclinical study Population: Fibroblasts derived from people with Hutchinson-Gilford progeria syndrome and mouse models of the genetic disease Key result: Single injection of an adenine base editor (ABE) using a viral vector corrects up to 90% of the mutations in fibroblasts and ameliorates vascular disease and extends lifespan in mice Next steps: ABE could be a feasible therapeutic strategy for progeria in humans
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Key words
CRISPR-Cas9 genome editing,Diseases,Genetics
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