Article

Mutation-Agnostic Base Editing of the Progerin Farnesylation Site Rescues Hutchinson-Gilford Progeria Syndrome Phenotypes in Neuromuscular Organoids

2026-02-25

Abstract excerpt

<title>Abstract</title> <p>Hutchinson Gilford progeria syndrome (HGPS) is a fatal premature aging disorder caused by pathogenic farnesylated lamin A variants that disrupt nuclear architecture and DNA repair. Current therapies, including farnesyltransferase inhibitors, provide only modest survival benefits and lack molecular specificity, while mutation-specific genome-editing strategies cannot address atypical lam...

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Literature Corpus work: 708f260b-1512-5c94-abe2-a12f2ccdda57
DOI: 10.21203/rs.3.rs-7431680/v1

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