Article

Isoprenylcysteine carboxylmethyltransferase-based therapy for Hutchinson–Gilford progeria syndrome

2020-07-24

Abstract excerpt

<h4>ABSTRACT</h4> Progerin is a mutant prelamin A variant that causes Hutchinson–Gilford progeria syndrome (HGPS, progeria), a rare genetic disease characterized by premature aging and death in childhood. Although several therapeutic approaches have been explored in experimental models, clinical trials have shown very limited benefits in HGPS patients. Here, we describe the development of UCM-13207, a new potent...

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Literature Corpus work: 7ae65d25-4587-57ce-bd5b-1f7c19aaf802
DOI: 10.1101/2020.07.23.217257

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