Farnesyltransferase inhibition in HGPS.
review · Level V
Where this comes from
- Record sourced from PubMed, PMID 33482093.
- Also identified by DOI 10.1016/j.cell.2020.12.029.
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Abstract
The ultra-rare, pediatric premature aging disorder Hutchinson-Gilford progeria syndrome (HGPS) is caused by mutation of LMNA, encoding the nuclear architectural protein lamin A. Patients develop atherosclerosis and typically die of heart failure in their teens. FDA-approved Zokinvy prevents farnesylation of lamin A, reduces vascular stiffness, and extends survival in HGPS patients. To view this Bench to Bedside, open or download the PDF.
Medical subject headings
- Enzyme Inhibitors
- Farnesyltranstransferase
- Progeria