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Sci Transl Med. 2013 Feb 6;5(171):171ps3. doi: 10.1126/scitranslmed.3005229.

Targeting protein prenylation in progeria.

Author information

1
Department of Medicine, David Geffen School of Medicine, University of California, Los Angeles, CA 90095, USA. sgyoung@mednet

Abstract

A clinical trial of a protein farnesyltransferase inhibitor (lonafarnib) for the treatment of Hutchinson-Gilford progeria syndrome (HGPS) was recently completed. Here, we discuss the mutation that causes HGPS, the rationale for inhibiting protein farnesyltransferase, the potential limitations of this therapeutic approach, and new potential strategies for treating the disease.

PMID:
23390246
PMCID:
PMC3725554
DOI:
10.1126/scitranslmed.3005229
[Indexed for MEDLINE]
Free PMC Article
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