4.8 Article

Targeting Protein Prenylation in Progeria

Journal

SCIENCE TRANSLATIONAL MEDICINE
Volume 5, Issue 171, Pages -

Publisher

AMER ASSOC ADVANCEMENT SCIENCE
DOI: 10.1126/scitranslmed.3005229

Keywords

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Funding

  1. U.S. National Institutes of Health [AG035626, HL086683, HL089781]

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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.

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