PerspectiveDrug Development

Targeting Protein Prenylation in Progeria

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Science Translational Medicine  06 Feb 2013:
Vol. 5, Issue 171, pp. 171ps3
DOI: 10.1126/scitranslmed.3005229

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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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