Progeria: Translational insights from cell biology

Author:

Gordon Leslie B.123,Cao Kan4,Collins Francis S.5

Affiliation:

1. The Progeria Research Foundation, Peabody, MA 01961

2. Department of Pediatrics, Alpert Medical School of Brown University and Hasbro Children’s Hospital, Providence, RI 02903

3. Department of Anesthesia, Boston Children’s Hospital and Harvard Medical School, Boston, MA 02115

4. Department of Cell Biology and Molecular Genetics, University of Maryland, College Park, MD 20742

5. National Institutes of Health, Bethesda, MD 20892

Abstract

Cell biologists love to think outside the box, pursuing many surprising twists and unexpected turns in their quest to unravel the mysteries of how cells work. But can cell biologists think outside the bench? We are certain that they can, and clearly some already do. To encourage more cell biologists to venture into the realm of translational research on a regular basis, we would like to share a handful of the many lessons that we have learned in our effort to develop experimental treatments for Hutchinson-Gilford progeria syndrome (HGPS), an endeavor that many view as a “poster child” for how basic cell biology can be translated to the clinic.

Publisher

Rockefeller University Press

Subject

Cell Biology

Reference34 articles.

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2. Differential temporal and spatial progerin expression during closure of the ductus arteriosus in neonates;Bökenkamp;PLoS ONE.,2011

3. A lamin A protein isoform overexpressed in Hutchinson-Gilford progeria syndrome interferes with mitosis in progeria and normal cells;Cao;Proc. Natl. Acad. Sci. USA.,2007

4. Rapamycin reverses cellular phenotypes and enhances mutant protein clearance in Hutchinson-Gilford progeria syndrome cells;Cao;Sci. Transl. Med.,2011

5. Progerin and telomere dysfunction collaborate to trigger cellular senescence in normal human fibroblasts;Cao;J. Clin. Invest.,2011

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