Progeria: a paradigm for translational medicine.

Gordon, Leslie B; Rothman, Frank G; López-Otín, Carlos; Misteli, Tom · Cell · 2014

review · Level V

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Abstract

Rare diseases are powerful windows into biological processes and can serve as models for the development of therapeutic strategies. The progress made on the premature aging disorder Progeria is a shining example of the impact that studies of rare diseases can have.

Medical subject headings