Open AccessInduced Pluripotent Stem Cells Reveal Functional Differences Between Drugs Currently Investigated in Patients With Hutchinson‐Gilford Progeria Syndrome
Author(s) -
Blondel Sophie,
Jaskowiak Anne-Laure,
Egesipe Anne-Laure,
Le Corf Amelie,
Navarro Claire,
Cordette Véronique,
Martinat Cécile,
Laabi Yacine,
Djabali Karima,
de Sandre-Giovannoli Annachiara,
Levy Nicolas,
Peschanski Marc,
Nissan Xavier
Publication year - 2014
Publication title -
stem cells translational medicine
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.781
H-Index - 71
eISSN - 2157-6580
pISSN - 2157-6564
DOI - 10.5966/sctm.2013-0168
Subject(s) - progeria , induced pluripotent stem cell , premature aging , cancer research , stem cell , farnesyltransferase , biology , pravastatin , idebenone , fasudil , pharmacology , prenylation , microbiology and biotechnology , genetics , endocrinology , biochemistry , rho associated protein kinase , embryonic stem cell , kinase , cholesterol , gene , enzyme
Hutchinson‐Gilford progeria syndrome is a rare congenital disease characterized by premature aging in children. The results of this systematic comparative study of the three main treatments currently administered or proposed to progeria‐affected children reveal the complexity of the modes of action of different drugs and underscore the use of induced pluripotent stem cell derivatives as a critical and powerful tool for standardized, comparative pharmacological studies.