期刊
METHODS
卷 190, 期 -, 页码 3-12出版社
ACADEMIC PRESS INC ELSEVIER SCIENCE
DOI: 10.1016/j.ymeth.2020.04.005
关键词
Progeria; Aging; Pluripotent stem cells; Drug repurposing; High-throughput screening
资金
- Association Francaise contre les Myopathies (AFM-Telethon)
- INSERM
- domaine d'interet majeur (DIM) Biotherapies, Genopole
- European Commission: the laboratoire d'Excellence Revive (Investissement d'Avenir) [ANR-10-LABX-73]
- Neu-rATRIS: A Translational Research Infrastructure for Biotherapies in Neurosciences (Investissement d'Avenir) [ANR-11-INBS-0011]
- INGES-TEM: the National Infrastructure Engineering for Pluripotent and differentiated Stem cells (Investissement d'Avenir) [ANR-11-INBS-0009]
- University of Paris-Saclay (BiotherAlliance)
- French National Agency [ANR-18-CE14-0036]
- Agence Nationale de la Recherche (ANR) [ANR-11-INBS-0009] Funding Source: Agence Nationale de la Recherche (ANR)
Drug repurposing is an efficient and low-cost strategy, which has made significant progress in treating rare diseases in recent years.
What if the next generation of successful treatments was hidden in the current pharmacopoeia? Identifying new indications for existing drugs, also called the drug repurposing or drug rediscovery process, is a highly efficient and low-cost strategy. First reported almost a century ago, drug repurposing has emerged as a valuable therapeutic option for diseases that do not have specific treatments and rare diseases, in particular. This review focuses on Hutchinson-Gilford progeria syndrome (HGPS), a rare genetic disorder that induces accelerated and precocious aging, for which drug repurposing has led to the discovery of several potential treatments over the past decade.
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