Guilbert Solenn M, Cardoso Déborah, Lévy Nicolas, Muchir Antoine, Nissan Xavier
CECS, I-STEM AFM, Institute for Stem Cell Therapy and Exploration of Monogenic Diseases, 28 rue Henri Desbruères, 91100 Corbeil-Essonnes, France.
Sorbonne Université, UPMC Paris 06, INSERM UMRS974, Center of Research in Myology, Institut de Myologie, F-75013 Paris, France.
Methods. 2021 Jun;190:3-12. doi: 10.1016/j.ymeth.2020.04.005. Epub 2020 Apr 9.
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.
如果下一代成功的治疗方法就隐藏在现有的药典中会怎样？确定现有药物的新适应症，也称为药物重新利用或药物再发现过程，是一种高效且低成本的策略。药物重新利用在近一个世纪前首次被报道，现已成为治疗尚无特定疗法的疾病尤其是罕见病的一种有价值的治疗选择。本综述聚焦于哈钦森-吉尔福德早衰综合征（HGPS），这是一种罕见的遗传性疾病，会导致加速和过早衰老，在过去十年中，药物重新利用已促成了针对该疾病的几种潜在治疗方法的发现。
