학술논문

Hutchinson-Gilford progeria syndrome: Rejuvenating old drugs to fight accelerated ageing.
Document Type
Article
Source
Methods. Jun2021, Vol. 190, p3-12. 10p.
Subject
*PROGERIA
*PRECOCIOUS puberty
*THERAPEUTICS
*GENETIC disorders
*DRUGS
Language
ISSN
1046-2023
Abstract
• Hutchinson Gilford progeria syndrome (HGPS) is a rare disease characterized by an accelerated aging. • Drug repurposing allows to speed up the therapeutic development by defining new indications of existing drugs. • With the discovery of over 35 potential therapeutical options in less than a decade, HGPS is a unique example to discuss benefits and drawbacks of drug repurposing strategies. 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. [ABSTRACT FROM AUTHOR]