Yi Chen, Guiquan Wang, Jingqiao Chen, Congying Wang, Xi Dong, Hsun-Ming Chang, Shuai Yuan, Yue Zhao, Liangshan Mu
Endocrine Reviews, First published online January 31, 2024, bnae002
https://doi.org/10.1210/endrev/bnae002
The treatment of polycystic ovary syndrome (PCOS) faces challenges as all known treatments are merely symptomatic. The US Food and Drug Administration has not approved any drug specifically for treating PCOS. As the significance of genetics and epigenetics rises in drug development, their pivotal insights have greatly enhanced the efficacy and success of drug target discovery and validation, offering promise for guiding the advancement of PCOS treatments. In this context, we outline the genetic and epigenetic advancement in PCOS, which provides novel insights into the pathogenesis of this complex disease. We also delve into the prospective method for harnessing genetic and epigenetic strategies to identify potential drug targets and ensure target safety. Additionally, we shed light on the preliminary evidence and distinctive challenges associated with gene and epigenetic therapies in the context of PCOS.
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