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Clin Epigenetics. 2023 Jan 11;15(1):6. doi: 10.1186/s13148-022-01419-z.
Modulating the epigenome has long been considered a potential opportunity for therapeutic intervention in numerous disease areas with several approved therapies marketed, primarily for cancer. Despite the overall promise of early approaches, however, these drugs have been plagued by poor pharmacokinetic and safety/tolerability profiles due in large part to off-target effects and a lack of specificity.
Recently, there has been marked progress in the field on a new generation of epigenomic therapies which address these challenges directly by targeting defined loci with highly precise, durable, and tunable approaches. Here, we review the promise and pitfalls of epigenetic drug development to date and provide an outlook on recent advances and their promise for future therapeutic applications.
Novel therapeutic modalities leveraging epigenetics and epigenomics with increased precision are well positioned to advance the field and treat patients across disease areas in the coming years.
长期以来，人们一直认为，对许多疾病领域的表观基因组进行调控可能是一种有潜力的治疗干预机会，目前已有几种获得批准的疗法上市，主要用于癌症治疗。然而，尽管早期方法有很大的希望，但这些药物由于脱靶效应和缺乏特异性，其药代动力学和安全性/耐受性特征一直存在问题。
最近，在新一代表观基因组疗法领域取得了显著进展，这些疗法通过使用高度精确、持久和可调的方法直接针对特定基因座，直接解决了这些挑战。在这里，我们回顾了迄今为止表观遗传学药物开发的前景和陷阱，并展望了最近的进展及其在未来治疗应用中的前景。
利用表观遗传学和表观基因组学提高精准度的新型治疗方式有望在未来几年推动该领域的发展，并为各个疾病领域的患者提供治疗。
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