Cell Chemical Biology ( IF 6.6 ) Pub Date : 2024-08-12 , DOI: 10.1016/j.chembiol.2024.07.007 Goldie V Roth 1 , Isabella R Gengaro 2 , Lei S Qi 3
The epigenome is a complex framework through which gene expression is precisely and flexibly modulated to incorporate heritable memory and responses to environmental stimuli. It governs diverse cellular processes, including cell fate, disease, and aging. The need to understand this system and precisely control gene expression outputs for therapeutic purposes has precipitated the development of a diverse set of epigenetic editing tools. Here, we review the existing toolbox for targeted epigenetic editing, technical considerations of the current technologies, and opportunities for future development. We describe applications of therapeutic epigenetic editing and their potential for treating disease, with a discussion of ongoing delivery challenges that impede certain clinical interventions, particularly in the brain. With simultaneous advancements in available engineering tools and appropriate delivery technologies, we predict that epigenetic editing will increasingly cement itself as a powerful approach for safely treating a wide range of disorders in all tissues of the body.
中文翻译:
精确表观遗传编辑:技术进步、持久挑战和治疗应用
表观基因组是一个复杂的框架,通过该框架,基因表达被精确和灵活地调节,以纳入可遗传的记忆和对环境刺激的反应。它控制着不同的细胞过程,包括细胞命运、疾病和衰老。了解该系统并精确控制基因表达输出以用于治疗目的的需求促使开发了一套多样化的表观遗传编辑工具。在这里,我们回顾了现有的靶向表观遗传编辑工具箱、当前技术的技术考虑以及未来发展的机会。我们描述了治疗性表观遗传编辑的应用及其治疗疾病的潜力,并讨论了阻碍某些临床干预(尤其是在大脑中)的持续递送挑战。随着可用工程工具和适当递送技术的同步进步,我们预测表观遗传编辑将日益巩固自己作为安全治疗身体所有组织中各种疾病的有力方法。