人类医学与兽医学中的表观遗传学和基因治疗。

Epigenetic and gene therapy in human and veterinary medicine.

作者信息

Bártová Eva

机构信息

Department of Cell Biology and Epigenetics, Institute of Biophysics, Academy of Sciences of the Czech Republic, Brno, 612 00, the Czech Republic.

出版信息

Environ Epigenet. 2024 May 10;10(1):dvae006. doi: 10.1093/eep/dvae006. eCollection 2024.

Abstract

Gene therapy is a focus of interest in both human and veterinary medicine, especially in recent years due to the potential applications of CRISPR/Cas9 technology. Another relatively new approach is that of epigenetic therapy, which involves an intervention based on epigenetic marks, including DNA methylation, histone post-translational modifications, and post-transcription modifications of distinct RNAs. The epigenome results from enzymatic reactions, which regulate gene expression without altering DNA sequences. In contrast to conventional CRISP/Cas9 techniques, the recently established methodology of epigenetic editing mediated by the CRISPR/dCas9 system is designed to target specific genes without causing DNA breaks. Both natural epigenetic processes and epigenetic editing regulate gene expression and thereby contribute to maintaining the balance between physiological functions and pathophysiological states. From this perspective, knowledge of specific epigenetic marks has immense potential in both human and veterinary medicine. For instance, the use of epigenetic drugs (chemical compounds with therapeutic potential affecting the epigenome) seems to be promising for the treatment of cancer, metabolic, and infectious diseases. Also, there is evidence that an epigenetic diet (nutrition-like factors affecting epigenome) should be considered as part of a healthy lifestyle and could contribute to the prevention of pathophysiological processes. In summary, epigenetic-based approaches in human and veterinary medicine have increasing significance in targeting aberrant gene expression associated with various diseases. In this case, CRISPR/dCas9, epigenetic targeting, and some epigenetic nutrition factors could contribute to reversing an abnormal epigenetic landscape to a healthy physiological state.

摘要

基因治疗是人类医学和兽医学共同关注的焦点,尤其是近年来,由于CRISPR/Cas9技术的潜在应用。另一种相对较新的方法是表观遗传治疗,它涉及基于表观遗传标记的干预,包括DNA甲基化、组蛋白翻译后修饰以及不同RNA的转录后修饰。表观基因组是由酶促反应产生的,这些反应在不改变DNA序列的情况下调节基因表达。与传统的CRISP/Cas9技术不同,最近建立的由CRISPR/dCas9系统介导的表观遗传编辑方法旨在靶向特定基因而不导致DNA断裂。天然的表观遗传过程和表观遗传编辑都调节基因表达,从而有助于维持生理功能和病理生理状态之间的平衡。从这个角度来看,特定表观遗传标记的知识在人类医学和兽医学中都具有巨大的潜力。例如,使用表观遗传药物(具有影响表观基因组治疗潜力的化合物)似乎有望用于治疗癌症、代谢性疾病和传染病。此外,有证据表明,表观遗传饮食(影响表观基因组的类似营养的因素)应被视为健康生活方式的一部分,并有助于预防病理生理过程。总之,基于表观遗传学的方法在人类医学和兽医学中对于靶向与各种疾病相关的异常基因表达具有越来越重要的意义。在这种情况下,CRISPR/dCas9、表观遗传靶向和一些表观遗传营养因素可能有助于将异常的表观遗传格局逆转到健康的生理状态。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/450e/11095531/6018608c9058/dvae006f1.jpg

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