The Promise of Epigenetic Editing for Treating Brain Disorders.

Q1 Biochemistry, Genetics and Molecular Biology Sub-cellular biochemistry Pub Date : 2025-01-01 DOI:10.1007/978-3-031-75980-2_4
Luis A González Molina, Amalia M Dolga, Marianne G Rots, Federica Sarno
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Abstract

Brain disorders, especially neurodegenerative diseases, affect millions of people worldwide. There is no causal treatment available; therefore, there is an unmet clinical need for finding therapeutic options for these diseases. Epigenetic research has resulted in identification of various genomic loci with differential disease-specific epigenetic modifications, mainly DNA methylation. These biomarkers, although not yet translated into clinically approved options, offer therapeutic targets as epigenetic modifications are reversible. Indeed, clinical trials are designed to inhibit epigenetic writers, erasers, or readers using epigenetic drugs to interfere with epigenetic dysregulation in brain disorders. However, since such drugs elicit genome-wide effects and potentially cause toxicity, the recent developments in the field of epigenetic editing are gaining widespread attention. In this review, we provide examples of epigenetic biomarkers and epi-drugs, while describing efforts in the field of epigenetic editing, to eventually make a difference for the currently incurable brain disorders.

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表观遗传编辑治疗脑部疾病的前景。
脑部疾病,特别是神经退行性疾病,影响着全世界数百万人。目前尚无因果治疗方法;因此,寻找这些疾病的治疗方案的临床需求尚未得到满足。表观遗传学研究已经确定了各种具有不同疾病特异性表观遗传修饰的基因组位点,主要是DNA甲基化。这些生物标志物虽然尚未转化为临床批准的选择,但由于表观遗传修饰是可逆的,因此提供了治疗靶点。事实上,临床试验旨在抑制表观遗传书写者、擦除者或读取者,使用表观遗传药物来干扰大脑疾病中的表观遗传失调。然而,由于这些药物引起全基因组效应并可能引起毒性,表观遗传编辑领域的最新进展正在引起广泛关注。在这篇综述中,我们提供了表观遗传生物标志物和表观遗传药物的例子,同时描述了表观遗传编辑领域的努力,最终对目前无法治愈的脑部疾病产生影响。
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来源期刊
Sub-cellular biochemistry
Sub-cellular biochemistry Biochemistry, Genetics and Molecular Biology-Biochemistry
CiteScore
5.90
自引率
0.00%
发文量
33
期刊介绍: The book series SUBCELLULAR BIOCHEMISTRY is a renowned and well recognized forum for disseminating advances of emerging topics in Cell Biology and related subjects. All volumes are edited by established scientists and the individual chapters are written by experts on the relevant topic. The individual chapters of each volume are fully citable and indexed in Medline/Pubmed to ensure maximum visibility of the work.
期刊最新文献
Basic Epigenetic Mechanisms. Environmental Enrichment and Epigenetic Changes in the Brain: From the Outside to the Deep Inside. Epigenetic Control in Schizophrenia. Epigenetics in Learning and Memory. Epigenetics in Neurodegenerative Diseases.
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