Toward the Development of Epigenome Editing-Based Therapeutics: Potentials and Challenges.
Toward the Development of Epigenome Editing-Based Therapeutics: Potentials and Challenges.
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DOI：
10.3390/ijms24054778
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发表时间：
2023-03-01
影响因子：
5.6
通讯作者：
中科院分区：
文献类型：
--
作者：
过去几十年来表观遗传学研究的进步导致了表观基因组编辑技术在治疗各种疾病方面的潜在应用。特别是，表观基因组编辑在治疗遗传和其他相关疾病（包括罕见的印记疾病）方面可能有用，因为它可以调节目标区域表观基因组的表达，从而调节致病基因的表达，而对基因组 DNA 的修饰很少或没有修饰。人们正在进行各种努力，以成功地在体内应用表观基因组编辑，例如提高靶点特异性、酶活性和药物递送，以开发可靠的治疗方法。在这篇综述中，我们介绍了最新的发现，总结了表观基因组编辑在疾病治疗实际应用中当前的局限性和未来的挑战，并介绍了需要考虑的重要因素，例如染色质可塑性，以实现更有效的基于表观基因组编辑的治疗。
The advancement in epigenetics research over the past several decades has led to the potential application of epigenome-editing technologies for the treatment of various diseases. In particular, epigenome editing is potentially useful in the treatment of genetic and other related diseases, including rare imprinted diseases, as it can regulate the expression of the epigenome of the target region, and thereby the causative gene, with minimal or no modification of the genomic DNA. Various efforts are underway to successfully apply epigenome editing in vivo, such as improving target specificity, enzymatic activity, and drug delivery for the development of reliable therapeutics. In this review, we introduce the latest findings, summarize the current limitations and future challenges in the practical application of epigenome editing for disease therapy, and introduce important factors to consider, such as chromatin plasticity, for a more effective epigenome editing-based therapy.
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影响因子：
48
作者：
Chavez A;Tuttle M;Pruitt BW;Ewen-Campen B;Chari R;Ter-Ovanesyan D;Haque SJ;Cecchi RJ;Kowal EJK;Buchthal J;Housden BE;Perrimon N;Collins JJ;Church G
通讯作者：
Church G
DOI：
10.1016/j.omtm.2020.09.001
发表时间：
2020-12-11
期刊：
Molecular therapy. Methods & clinical development
影响因子：
--
作者：
Backstrom JR;Sheng J;Wang MC;Bernardo-Colón A;Rex TS
通讯作者：
Rex TS
影响因子：
16.6
作者：
Braun SMG;Kirkland JG;Chory EJ;Husmann D;Calarco JP;Crabtree GR
通讯作者：
Crabtree GR
影响因子：
12.6
作者：
Aguiar S;van der Gaag B;Cortese FAB
通讯作者：
Cortese FAB
影响因子：
8
作者：
Amina SJ;Guo B
通讯作者：
Guo B