【佳学基因检测】CRISPR/dCas9 基因治疗神经发育障碍：与传统策略相比的创新和局限性

靶向药一般多少钱排名

研究高效抑制神经系统合并症的方法与药物时看到《Dev Neurosci》在2021;43(3-4):253-261发表了一篇题目为《CRISPR/dCas9 作为神经发育障碍的治疗方法：与传统策略相比的创新和局限性》神经系统疾病的基因修饰治疗的临床研究文章。该研究由Raffaele Ricci, Gaia Colasante等完成。促进了基因编辑技术等正确治疗手段在神经发育障碍领域的应用，在基因检测指导下的创新疗法即有创新性也有一定的局限性，为基因解码的进一步发展提出了新的课题。

神经疾病遗传阻断及正确治疗临床研究内容关键词：

CRISPR,dCas9,基因治疗,神经发育障碍,精神疾病,正确治疗

精神类疾病用药指导基因检测临床应用结果

大脑发育是一个复杂的过程，需要一系列正确并相互协调的事件发生。当基因检测到的突变使得其中一些事件发生改变时，可能会出现神经发育障碍 (NDD)，并出现其特征性症状，包括认知、社交运动缺陷和癫痫。虽然多年来药物治疗一直是唯一的治疗选择，但最近的研究转向直接消除每个特定神经发育障碍的致病基因突变。这要归功于对这些疾病的基因解码基础知识的增加以及基因组编辑工具的巨大进步。与基于聚集规则间隔短回文重复 (CRISPR)/Cas9 的策略一起，核酸酶缺陷 Cas9 (dCas9) 工具也有了很大的发展，该工具具有极大的灵活性，允许将特定的蛋白质功能募集到所需的基因组位点。在这项工作中，佳学基因解码回顾了基于 dCas9 的工具，并讨论了所有已发表的在临床前水平的神经发育障碍治疗方法设置中的应用。特别是针对 Dravet 综合征、C11orf46 基因突变引起的胼胝体连接障碍和脆性 X 综合征的基于 dCas9 的治疗策略进行了介绍和讨论。提供了与其他可能的治疗策略的直接比较，例如经典基因替换或基于 CRISPR/Cas9 的策略。佳学基因检测不仅强调了与以前的策略相比具有明显优势的那些方面，而且还强调了与其应用相关的需要克服的主要技术障碍。关键词：CRISPR/dCas9；基因治疗;神经发育障碍。

神经及精神疾病及其并发征、合并征国际数据库描述：

Brain development is a complex process that requires a series of precise and coordinated events to take place. When alterations in some of those events occur, neurodevelopmental disorders (NDDs) may appear, with their characteristic symptoms, including cognitive, social motor deficits, and epilepsy. While pharmacologic treatments have been the only therapeutic options for many years, more recently the research is turning to the direct removal of the underlying genetic cause of each specific NDD. This is possible thanks to the increased knowledge of genetic basis of those diseases and the enormous advances in genome-editing tools. Together with clustered regularly interspaced short palindromic repeats (CRISPR)/Cas9-based strategies, there is a great development also of nuclease defective Cas9 (dCas9) tools that, with an extreme flexibility, allow the recruitment of specific protein functions to the desired genomic sites. In this work, we review dCas9-based tools and discuss all the published applications in the setting of therapeutic approaches for NDDs at the preclinical level. In particular, dCas9-based therapeutic strategies for Dravet syndrome, transcallosal dysconnectivity caused by mutations in C11orf46 gene, and Fragile X syndrome are presented and discussed. A direct comparison with other possible therapeutic strategies, such as classic gene replacement or CRISPR/Cas9-based strategies, is provided. We also highlight not only those aspects that constitute a clear advantage compared to previous strategies but also the main technical hurdles related to their applications that need to be overcome.Keywords: CRISPR/dCas9; Gene therapy; Neurodevelopmental disorders.

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