CRISPR/Cas9基因组编辑:Duane回缩综合征的未来治疗

B. W. Wardhani
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引用次数: 0

摘要

Duane挛缩综合征(DRS)以眼球运动受限为特征。其原因之一是CHN1、MAFB或SALL4基因的突变。目前,DRS的治疗仅限于眼镜、遮挡和手术。然而,这种治疗方法并不能治愈这种疾病的遗传问题。另一种被考虑用于治疗的策略是CRISPR/Cas9,这是一种进行基因编辑的工具,具有广泛的应用,包括治疗遗传性疾病。我们通过CCTop网站制作了sgRNA,作为使用CRISPR/Cas9作为硅片治疗DRS的第一步。通过计算sgRNA,进行测试并分析结果,CRISPR/Cas9可以修复基因突变。目前,尚无关于CRISPR/Cas9在DRS中使用的报道。因此,这项研究将是使用CRISPR/Cas9作为DRS治疗的一个非常有用的起点。然而,它需要进一步通过体内、体外和临床试验研究来证明。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
CRISPR/Cas9 Genome Editing: Future Treatment of Duane Retraction Syndrome
Duane Retraction Syndrome (DRS) is characterized by limited eye movement. One of its causes is a mutation in the CHN1, MAFB, or SALL4 gene. Nowadays, the treatment for DRS is limited to glasses, occlusion, and surgery. However, this treatment has not been able to cure the disease’s hereditary issue. Another strategy to be considered for the treatment is CRISPR/Cas9, a tool for performing gene editing with a wide range of applications, including treating genetic diseases. We made sgRNA as a first step in using CRISPR/Cas9 as a treatment for DRS in silico using the CCTop website. By computing sgRNA, conducting tests, and analyzing the results, CRISPR/Cas9 may repair genetic mutations. Currently, there are no reports on the use of CRISPR/Cas9 in DRS. Hence, this study would be very useful as a starting point for using CRISPR/Cas9 as a DRS treatment. However, it needs to be further proven through in vivo, in vitro, and clinical trials study.
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