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CRISPR Diagnosis and Therapeutics with Single Base Pair Precision.
Trends in Molecular Medicine ( IF 12.8 ) Pub Date : 2019-11-29 , DOI: 10.1016/j.molmed.2019.09.008
Seung Hwan Lee 1 , Young-Ho Park 2 , Yeung Bae Jin 1 , Sun-Uk Kim 3 , Junho K Hur 4
Affiliation  

Clustered regularly interspaced short palindromic repeats, or CRISPR, has been widely accepted as a versatile genome editing tool with significant potential for medical application. Reliable allele specificity is one of the most critical elements for successful application of this technology to develop high-precision therapeutics and diagnostics. CRISPR-based genome editing tools achieve high-fidelity distinction of single-base differences in target genomic loci by structural identification of CRISPR-associated (Cas) proteins and sequences of the guide RNAs. In this review, we describe the structural features of ribonucleoprotein complex formation by CRISPR proteins and guide RNAs that eventually recognize target DNA sequences. This structural understanding provides the basis for the recent applications of enhanced single-base precision genome editing technologies for effective distinction of specific alleles.

中文翻译:

具有单碱基对精度的CRISPR诊断和治疗。

簇状规则间隔的短回文重复序列或CRISPR已被广泛接受为具有广泛医学应用潜力的通用基因组编辑工具。可靠的等位基因特异性是成功应用该技术开发高精度治疗和诊断方法的最关键要素之一。基于CRISPR的基因组编辑工具通过与CRISPR相关的(Cas)蛋白质和指导RNA序列的结构鉴定,实现了目标基因组基因座中单碱基差异的高保真区分。在这篇综述中,我们描述了由CRISPR蛋白形成的核糖核蛋白复合物的结构特征,并指导了最终识别靶DNA序列的RNA。
更新日期:2019-11-30
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