基于CRISPR/ cas的诊断和基因治疗

Qiu Meiyu, Liping Pei
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引用次数: 3

摘要

聚类规则间隔短回文重复序列(CRISPR)技术是一种简单、快速、经济、精确的基因编辑技术,已经彻底改变了诊断和基因治疗。快速、准确的疾病诊断对于即时检测(POCT)和专业医疗机构至关重要。crispr相关(Cas)蛋白系统以其优势揭示了在护理点(POC)的新诊断方法。此外,基于CRISPR/ cas的基因编辑技术也带来了基因治疗方面的各种突破。它已被用于临床试验,治疗各种不治之症,包括癌症、血液疾病和其他综合症。目前,CRISPR/Cas的临床应用主要集中在体外治疗。近年来,基于CRISPR-Cas9的体外基因治疗已经取得了巨大的进展。尽管做出了这些努力,体内CRISPR/Cas基因治疗仍处于初级阶段。在这里,我们回顾了CRISPR/Cas技术在诊断和基因治疗领域的里程碑。我们还重点介绍了基于CRISPR/Cas技术的诊断和基因治疗的最新进展。在最后一节中,我们讨论了CRISPR/Cas技术在诊断和基因治疗方面未来临床应用的优势和重大挑战。
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CRISPR/Cas-based Diagnostics and Gene Therapy
Clustered regularly interspaced short palindromic repeats (CRISPR) technology, an easy, rapid, cost-effective, and precise gene-editing technique, has revolutionized diagnostics and gene therapy. Fast and accurate diagnosis of diseases is essential for point-of-care-testing (POCT) and specialized medical institutes. The CRISPR-associated (Cas) proteins system shed light on the new diagnostics methods at point-of-care (POC) owning to its advantages. In addition, CRISPR/Cas-based gene-editing technology has led to various breakthroughs in gene therapy. It has been employed in clinical trials for a variety of untreatable diseases, including cancer, blood disorders, and other syndromes. Currently, the clinical application of CRISPR/Cas has been mainly focused on ex vivo therapies. Recently, tremendous efforts have been made in the development of ex vivo gene therapy based on CRISPR-Cas9. Despite these efforts, in vivo CRISPR/Cas gene therapy is only in its initial stage. Here, we review the milestones of CRISPR/Cas technologies that advanced the field of diagnostics and gene therapy. We also highlight the recent advances of diagnostics and gene therapy based on CRISPR/Cas technology. In the last section, we discuss the strength and significant challenges of the CRISPR/Cas technology for its future clinical usage in diagnosis and gene therapy.
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