CRISPR/Cas9 技术在临床中的应用：基因编辑的最新用途和肿瘤治疗的新应用。
CRISPR/Cas9 for the Clinician: Current uses of gene editing and applications for new therapeutics in oncology.
机构信息
Drexel University College of Medicine, Philadelphia, PA.
George Washington University Hospital, Washington, DC 20037.
出版信息
Perm J. 2020 Dec;24:1-3. doi: 10.7812/TPP/20.040.
Precise genomic editing has given rise to treatments in previously untreatable genetic diseases and has led to revolutions in treatment for cancer. In the past decade, the discovery and development of clustered regularly interspaced short palindromic repeats (CRISPR) technologies has led to advances across medicine and biotechnology. Specifically, the CRISPR/Cas9 system has improved translational discovery and therapeutics for oncology across tumor types. In this review, we briefly summarize the history and development of CRISPR, explain CRISPR-Cas systems and CRISPR gene editing tools, highlight the development and application of CRISPR technologies for translational and therapeutic purposes in different oncologic tumors, and review novel treatment paradigms using CRISPR in immuno-oncology, including checkpoint inhibitors and chimeric antigen receptor T cell therapy.
精确的基因组编辑为以前无法治疗的遗传疾病带来了治疗方法，并引发了癌症治疗的革命。在过去的十年中，规律成簇间隔短回文重复序列（CRISPR）技术的发现和发展推动了医学和生物技术的进步。具体来说，CRISPR/Cas9 系统提高了跨肿瘤类型的肿瘤学的转化发现和治疗。在这篇综述中，我们简要总结了 CRISPR 的历史和发展，解释了 CRISPR-Cas 系统和 CRISPR 基因编辑工具，强调了 CRISPR 技术在不同肿瘤的转化和治疗目的中的开发和应用，并回顾了使用 CRISPR 在免疫肿瘤学中的新型治疗模式，包括检查点抑制剂和嵌合抗原受体 T 细胞疗法。
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