CRISPR 基因编辑治疗：实验室医学视角。
Therapeutic Gene Editing with CRISPR: A Laboratory Medicine Perspective.
机构信息
Department of Laboratory Medicine and Pathobiology, University of Toronto, Medical Sciences Building, Room 6231, 1 King's College Circle, Toronto, Ontario M5S 1A8, Canada.
Department of Pathology and Laboratory Medicine, Children's Hospital Los Angeles, Los Angeles, California 90027, USA; Department of Pathology, Keck School of Medicine of USC, Los Angeles, California 90033, USA.
出版信息
Clin Lab Med. 2020 Jun;40(2):205-219. doi: 10.1016/j.cll.2020.02.008.
Therapeutic gene editing with the clustered regularly interspaced short palindromic repeat (CRISPR)-Cas system offers significant improvements in specificity and programmability compared with previous methods. CRISPR editing strategies can be used ex vivo and in vivo with many theoretic disease applications. Off-target effects of CRISPR-mediated gene editing are an important outcome to be aware of, minimize, and detect. The current methods of regulatory approval for personalized therapies are complex and may be proved inefficient as these therapies are implemented more widely. The role of pathologists and laboratory medicine practitioners is vital to the clinical implementation of therapeutic gene editing.
利用成簇规律间隔短回文重复（CRISPR）-Cas 系统进行治疗性基因编辑与以前的方法相比，在特异性和可编程性方面有了显著的提高。CRISPR 编辑策略可用于离体和体内，具有许多理论疾病应用。CRISPR 介导的基因编辑的脱靶效应是需要注意、最小化和检测的重要结果。目前用于个性化治疗的监管批准方法很复杂，随着这些治疗方法的更广泛应用，可能会被证明效率低下。病理学家和实验室医学从业者在治疗性基因编辑的临床实施中发挥着至关重要的作用。
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