Cushman-Vokoun Allison, Schmidt Ryan J, Hiemenz Matthew Charles, Fung Mark, Zhang Bing Melody, Bradshaw Georganne, Gandhi Manish, Yao JinJuan, Yohe Sophia, Beckman Amy, Grody Wayne W, Giannikopoulos Petros
From the Department of Pathology & Microbiology, University of Nebraska Medical Center, Omaha (Cushman-Vokoun).
the Department of Pathology and Laboratory Medicine, Children's Hospital of Los Angeles, Los Angeles, California (Schmidt).
Arch Pathol Lab Med. 2023 Aug 23. doi: 10.5858/arpa.2022-0410-CP.
CONTEXT.—: Gene editing-based therapies are currently in development in the areas of oncology, inherited disease, and infectious disease. These potentially life-altering therapies are derived from decades of research in both academic and industry settings that developed technologies rooted in principles and products of nature. However, with such technologic developments come many important considerations, including adverse risks, high cost, and ethical questions.
OBJECTIVE.—: To educate pathologists about gene editing technologies, inform them of potential indications and risks, outline regulatory and practical issues that could affect hospital-based practice and laboratory testing, and advocate that pathologists need to be present at discussions among industry and regulators pertaining to gene editing-based therapies.
DESIGN.—: A Gene Editing Workgroup, facilitated by the College of American Pathologists Personalized Health Care Committee and consisting of pathologists of various backgrounds, was convened to develop an educational paper to serve as a stimulus to increase pathologist involvement and inquiry in gene editing therapeutic and diagnostic implementation.
RESULTS.—: Through multiple discussions and literature review, the workgroup identified potential gaps in pathologists' knowledge of gene editing. Additional topics that could impact pathology and laboratory medicine were also identified and summarized in order to facilitate pathologists as stakeholders in gene editing therapy administration and monitoring and potential use in diagnostics.
CONCLUSIONS.—: Gene editing therapy is a complex but potentially transformative area of medicine. This article serves as an introduction to pathologists to assist them in future discussions with colleagues and potentially identify and alter pathology practices that relate to gene editing.
基于基因编辑的疗法目前正在肿瘤学、遗传性疾病和传染病领域进行研发。这些可能改变生命的疗法源自学术和工业领域数十年的研究，这些研究开发了基于自然原理和产物的技术。然而，随着此类技术的发展，也带来了许多重要的考量因素，包括不良风险、高成本和伦理问题。
对病理学家进行基因编辑技术方面的教育，告知他们潜在的适应症和风险，概述可能影响医院实践和实验室检测的监管及实际问题，并倡导病理学家参与行业与监管机构之间有关基于基因编辑的疗法的讨论。
由美国病理学家学会个性化医疗委员会促成、由不同背景的病理学家组成的基因编辑工作组召开会议，以撰写一篇教育论文，作为促进病理学家更多参与和探讨基因编辑治疗与诊断应用的一种激励。
通过多次讨论和文献回顾，工作组确定了病理学家在基因编辑知识方面的潜在差距。还确定并总结了可能影响病理学和检验医学的其他主题，以方便病理学家作为基因编辑治疗管理与监测以及诊断潜在应用方面的利益相关者。
基因编辑疗法是医学中一个复杂但可能具有变革性的领域。本文旨在向病理学家介绍相关内容，以帮助他们在未来与同事的讨论中，有可能识别并改变与基因编辑相关的病理学实践。
