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碱基编辑助力免疫治疗精准化

英文原题:Immunotherapy to get on point with base editing.

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Immunotherapy to get on point with base editing.

PubMed 2021/04/20(内容时间) Drug Discov Today Q1 · IF 8.7(JCR 2025)

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中文摘要

工程化免疫细胞疗法正在革新癌症治疗领域。美国食品药品监督管理局(FDA)批准了两种用于血液系统恶性肿瘤的嵌合抗原受体(CAR)T 细胞产品,为个体化癌症治疗开辟道路。然而,要成功治疗难治性恶性肿瘤,尤其是实体瘤,仍需进行多重基因编辑以提高 CAR-T 疗效。CRISPR-Cas9 多重编辑的脱靶效应可能妨碍其安全性和临床应用。新型碱基编辑技术提供了一种有前景且更安全的同步编辑替代方案,有望增强用于靶向实体瘤及其他复杂人类疾病的异体工程化免疫疗法。

展开英文摘要原文

Engineered immune cell therapy is revolutionising the field of cancer therapeutics. US Food and Drug Administration (FDA) approval of two chimeric antigen receptor (CAR)-T cell products for the treatment of haematological malignancies paved the way for individualised cancer treatment.

However, multiple genetic edits will be required to improve the efficacy of CAR-T cell therapies if they are to treat refractory malignancies successfully, particularly solid tumours. Off-target effects of CRISPR-Cas9-mediated multiplex editing are likely to hinder its safety and application in the clinic. Novel base editing technologies offer a promising and safer alternative for simultaneous editing that could enhance allogeneic engineered immunotherapies for targeting solid tumours and other complex human diseases.

论文信息

作者
Harbottle JA
单位
Horizon Discovery Ltd, 8100 Cambridge Research Park, Cambridge CB25 9TL, UK. Electronic address: jennifer.harbottle@horizondiscovery.com.United Kingdom
文献类型
综述
期刊
Drug discovery today2021 Oct
原文标识
PubMed 33857616 · DOI 10.1016/j.drudis.2021.04.003