基因组编辑 T 细胞疗法的监管注意事项。
Regulatory Considerations for Genome-Edited T-cell Therapies.
发表日期:2024 Jul 17
作者:
Julie K Jadlowsky, Ju-Fang Chang, David H Spencer, John M Warrington, Bruce L Levine, Carl H June, Joseph A Fraietta, Nathan Singh
来源:
Cancer Immunology Research
摘要:
用于治疗干预的人类细胞基因组工程方法继续以惊人的速度发展。嵌合抗原受体工程化 T 淋巴细胞开创了这些疗法的先河,最初是使用经典基因治疗载体将嵌合抗原受体转基因插入 T 细胞基因组。广泛使用基于成簇规则间隔短回文重复序列 (CRISPR) 的技术来编辑内源基因,现已打开了精准医学新时代的大门。为了增加复杂性,许多正在开发的工程细胞疗法将基因疗法与基因组编辑相结合,以引入新的生物功能并增强治疗效果。在此,我们回顾了基因编辑细胞产品的科学、转化和监管监督的现状。©2024 美国癌症研究协会。
Methods to engineer the genomes of human cells for therapeutic intervention continue to advance at a remarkable pace. Chimeric antigen receptor-engineered T lymphocytes have pioneered the way for these therapies, initially beginning with insertions of chimeric antigen receptor transgenes into T-cell genomes using classical gene therapy vectors. The broad use of clustered regularly interspaced short palindromic repeats (CRISPR)-based technologies to edit endogenous genes has now opened the door to a new era of precision medicine. To add complexity, many engineered cellular therapies under development integrate gene therapy with genome editing to introduce novel biological functions and enhance therapeutic efficacy. Here, we review the current state of scientific, translational, and regulatory oversight of gene-edited cell products.©2024 American Association for Cancer Research.