Hurdles Associated with the Translational Use of Genetically Modified Cells.

IF 2.3 Q4 CELL & TISSUE ENGINEERING

Current Stem Cell Reports Pub Date : 2018-03-01 Epub Date: 2018-02-17 DOI:10.1007/s40778-018-0115-y

Sunil S Raikar, H Trent Spencer

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Abstract

Purpose of review: Recent advancements in the use of genetically modified hematopoietic stem cells (HSCs) and the emergent use of chimeric antigen receptor (CAR) T-cell immunotherapy has highlighted issues associated with the use of genetically engineered cellular products. This review explores some of the challenges linked with translating the use of genetically modified cells.

Recent findings: The use of genetically modified HSCs for ADA-SCID now has European approval and the U.S. Food and Drug Administration recently approved the use of CAR-T cells for relapsed/refractory B-cell acute lymphoblastic leukemia. Current good manufacturing processes have now been developed for the collection, expansion, storage, modification, and administration of genetically modified cells.

Summary: Genetically engineered cells can be used for several therapeutic purposes. However, significant challenges remain in making these cellular therapeutics readily available. A better understanding of this technology along with improvements in the manufacturing process is allowing the translation process to become more standardized.

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与转基因细胞转化使用相关的障碍。

综述目的:最近在使用转基因造血干细胞(hsc)和嵌合抗原受体(CAR) t细胞免疫治疗方面的进展突出了与使用基因工程细胞产品相关的问题。这篇综述探讨了一些与翻译使用转基因细胞相关的挑战。最近的发现:基因修饰的造血干细胞用于ADA-SCID目前已获得欧洲批准，美国食品和药物管理局最近批准使用CAR-T细胞治疗复发/难治性b细胞急性淋巴细胞白血病。目前良好的生产工艺已经开发出来，用于收集、扩增、储存、修改和管理转基因细胞。摘要:基因工程细胞可用于多种治疗目的。然而，在使这些细胞疗法易于获得方面仍然存在重大挑战。对这项技术的更好理解以及制造过程的改进使翻译过程变得更加标准化。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Current Stem Cell Reports Biochemistry, Genetics and Molecular Biology-Genetics

CiteScore

3.30

自引率

0.00%

发文量

期刊介绍： The goal of this journal is to publish cutting-edge reviews on subjects pertinent to all aspects of stem cell research, therapy, ethics, commercialization, and policy. We aim to provide incisive, insightful, and balanced contributions from leading experts in each relevant domain that will be of immediate interest to a wide readership of clinicians, basic scientists, and translational investigators. We accomplish this aim by appointing major authorities to serve as Section Editors in key subject areas across the discipline. Section Editors select topics to be reviewed by leading experts who emphasize recent developments and highlight important papers published over the past year on their topics, in a crisp and readable format. We also provide commentaries from well-known figures in the field, and an Editorial Board of internationally diverse members suggests topics of special interest to their country/region and ensures that topics are current and include emerging research.

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