转基因细胞转化应用相关的障碍
Hurdles Associated with the Translational Use of Genetically Modified Cells.
作者信息
Raikar Sunil S, Spencer H Trent
机构信息
Cell and Gene Therapy Program, Department of Pediatrics, Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta and Emory University, Atlanta, GA, USA.
出版信息
Curr Stem Cell Rep. 2018 Mar;4(1):39-45. doi: 10.1007/s40778-018-0115-y. Epub 2018 Feb 17.
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.
综述目的
基因改造造血干细胞（HSCs）应用的最新进展以及嵌合抗原受体（CAR）T细胞免疫疗法的新兴应用凸显了与使用基因工程细胞产品相关的问题。本综述探讨了与基因改造细胞应用转化相关的一些挑战。
最新发现
基因改造的造血干细胞用于腺苷脱氨酶缺乏症重症联合免疫缺陷病（ADA - SCID）目前已获欧洲批准，美国食品药品监督管理局最近批准了CAR - T细胞用于复发/难治性B细胞急性淋巴细胞白血病的治疗。目前已经开发出了用于基因改造细胞的采集、扩增、储存、改造及给药的现行良好生产规范。
总结
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存在重大挑战。对该技术的更好理解以及生产工艺的改进正使转化过程变得更加标准化。
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