Single-cell transfection technologies for cell therapies and gene editing

转染 瓶颈 计算机科学 可扩展性 过程(计算) 基因传递 风险分析(工程) 细胞培养 计算生物学 医学 生物 嵌入式系统 遗传学 数据库 操作系统
作者
Bastien Duckert,Steven Vinkx,Dries Braeken,Maarten Fauvart
出处
期刊:Journal of Controlled Release [Elsevier]
卷期号:330: 963-975 被引量:33
标识
DOI:10.1016/j.jconrel.2020.10.068
摘要

Advances in gene editing and cell therapies have recently led to outstanding clinical successes. However, the lack of a cost-effective manufacturing process prevents the democratization of these innovative medical tools. Due to the common use of viral vectors, the step of transfection in which cells are engineered to gain new functions, is a major bottleneck in making safe and affordable cell products. A promising opportunity lies in Single-Cell Transfection Technologies (SCTTs). SCTTs have demonstrated higher efficiency, safety and scalability than conventional transfection methods. They can also feature unique abilities such as substantial dosage control over the cargo delivery, single-cell addressability and integration in microdevices comprising multiple monitoring modalities. Unfortunately, the potential of SCTTs is not fully appreciated: they are most often restricted to research settings with little adoption in clinical settings. To encourage their adoption, we review and compare recent developments in SCTTs, and how they can enable selected clinical applications. To help bridge the gap between fundamental research and its translation to the clinic, we also describe how Good Manufacturing Practices (GMP) can be integrated in the design of SCTTs.

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