4.8 Review

Single-cell transfection technologies for cell therapies and gene editing

Journal

JOURNAL OF CONTROLLED RELEASE
Volume 330, Issue -, Pages 963-975

Publisher

ELSEVIER
DOI: 10.1016/j.jconrel.2020.10.068

Keywords

Single-cell; Transfection; Intracellular delivery; Cell therapies; Gene editing; Electroporation; Optoporation; Cell squeezing; Microfluidic vortex shedding; Microinjection; Good manufacturing practices

Funding

  1. [1S84919N]

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Recent advancements in gene editing and cell therapies have shown significant clinical successes, but the lack of a cost-effective manufacturing process hinders widespread adoption. Single-Cell Transfection Technologies (SCTTs) offer promising advantages such as higher efficiency and safety, but are currently underutilized in clinical settings.
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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