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European Biopharmaceutical Review

Novel Approaches to Overcome CRISPR In Vitro Delivery Challenges

The discovery and development of the CRISPR system as a gene editing tool has been a major revolution in life science, from human health in gene and cell therapy, agricultural sciences, to fundamental biology where it is now used as a standard tool (1). Although CRISPR is widely used in research laboratories, many challenges regarding delivery and safety remain to be overcome, for both in vivo and in vitro applications, to fulfil its potential.

Safety is a key concern when considering gene therapy. Offtarget mutations of the genome need to be minimised (2). This is also true when working in fundamental science, for example, when studying a signalling pathway where scientists need to ensure that the observed effect is caused by the targeted mutation and not off-target sites.

Another major challenge is the delivery of the CRISPR/Cas system into the nucleus of the targeted cells. In vivo is needed to pass through several barriers and be able to target a specific cell inside of a living organism. In vitro, the delivery of CRISPR/ Cas system components into cell nuclei is often limited by cytotoxic and low efficiency methods. Batch transfection using lipofectamine or electroporation is well suited when working with immortalised cell lines and performing single knock out, which requires the delivery of a single guide RNA (gRNA) together with the Cas9 protein. The inherent cytotoxicity of these standard methods is overcome by the high number of cells that are available. In turn, these methods are much less appropriate when working with hard-to-transfect cells, large repair templates for homologous directed repair, or for multiplexing projects where many edits are required. Here, the use of different and novel transfection approaches becomes critical.

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Dr Paul Monnier obtained his PhD in Molecular Biology from University Pierre and Marie Curie in Paris, France, while working on non-coding RNAs involved in epigenetic growth control of the embryo. After a postdoctoral position at ETH Zurich, Switzerland, and working on CRISPR genetic screens in stem cells and X-chromosome inactivation, he joined Cytosurge to set up their new biology lab. Here, he develops applications and protocols related to FluidFM technology in the life science field.
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Dr Paul Monnier
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