Genomics


Gene editing for improved model generation Precision gene editing techniques, such as CRISPR- Cas9, are enabling the development of more com- plex disease cell models, especially in cell lines that are more difficult to culture and transfect, such as primary cells or induced pluripotent stem cells. Able to create more cleavage sites in the genome, these tools allow mutations to be inserted or repaired more efficiently and with greater precision in order to create cell lines that more closely model human disease states. The improved efficiency of CRISPR-Cas9 potentially allows proteins to be studied at native levels, in contrast to more tradi- tional approaches that typically rely on protein overexpression. Moreover, the cleavage efficiency that is possible


with the latest generation of CRISPR-Cas9 tools could potentially help to bring down the cost of developing new models, an opportunity that is exciting Jon Chesnut, Senior Director of Synthetic Biology R&D at Thermo Fisher Scientific. “In many cases we can routinely cleave up to 90% or even higher of the gene loci in the popula-


tion,” he tells DDW. “At this level of efficiency, we can start to think about automating these pro- cesses.” Streamlining workflows in that way could


improve the predictability of getting the desired clone and would ultimately lower the cost of pro- ducing cell lines. “If we’re able to produce more cell lines, more


predictably, at a reasonable cost, we can look dif- ferently at how we set up our experiments,” says Chesnut. “Instead of looking at one mutation at a time, we might be able to look at multiple muta- tions in a particular cell, for the same cost, using a more systems-based approach.”


Commercially-available gene editing technologies A number of manufacturers have made high- throughput CRISPR-Cas9 gene editing tools avail- able on the market. Nucleofection is a proven method for the deliv-


ery of genome editing substrates, including DNA, mRNA and Cas9 RNPs, and Lonza offers a range of technologies built around this approach. The


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Drug Discovery World Spring 2018


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