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European Biopharmaceutical Review

In the Genes

Many people take pleasure in knowing that the phrase “D’oh” originates from the TV series ‘The Simpsons’, whereas those who really know smugly attribute it to Jimmy Finlayson from the Laurel and Hardy films of the 1920s and 1930s. In much the same way, many people think that stem cells and gene-editing are also new inventions. Indeed, if one were to anthropomorphise two of the newest biotechnology tools, most people would regard stem cell technology as a junior-school prodigy, and its younger brother, gene-editing, as a precocious preschooler.

This year, 2016, does indeed mark the tenth anniversary of the first description of pluripotent stem cells induced from mammalian adult fibroblast cultures by defined factors – and fewer than four years ago, a programmable, dual-RNA-guided DNA endonuclease called Cas9 caught the imagination of thousands of laboratories across the globe. Variants of these tools have enjoyed a long and successful history, winning Nobel prizes and saving lives long before their current incarnations were even a glint in their inventors’ eyes.

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Patrick Harrison is a Molecular Biologist at University College Cork, and his research is sponsored by the Cystic Fibrosis Trust. He began using gene-editing as a research tool for rare diseases in 2005, and his group was the first to publish a gene-editing strategy to correct the most common CF mutation in cells using ZFNs. His lab is currently using CRISPR-Cas9 and Cpf1 to study CF and other rare diseases, including cystinosis and epidermolysis bullosa.
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