Therapeutic genome editing: prospects and challenges

DBT Cox, RJ Platt, F Zhang - Nature medicine, 2015 - nature.com
Nature medicine, 2015nature.com
Recent advances in the development of genome editing technologies based on
programmable nucleases have substantially improved our ability to make precise changes
in the genomes of eukaryotic cells. Genome editing is already broadening our ability to
elucidate the contribution of genetics to disease by facilitating the creation of more accurate
cellular and animal models of pathological processes. A particularly tantalizing application
of programmable nucleases is the potential to directly correct genetic mutations in affected …
Abstract
Recent advances in the development of genome editing technologies based on programmable nucleases have substantially improved our ability to make precise changes in the genomes of eukaryotic cells. Genome editing is already broadening our ability to elucidate the contribution of genetics to disease by facilitating the creation of more accurate cellular and animal models of pathological processes. A particularly tantalizing application of programmable nucleases is the potential to directly correct genetic mutations in affected tissues and cells to treat diseases that are refractory to traditional therapies. Here we discuss current progress toward developing programmable nuclease–based therapies as well as future prospects and challenges.
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