Therapeutic genome editing: prospects and challenges
Top Cited Papers
Open Access
- 5 February 2015
- journal article
- review article
- Published by Springer Science and Business Media LLC in Nature Medicine
- Vol. 21 (2), 121-131
- https://doi.org/10.1038/nm.3793
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.Keywords
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