Therapeutic genome editing: prospects and challenges.
Nat Med
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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. |
Year of Publication | 2015
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Journal | Nat Med
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Volume | 21
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Issue | 2
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Pages | 121-31
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Date Published | 2015 Feb
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ISSN | 1546-170X
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URL | |
DOI | 10.1038/nm.3793
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PubMed ID | 25654603
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PubMed Central ID | PMC4492683
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Grant list | DP1 MH100706 / MH / NIMH NIH HHS / United States
R01 NS073124 / NS / NINDS NIH HHS / United States
T32 GM007753 / GM / NIGMS NIH HHS / United States
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