Journal
NATURE MEDICINE
Volume 21, Issue 2, Pages 121-131Publisher
NATURE PUBLISHING GROUP
DOI: 10.1038/nm.3793
Keywords
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Funding
- National Institute of General Medical Sciences [T32GM007753]
- National Science Foundation (NSF) Graduate Research Fellowship [1122374]
- National Institute of Mental Health through a US National Institutes of Health (NIH) Director's Pioneer Award [DP1-MH100706]
- National Institute of Neurological Disorders and Stroke through an NIH Transformative R01 grant [R01-NS 07312401]
- NSF Waterman Award
- Keck Foundation
- Damon Runyon Foundation
- Searle Scholars Foundation
- Klingenstein Foundation
- Vallee Foundation
- Merkin Foundation
- Simons Foundation
- Bob Metcalfe
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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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