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Nat Med. 2015 Feb;21(2):121-31. doi: 10.1038/nm.3793.

Therapeutic genome editing: prospects and challenges.

Author information

1
1] Broad Institute of Harvard and MIT, Cambridge, Massachusetts, USA. [2] Department of Health Sciences and Technology, Massachusetts Institute of Technology, Cambridge, Massachusetts, USA. [3] Department of Biology, Massachusetts Institute of Technology, Cambridge, Massachusetts, USA. [4] McGovern Institute for Brain Research at MIT, Cambridge, Massachusetts, USA.
2
1] Broad Institute of Harvard and MIT, Cambridge, Massachusetts, USA. [2] McGovern Institute for Brain Research at MIT, Cambridge, Massachusetts, USA. [3] Department of Brain and Cognitive Sciences, Massachusetts Institute of Technology, Cambridge, Massachusetts, USA. [4] Department of Biological Engineering, Massachusetts Institute of Technology, Cambridge, Massachusetts, USA.

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.

PMID:
25654603
PMCID:
PMC4492683
DOI:
10.1038/nm.3793
[Indexed for MEDLINE]
Free PMC Article

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