Therapeutic genome editing: prospects and challenges
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Zhang_Therapeutic genome.pdf
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444.18 KB
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Adobe PDF
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35818e0cd590bf73a0f9eb39ebd7a387
Author(s)
Cox, David Benjamin Turitz
Platt, Randall Jeffrey
Zhang, Feng
Date Issued
February 2015
Journal
Nature Medicine
Publisher
Springer Nature
Citation
Cox, David Benjamin Turitz, Randall Jeffrey Platt, and Feng Zhang. "Therapeutic genome editing: prospects and challenges." Nature Medicine 21:2 (February 2015), pp.121-131.
Version
Author's final manuscript
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.
Description
Available in PMC 2015 July 06
MIT Department
Harvard University--MIT Division of Health Sciences and Technology
Massachusetts Institute of Technology. Department of Biological Engineering
Massachusetts Institute of Technology. Department of Biology
Massachusetts Institute of Technology. Department of Brain and Cognitive Sciences
McGovern Institute for Brain Research at MIT
Terms of Use
Creative Commons Attribution-Noncommercial-Share Alike
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DOI of Published Version
https://doi.org/10.1038/nm.3793
