Therapeutic genome editing: prospects and challenges


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Date

2015-02

Publication Type

Journal Article

ETH Bibliography

no

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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.

Publication status

published

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Volume

21 (2)

Pages / Article No.

121 - 131

Publisher

Nature

Event

Edition / version

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Organisational unit

09580 - Platt, Randall / Platt, Randall check_circle

Notes

Published online 5 February 2015.

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