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Genome Biol. 2015 Dec 22;16:286. doi: 10.1186/s13059-015-0859-y.

Towards a new era in medicine: therapeutic genome editing.

Author information

1
Department of Pediatrics, Stanford University, Welch Road, Stanford, CA, 94305, USA. mporteus@stanford.edu.

Abstract

Genome editing is the process of precisely modifying the nucleotide sequence of the genome. It has provided a powerful approach to research questions but, with the development of a new set of tools, it is now possible to achieve frequencies of genome editing that are high enough to be useful therapeutically. Genome editing is being developed to treat not only monogenic diseases but also infectious diseases and diseases that have both a genetic and an environmental component.

PMID:
26694713
PMCID:
PMC4699361
DOI:
10.1186/s13059-015-0859-y
[Indexed for MEDLINE]
Free PMC Article

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