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Genome Biology

, 16:286

First Online: 22 December 2015


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.

AbbreviationsAAVAdeno-associated virus

allo-HSCTAllogeneic hematopoietic stem cell transplantation

bpBase pairs

CRISPRClustered regularly interspaced short palindromic repeats

DSBDouble-stranded break

FAHFumarylacetoacetate hydrolase

FnFokI nuclease

HRHomologous recombination

HSPCHematopoietic stem-progenitor cell

NHEJNonhomologous end-joining

RNAiRNA interference


RVDRepeat variable di-residue

sgRNASingle-guide RNA

ssODNSingle-stranded oligonucleotide

TALENTAL effector nuclease

ZFNZinc-finger nuclease

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Author: Matthew H. Porteus


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