Transcription activator-like effector nuclease-mediated transduction of exogenous gene into IL2RG locus.

Abstract:

:X-linked severe combined immunodeficiency (SCID-X1) caused by mutations in interleukin 2 receptor gamma (IL2RG) gene threatens the survival of affected boys during the first year of life unless hematopoietic stem cell transplantation is provided. Although viral vector-mediated gene therapy has been successfully performed in patients with no HLA-matched donors, leukemia caused by vector-mediated insertional mutagenesis has been reported in some individuals. Transcription activator-like effector nuclease (TALEN) is an artificial sequence-specific endonuclease that is expected to revolutionize the precise correction of disease-causing mutations and eliminate the risk of insertional mutagenesis. Here, we report TALEN-mediated genome editing of the IL2RG locus. We transfected TALENs along with a targeting vector into Jurkat cells, and we confirmed the precise introduction of the exogenous gene into the IL2RG locus. In addition, we found that the length of homology arm in the targeting vector influenced the efficiency of TALEN-mediated homologous recombination.

journal_name

Sci Rep

journal_title

Scientific reports

authors

Matsubara Y,Chiba T,Kashimada K,Morio T,Takada S,Mizutani S,Asahara H

doi

10.1038/srep05043

subject

Has Abstract

pub_date

2014-05-23 00:00:00

pages

5043

issn

2045-2322

pii

srep05043

journal_volume

4

pub_type

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