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Sci Rep. 2014 May 23;4:5043. doi: 10.1038/srep05043.

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

Author information

1
1] Department of Systems BioMedicine, Tokyo Medical and Dental University Graduate School of Medical and Dental Sciences, Tokyo 113-8510, Japan [2] Department of Pediatrics and Developmental Biology, Tokyo Medical and Dental University Graduate School of Medical and Dental Sciences, Tokyo 113-8510, Japan [3].
2
1] Department of Systems BioMedicine, Tokyo Medical and Dental University Graduate School of Medical and Dental Sciences, Tokyo 113-8510, Japan [2].
3
Department of Pediatrics and Developmental Biology, Tokyo Medical and Dental University Graduate School of Medical and Dental Sciences, Tokyo 113-8510, Japan.
4
Department of Systems BioMedicine, National Research Institute for Child Health and Development, Tokyo 157-8535, Japan.
5
1] Department of Systems BioMedicine, Tokyo Medical and Dental University Graduate School of Medical and Dental Sciences, Tokyo 113-8510, Japan [2] Department of Systems BioMedicine, National Research Institute for Child Health and Development, Tokyo 157-8535, Japan [3] CREST, Japan Science and Technology Agency (JST), Saitama 332-0012, Japan [4] Department of Molecular and Experimental Medicine, The Scripps research Institute, La Jolla, CA 92037, USA.

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.

PMID:
24853770
PMCID:
PMC4031469
DOI:
10.1038/srep05043
[Indexed for MEDLINE]
Free PMC Article

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