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

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...

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Bibliographic Details
Main Authors: Matsubara, Yohei, Chiba, Tomoki, Kashimada, Kenichi, Morio, Tomohiro, Takada, Shuji, Mizutani, Shuki, Asahara, Hiroshi
Format: Artigo
Language:Inglês
Published: Nature Publishing Group 2014
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Article
Online Access:https://ncbi.nlm.nih.gov/pmc/articles/PMC4031469/
https://ncbi.nlm.nih.gov/pubmed/24853770
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/srep05043
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