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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...
Autores principales: | Matsubara, Yohei, Chiba, Tomoki, Kashimada, Kenichi, Morio, Tomohiro, Takada, Shuji, Mizutani, Shuki, Asahara, Hiroshi |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Nature Publishing Group
2014
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4031469/ https://www.ncbi.nlm.nih.gov/pubmed/24853770 http://dx.doi.org/10.1038/srep05043 |
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