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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...
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| Main Authors: | , , , , , , |
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| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
Nature Publishing Group
2014
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| Assuntos: | |
| Acesso em linha: | 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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