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Generation of a lentiviral vector producer cell clone for human Wiskott-Aldrich syndrome gene therapy
We have developed a producer cell line that generates lentiviral vector particles of high titer. The vector encodes the Wiskott-Aldrich syndrome (WAS) protein. An insulator element has been added to the long terminal repeats of the integrated vector to limit proto-oncogene activation. The vector pro...
Autores principales: | Wielgosz, Matthew M, Kim, Yoon-Sang, Carney, Gael G, Zhan, Jun, Reddivari, Muralidhar, Coop, Terry, Heath, Richard J, Brown, Scott A, Nienhuis, Arthur W |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Nature Publishing Group
2015
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4449020/ https://www.ncbi.nlm.nih.gov/pubmed/26052531 http://dx.doi.org/10.1038/mtm.2014.63 |
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