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Comparison of Insulators and Promoters for Expression of the Wiskott–Aldrich Syndrome Protein Using Lentiviral Vectors

Gene therapy for the treatment of Wiskott–Aldrich syndrome (WAS) presents an alternative to the current use of allogeneic bone marrow transplantation. We describe the development of a self-inactivating lentiviral vector containing chromatin insulators for treatment of WAS and compare a gammaretrovir...

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Autors principals: Koldej, Rachel M., Carney, Gael, Wielgosz, Matthew M., Zhou, Sheng, Zhan, Jun, Sorrentino, Brian P., Nienhuis, Arthur W.
Format: Artigo
Idioma:Inglês
Publicat: Mary Ann Liebert, Inc. 2013
Matèries:
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC4003467/
https://ncbi.nlm.nih.gov/pubmed/23786330
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/humc.2012.244
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