Human miR223 Promoter as a Novel Myelo-Specific Promoter for Chronic Granulomatous Disease Gene Therapy

Targeting transgene expression to specific hematopoietic cell lineages could contribute to the safety of retroviral vectors in gene therapeutic applications. Chronic granulomatous disease (CGD), a defect of phagocytic cells, can be managed by gene therapy, using retroviral vectors with targeted expr...

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Autori principali: Brendel, Christian, Hänseler, Walther, Wohlgensinger, Vital, Bianchi, Matteo, Tokmak, Serap, Chen-Wichmann, Linping, Kuzmenko, Elena, Cesarovic, Nikola, Nicholls, Flora, Reichenbach, Janine, Seger, Reinhard, Grez, Manuel, Siler, Ulrich
Natura: Artigo
Lingua:Inglês
Pubblicazione: Mary Ann Liebert, Inc. 2013
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Article
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC3732129/
https://ncbi.nlm.nih.gov/pubmed/23489116
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hgtb.2012.157
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