Integrating HDAd5/35++ Vectors as a New Platform for HSC Gene Therapy of Hemoglobinopathies

We generated an integrating, CD46-targeted, helper-dependent adenovirus HDAd5/35++ vector system for hematopoietic stem cell (HSC) gene therapy. The ∼12-kb transgene cassette included a β-globin locus control region (LCR)/promoter driven human γ-globin gene and an elongation factor alpha-1 (EF1α)-mg...

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Detalhes bibliográficos
Publicado no:Mol Ther Methods Clin Dev
Main Authors: Li, Chang, Psatha, Nikoletta, Wang, Hongjie, Singh, Manvendra, Samal, Himanshu Bhusan, Zhang, Wenli, Ehrhardt, Anja, Izsvák, Zsuzsanna, Papayannopoulou, Thalia, Lieber, André
Formato: Artigo
Idioma:Inglês
Publicado em: American Society of Gene & Cell Therapy 2018
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5948227/
https://ncbi.nlm.nih.gov/pubmed/29766024
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2018.02.004
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