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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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Detalles Bibliográficos
Autores principales:	Li, Chang, Psatha, Nikoletta, Wang, Hongjie, Singh, Manvendra, Samal, Himanshu Bhusan, Zhang, Wenli, Ehrhardt, Anja, Izsvák, Zsuzsanna, Papayannopoulou, Thalia, Lieber, André
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	American Society of Gene & Cell Therapy 2018
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5948227/ https://www.ncbi.nlm.nih.gov/pubmed/29766024 http://dx.doi.org/10.1016/j.omtm.2018.02.004

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