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Safe and efficient in vivo hematopoietic stem cell transduction in nonhuman primates using HDAd5/35++ vectors

We tested a new in vivo hematopoietic stem cell (HSC) transduction/selection approach in rhesus macaques using HSC-tropic, integrating, helper-dependent adenovirus vectors (HDAd5/35++) designed for the expression of human γ-globin in red blood cells (RBCs) to treat hemoglobinopathies. We show that H...

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Detalles Bibliográficos
Autores principales: Li, Chang, Wang, Hongjie, Gil, Sucheol, Germond, Audrey, Fountain, Connie, Baldessari, Audrey, Kim, Jiho, Liu, Zhinan, Georgakopoulou, Aphrodite, Radtke, Stefan, Raskó, Tamás, Pande, Amit, Chiang, Christina, Chin, Eli, Yannaki, Evangelia, Izsvák, Zsuzsanna, Papayannopoulou, Thalia, Kiem, Hans-Peter, Lieber, André
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8741415/
https://www.ncbi.nlm.nih.gov/pubmed/35036470
http://dx.doi.org/10.1016/j.omtm.2021.12.003