Ontology highlight
ABSTRACT:
SUBMITTER: Li C
PROVIDER: S-EPMC8741415 | biostudies-literature | 2022 Mar
REPOSITORIES: biostudies-literature
Li Chang C Wang Hongjie H Gil Sucheol S Germond Audrey A Fountain Connie C Baldessari Audrey A Kim Jiho J Liu Zhinan Z Georgakopoulou Aphrodite A Radtke Stefan S Raskó Tamás T Pande Amit A Chiang Christina C Chin Eli E Yannaki Evangelia E Izsvák Zsuzsanna Z Papayannopoulou Thalia T Kiem Hans-Peter HP Lieber André A
Molecular therapy. Methods & clinical development 20211206
We tested a new <i>in vivo</i> 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 HDAd5/35++ vectors preferentially transduce HSCs <i>in vivo</i> after intravenous injection into granulocyte colony-stimulating factor (G-CSF)/AMD3100-mobilized animals and that transduced cells ...[more]