Safe and Efficient In Vivo Hematopoietic Stem Cell Transduction in Nonhuman Primates Using HDAd5/35⁺⁺ Vectors

The authors 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 expression of human γ−globin in red blood cells to treat hemoglobinopathies.