“…These concerns are heightened in the case of b-thalassemia and sickle cell disease, which use complex tissue-specific vectors with lower titers than the more commonly used complementary DNA-encoding vectors. 13,16,17 Furthermore, adult subjects will be treated on these trials, in contrast to the primarily pediatric subjects treated for metabolic disorders and severe immune deficiencies. 18 Li et al 19 previously administered G-CSF in 20 patients with b thalassemia aged 3-to 21-year-old, but no quantitative data on the yield or composition of the apheresis products were provided.…”