[HTML][HTML] Gene therapy of Diamond Blackfan anemia CD34+ cells leads to improved erythroid development and engraftment following transplantation
OBJECTIVE: Diamond-Blackfan anemia (DBA) is a rare congenital hypoplastic anemia
caused by mutations in ribosomal protein (RP) genes. Our aim is to develop gene therapy
for DBA patients with mutations in RPS19. We previously demonstrated that RPS19 gene
transfer partially corrects erythroid development in vitro. In this study, we asked if RPS19
gene transfer corrects erythroid development in unsorted cells transplanted to
immunodeficient mice and if the RPS19-corrected fraction has a proliferative advantage after …
caused by mutations in ribosomal protein (RP) genes. Our aim is to develop gene therapy
for DBA patients with mutations in RPS19. We previously demonstrated that RPS19 gene
transfer partially corrects erythroid development in vitro. In this study, we asked if RPS19
gene transfer corrects erythroid development in unsorted cells transplanted to
immunodeficient mice and if the RPS19-corrected fraction has a proliferative advantage after …
