Adenovirus vectors in hematopoietic stem cell genome editing.

ABSTRACT: Genome editing of hematopoietic stem cells (HSCs) represents a therapeutic option for a number of hematological genetic diseases, as HSCs have the potential for self-renewal and differentiation into all blood cell lineages. This review presents advances of genome editing in HSCs utilizing adenovirus vectors as delivery vehicles. We focus on capsid-modified, helper-dependent adenovirus vectors that are devoid of all viral genes and therefore exhibit an improved safety profile. We discuss HSC genome engineering for several inherited disorders and infectious diseases including hemoglobinopathies, Fanconi anemia, hemophilia, and HIV-1 infection by ex vivo and in vivo editing in transgenic mice, nonhuman primates, as well as in human CD34⁺ cells. Mechanisms of therapeutic gene transf

SUBMITTER: Li C 

PROVIDER: S-EPMC10473235 | biostudies-literature | 2019 Dec

REPOSITORIES: biostudies-literature