Ontology highlight
ABSTRACT:
SUBMITTER: Han J
PROVIDER: S-EPMC7897704 | biostudies-literature | 2021 Jan
REPOSITORIES: biostudies-literature
Han Jiaying J Tam Kevin K Ma Feiyang F Tam Curtis C Aleshe Bamidele B Wang Xiaoyan X Quintos Jason P JP Morselli Marco M Pellegrini Matteo M Hollis Roger P RP Kohn Donald B DB
Stem cell reports 20201112 1
Lentiviral vectors (LVs) commonly used for the treatment of hemoglobinopathies often have low titers and sub-optimal gene transfer efficiency for human hematopoietic stem and progenitor cells (HSPCs), hindering clinical translation and commercialization for ex vivo gene therapy. We observed that a high percentage of β-globin LV viral genomic RNAs were incomplete toward the 3' end in packaging cells and in released vector particles. The incomplete vector genomes impeded reverse transcription in t ...[more]