β-Globin Lentiviral Vectors Have Reduced Titers due to Incomplete Vector RNA Genomes and Lowered Virion Production

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 h...

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Publié dans:Stem Cell Reports
Auteurs principaux: Han, Jiaying, Tam, Kevin, Ma, Feiyang, Tam, Curtis, Aleshe, Bamidele, Wang, Xiaoyan, Quintos, Jason P., Morselli, Marco, Pellegrini, Matteo, Hollis, Roger P., Kohn, Donald B.
Format: Artigo
Langue:Inglês
Publié: Elsevier 2020
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Article
Accès en ligne:https://ncbi.nlm.nih.gov/pmc/articles/PMC7897704/
https://ncbi.nlm.nih.gov/pubmed/33186538
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.stemcr.2020.10.007
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