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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Gepubliceerd in:Stem Cell Reports
Hoofdauteurs: 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.
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: Elsevier 2020
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Online toegang: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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