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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Detalhes bibliográficos
Publicado no:Stem Cell Reports
Main Authors: 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.
Formato: Artigo
Idioma:Inglês
Publicado em: Elsevier 2020
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Article
Acesso em linha: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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