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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Detalles Bibliográficos
Autores principales: 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: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2020
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7897704/
https://www.ncbi.nlm.nih.gov/pubmed/33186538
http://dx.doi.org/10.1016/j.stemcr.2020.10.007

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