Effective, safe, and sustained correction of murine XLA using a UCOE-BTK promoter-based lentiviral vector

X-linked agammaglobulinemia (XLA) is an immune disorder caused by mutations in Bruton’s tyrosine kinase (BTK). BTK is expressed in B and myeloid cells, and its deficiency results in a lack of mature B cells and protective antibodies. We previously reported a lentivirus (LV) BTK replacement therapy t...

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Detalhes bibliográficos
Publicado no:Mol Ther Methods Clin Dev
Main Authors: Seymour, Brenda J., Singh, Swati, Certo, Hannah M., Sommer, Karen, Sather, Blythe D., Khim, Socheath, Clough, Courtnee, Hale, Malika, Pangallo, Joseph, Ryu, Byoung Y., Khan, Iram F., Adair, Jennifer E., Rawlings, David J.
Formato: Artigo
Idioma:Inglês
Publicado em: American Society of Gene & Cell Therapy 2021
Assuntos:
Original Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7907679/
https://ncbi.nlm.nih.gov/pubmed/33718514
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2021.01.007
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