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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Vydáno v:Mol Ther Methods Clin Dev
Hlavní autoři: 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.
Médium: Artigo
Jazyk:Inglês
Vydáno: American Society of Gene & Cell Therapy 2021
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Original Article
On-line přístup: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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