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

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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Bibliographic Details
Main Authors: Brenda J. Seymour, Swati Singh, Hannah M. Certo, Karen Sommer, Blythe D. Sather, Socheath Khim, Courtnee Clough, Malika Hale, Joseph Pangallo, Byoung Y. Ryu, Iram F. Khan, Jennifer E. Adair, David J. Rawlings
Format: Article
Language:English
Published: Elsevier 2021-03-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
gene therapy
XLA
UCOE
lentivirus
DNase hypersensitive sites
codon optimization
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050121000073

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