Ontology highlight
ABSTRACT:
SUBMITTER: Seymour BJ
PROVIDER: S-EPMC7907679 | biostudies-literature | 2021 Mar
REPOSITORIES: biostudies-literature
Seymour Brenda J BJ Singh Swati S Certo Hannah M HM Sommer Karen K Sather Blythe D BD Khim Socheath S Clough Courtnee C Hale Malika M Pangallo Joseph J Ryu Byoung Y BY Khan Iram F IF Adair Jennifer E JE Rawlings David J DJ
Molecular therapy. Methods & clinical development 20210120
X-linked agammaglobulinemia (XLA) is an immune disorder caused by mutations in Bruton's tyrosine kinase (<i>BTK</i>). 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 that restored B cell development and function in <i>Btk</i> and <i>Tec</i> double knockout mice (a phenocopy of human XLA). In this study, with the goal of optimizing both the level and lineage ...[more]