Article
Splice-correction strategies for treatment of X-linked agammaglobulinemia.
Current allergy and asthma reports - 1 Mar 2015
Bestas Burcu, Turunen Janne J, Blomberg K Emelie M, Wang Qing, Månsson Robert, El Andaloussi Samir, Berglöf Anna, Smith C I Edvard
Abstract excerpt
X-linked agammaglobulinemia (XLA) is a primary immunodeficiency disease caused by mutations in the gene coding for Bruton's tyrosine kinase (BTK). Deficiency of BTK leads to a developmental block in B cell differentiation; hence, the patients essentially lack antibody-producing plasma cells and a...
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