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Splice-Correction Strategies for Treatment of X-Linked Agammaglobulinemia
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 are...
Autores principales: | Bestas, Burcu, Turunen, Janne J., Blomberg, K. Emelie M., Wang, Qing, Månsson, Robert, EL Andaloussi, Samir, Berglöf, Anna, Smith, C. I. Edvard |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Springer US
2015
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4312560/ https://www.ncbi.nlm.nih.gov/pubmed/25638286 http://dx.doi.org/10.1007/s11882-014-0510-0 |
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