Splice-correcting oligonucleotides restore BTK function in X-linked agammaglobulinemia model

X-linked agammaglobulinemia (XLA) is an inherited immunodeficiency that results from mutations within the gene encoding Bruton’s tyrosine kinase (BTK). Many XLA-associated mutations affect splicing of BTK pre-mRNA and severely impair B cell development. Here, we assessed the potential of antisense,...

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Autors principals: Bestas, Burcu, Moreno, Pedro M.D., Blomberg, K. Emelie M., Mohammad, Dara K., Saleh, Amer F., Sutlu, Tolga, Nordin, Joel Z., Guterstam, Peter, Gustafsson, Manuela O., Kharazi, Shabnam, Piątosa, Barbara, Roberts, Thomas C., Behlke, Mark A., Wood, Matthew J.A., Gait, Michael J., Lundin, Karin E., El Andaloussi, Samir, Månsson, Robert, Berglöf, Anna, Wengel, Jesper, Smith, C.I. Edvard
Format: Artigo
Idioma:Inglês
Publicat: American Society for Clinical Investigation 2014
Matèries:
Research Article
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC4151202/
https://ncbi.nlm.nih.gov/pubmed/25105368
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1172/JCI76175
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