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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Main Authors: 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
Sprog:Inglês
Udgivet: American Society for Clinical Investigation 2014
Fag:
Research Article
Online adgang: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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