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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主要な著者: 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
フォーマット: Artigo
言語:Inglês
出版事項: American Society for Clinical Investigation 2014
主題:
Research Article
オンライン･アクセス: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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