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

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; Piatosa, 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

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; Piatosa, 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.

J Clin Invest ; 124(9): 4067-81, 2014 Sep.

Article in En | MEDLINE | ID: mdl-25105368

Subject(s)

Agammaglobulinemia/therapy; Genetic Diseases, X-Linked/therapy; Oligonucleotides/genetics; Protein-Tyrosine Kinases/physiology; RNA Splicing; Agammaglobulinaemia Tyrosine Kinase; Agammaglobulinemia/enzymology; Animals; B-Lymphocytes/metabolism; Cells, Cultured; Genetic Diseases, X-Linked/enzymology; Humans; Luciferases/genetics; Mice, Transgenic; Monocytes/enzymology; Protein-Tyrosine Kinases/genetics

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Full text: 1 Database: MEDLINE Main subject: Oligonucleotides / Protein-Tyrosine Kinases / RNA Splicing / Agammaglobulinemia / Genetic Diseases, X-Linked Limits: Animals / Humans Language: En Journal: J Clin Invest Year: 2014 Type: Article

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