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,...

ver descrição completa

Na minha lista:
Detalhes bibliográficos
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
Formato: Artigo
Idioma:Inglês
Publicado em: American Society for Clinical Investigation 2014
Assuntos:
Research Article
Acesso em linha: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
Tags: Adicionar Tag
Sem tags, seja o primeiro a adicionar uma tag!

Registos relacionados