WS1.5 Restoration of the CFTR function by antisense oligonucleotide splicing modulation
نویسندگان
چکیده
منابع مشابه
Local dystrophin restoration with antisense oligonucleotide PRO051.
BACKGROUND Duchenne's muscular dystrophy is associated with severe, progressive muscle weakness and typically leads to death between the ages of 20 and 35 years. By inducing specific exon skipping during messenger RNA (mRNA) splicing, antisense compounds were recently shown to correct the open reading frame of the DMD gene and thus to restore dystrophin expression in vitro and in animal models ...
متن کاملRestoration of the cystic fibrosis transmembrane conductance regulator function by splicing modulation.
A significant fraction of disease-causing mutations affects pre-mRNA splicing. These mutations can generate both aberrant and correct transcripts, the level of which varies among different patients. An inverse correlation was found between this level and disease severity, suggesting a role for splicing regulation as a genetic modifier. Overexpression of splicing factors increased the level of c...
متن کاملCorrection of aberrant splicing of the cystic fibrosis transmembrane conductance regulator (CFTR) gene by antisense oligonucleotides.
The CFTR splicing mutation 3849 + 10 kb C --> T creates a novel donor site 10 kilobases (kb) into intron 19 of the gene and is one of the more common splicing mutations that causes cystic fibrosis (CF). It has an elevated prevalence among patients with atypically mild disease and normal sweat electrolytes and is especially prominent in Ashkenazi Jews. This class of splicing mutations, reported ...
متن کاملInhibition of IL-13 by Antisense Oligonucleotide Changes Immunoglobulin Isotype Profile in Cultured B-Lymphocytes
The link between IL-13 and bronchial hyper-responsiveness has brought this cytokine as a potential therapeutic target for asthma and allergic diseases. At the present study, we address the role of B cell derived IL-13 in the IgE and other immunoglobulin development. Antisense oligo for human IL-13 m-RNA was used to study IgE down regulation. Human B-lymphocytes were purified by positive selecti...
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ژورنال
عنوان ژورنال: Journal of Cystic Fibrosis
سال: 2014
ISSN: 1569-1993
DOI: 10.1016/s1569-1993(14)60008-4