Allele-specific Col1a1 silencing reduces mutant collagen in fibroblasts from Brtl mouse, a model for classical osteogenesis imperfecta

Gene silencing approaches have the potential to become a powerful curative tool for a variety of monogenic diseases caused by gain-of-function mutations. Classical osteogenesis imperfecta (OI), a dominantly inherited bone dysplasia, is characterized in its more severe forms by synthesis of structura...

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Xehetasun bibliografikoak
Egile Nagusiak: Rousseau, Julie, Gioia, Roberta, Layrolle, Pierre, Lieubeau, Blandine, Heymann, Dominique, Rossi, Antonio, Marini, Joan C, Trichet, Valerie, Forlino, Antonella
Formatua: Artigo
Hizkuntza:Inglês
Argitaratua: Nature Publishing Group 2014
Gaiak:
Article
Sarrera elektronikoa:https://ncbi.nlm.nih.gov/pmc/articles/PMC3992561/
https://ncbi.nlm.nih.gov/pubmed/24022296
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/ejhg.2013.198
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