siRNA-mediated Allele-specific Silencing of a COL6A3 Mutation in a Cellular Model of Dominant Ullrich Muscular Dystrophy
Author:
Publisher
Elsevier BV
Subject
Drug Discovery,Molecular Medicine
Reference42 articles.
1. Ullrich scleroatonic muscular dystrophy is caused by recessive mutations in collagen type VI;Camacho Vanegas;Proc Natl Acad Sci USA.,2001
2. Mutations in COL6A3 cause severe and mild phenotypes of Ullrich congenital muscular dystrophy;Demir;Am J Hum Genet,2002
3. New molecular mechanism for Ullrich congenital muscular dystrophy: a heterozygous in-frame deletion in the COL6A1 gene causes a severe phenotype;Pan;Am J Hum Genet,2003
4. The collagen VI-related myopathies: muscle meets its matrix;Bönnemann;Nat Rev Neurol,2011
5. Natural history of Ullrich congenital muscular dystrophy;Nadeau;Neurology,2009
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3. Optimized allele-specific silencing of the dominant-negative COL6A1 G293R substitution causing collagen VI-related dystrophy;Molecular Therapy - Nucleic Acids;2024-06
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