siRNA-mediated Allele-specific Silencing of a COL6A3 Mutation in a Cellular Model of Dominant Ullrich Muscular Dystrophy

siRNA-mediated Allele-specific Silencing of a COL6A3 Mutation in a Cellular Model of Dominant Ullrich Muscular Dystrophy

Congenital muscular dystrophy type Ullrich (UCMD) is a severe disorder of early childhood onset for which currently there is no effective treatment. UCMD commonly is caused by dominant-negative mutations in the genes coding for collagen type VI, a major microfibrillar component of the extracellular...

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Bibliographic Details
Main Authors: Véronique Bolduc, Yaqun Zou, Dayoung Ko, Carsten G Bönnemann
Format: Article
Language:English
Published: Elsevier 2014-01-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
allele-specific siRNA
collagen type VI
dominant negative
Ullrich congenital muscular dystrophy
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253116302876

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