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

• Main Authors: Véronique Bolduc (Author), Yaqun Zou (Author), Dayoung Ko (Author), Carsten G Bönnemann (Author)
• Formato: Livro
• Publicado em: Elsevier, 2014-01-01T00:00:00Z.
• Assuntos: article
• Acesso em linha: Connect to this object online.

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...