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Allele Dependent Silencing of Collagen Type I Using Small Interfering RNAs Targeting 3'UTR Indels - a Novel Therapeutic Approach in Osteogenesis Imperfecta

Osteogenesis imperfecta, also known as “brittle bone disease”, is a heterogeneous disorder of connective tissue generally caused by dominant mutations in the genes COL1A1 and COL1A2, encoding the α1 and α2 chains of type I (pro)collagen. Symptomatic patients are usually prescribed bisphosphonates, b...

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Detalles Bibliográficos
Autores principales: Lindahl, Katarina, Kindmark, Andreas, Laxman, Navya, Åström, Eva, Rubin, Carl-Johan, Ljunggren, Östen
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Ivyspring International Publisher 2013
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3752721/
https://www.ncbi.nlm.nih.gov/pubmed/23983594
http://dx.doi.org/10.7150/ijms.5774