Article
Allele dependent silencing of collagen type I using small interfering RNAs targeting 3'UTR Indels - a novel therapeutic approach in osteogenesis imperfecta.
International journal of medical sciences - 1 Jan 2013
Lindahl Katarina, Kindmark Andreas, Laxman Navya, Åström Eva, Rubin Carl-Johan, Ljunggren Östen
Abstract excerpt
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, but this treatment is neither curative nor sufficient. A promising field is gene silencing through RNA interference....
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