RNAi of COL1A1 in mesenchymal progenitor cells

被引：0
作者：
Sophia Millington-Ward
Helena P McMahon
Danny Allen
Gearóid Tuohy
Anna-Sophia Kiang
Arpad Palfi
Paul F Kenna
Peter Humphries
G Jane Farrar
机构：
[1] Ocular Genetics Unit,Department of Genetics
[2] Trinity College Dublin,undefined
来源：
European Journal of Human Genetics | 2004年 / 12卷
关键词：
RNAi; polymorphism; gene therapy; stem cell; Osteogenesis Imperfecta;
D O I：
暂无
中图分类号：
学科分类号：
摘要：
Given that mutant COL1A1 is known to cause Osteogenesis Imperfecta (OI), tools to modulate COL1A1 expression are likely to be of significant therapeutic value. In this context, we have evaluated RNA interference (RNAi) as a means to downregulate COL1A1 expression in Cos-7 cells and in human mesenchymal progenitor stem cells (MPCs), the latter cells giving rise to bone and therefore representing a target cell type for collagen-related disorders. In addition, allele-specificity, a key factor to the success of RNAi-based suppression, was explored with a view to developing a mutation-independent RNAi-based therapeutic for OI by targeting an intragenic SNP within transcripts derived from the COL1A1 gene. Preferential suppression of individual polymorphic alleles that differed by a single nucleotide was observed.
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页码：864 / 866
页数：2
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