RNAi of COL1A1 in mesenchymal progenitor cells
Citation:
Millington-Ward SM, McMahon HP, Allen D, Tuohy G, Kiang A-S, Palfi A, Kenna PF, Humphries P and GJ Farrar `RNAi of COL1A1 in mesenchymal progenitor cells? in European Journal of Human Genetics, 12, (10), 2004, pp 864 - 866Download Item:
RNAi of COL1A1 in mesenchymal progenitor cells.pdf (published (publisher copy) peer-reviewed) 68.19Kb
Abstract:
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.
Sponsor
Grant Number
Enterprise Ireland
Health Research Board
Author's Homepage:
http://people.tcd.ie/phumphrsDescription:
PUBLISHED
Author: HUMPHRIES, PETER
Publisher:
Nature Publishing GroupType of material:
Journal ArticleCollections:
Series/Report no:
European Journal of Human Genetics12
10
Availability:
Full text availableLicences: