RNAi of COL1A1 in mesenchymal progenitor cells

European Journal of Human Genetics : EJHG
Sophia Millington-WardG J Farrar

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.

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Citations

Mar 5, 2008·Cell and Tissue Research·Noriko KotobukiHajime Ohgushi
Nov 23, 2006·Current Osteoporosis Reports·Frank Rauch, Francis H Glorieux
Jun 14, 2005·Trends in Molecular Medicine·Sophia Millington-WardG Jane Farrar
Jul 16, 2005·Molecular Therapy : the Journal of the American Society of Gene Therapy·Edgardo Rodriguez-LebronRonald J Mandel
Dec 11, 2008·Molecular Therapy : the Journal of the American Society of Gene Therapy·Christopher H EvansPaul D Robbins
Nov 1, 2011·FASEB Journal : Official Publication of the Federation of American Societies for Experimental Biology·Gerd A MüllerWilfried Briest
Jul 3, 2007·PLoS Genetics·Matthew WoodGraham McClorey
Feb 1, 2009·International Journal of Clinical Rheumatology·Christopher Niyibizi, Feng Li
Nov 19, 2008·International Journal of Medical Sciences·Katarina LindahlOsten Ljunggren
Nov 6, 2012·Clinical and Experimental Medicine·Yi LiuJinxiang Han
Apr 25, 2014·European Journal of Endocrinology·Katarina LindahlAndreas Kindmark
Apr 7, 2012·Advanced Drug Delivery Reviews·C H Evans
Dec 7, 2007·Journal of Dermatological Science·Danny AllenG Jane Farrar
Jul 14, 2012·American Journal of Medical Genetics. Part C, Seminars in Medical Genetics·Marianne Rohrbach, Cecilia Giunta
Oct 9, 2007·Vision Research·Mary O'ReillyG Jane Farrar
May 28, 2013·The International Journal of Biochemistry & Cell Biology·William V Arnold, Andrzej Fertala
Jun 22, 2005·Molecular Therapy : the Journal of the American Society of Gene Therapy·Anna-Sophia KiangG Jane Farrar
Jun 20, 2007·Experimental Dermatology·Qiong WangZhengxiao Li
Nov 10, 2005·American Journal of Medical Genetics. Part C, Seminars in Medical Genetics·Frank Rauch, Francis H Glorieux
Aug 23, 2005·The Journal of the American Academy of Orthopaedic Surgeons·Christopher H EvansPaul D Robbins
Jun 18, 2010·Journal of Cell Science·Kwok Yeung TsangKathryn S E Cheah
Jun 23, 2021·Proceedings of the National Academy of Sciences of the United States of America·Ciro Maurizio Amato, Humphrey Hung-Chang Yao

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