Fibroblast growth factor receptor 3 inhibition by small interfering RNAs in achondroplasia

被引：0
作者：
Guzman-Aranguez, Ana [1 ]
Legeai-Mallet, Laurence [2 ]
Pintor, Jesus [1 ]
机构：
[1] Univ Complutense Madrid, EU Opt, Dept Bioquim & Biol Mol, E-28040 Madrid, Spain
[2] Univ Paris 05, INSERM, U781, Hop Necker Enfants Malad, F-75015 Paris, France
来源：
ANALES DE LA REAL ACADEMIA NACIONAL DE FARMACIA | 2011年 / 77卷 / 01期
关键词：
Achondroplasia; chondrocytes; fibroblast growth factor receptor 3; RNA interference; ERK1/2;
D O I：
暂无
中图分类号：
R9 [药学];
学科分类号：
1007 ;
摘要：
Achondroplasia is a short-limbed dwarfism resulting from mutation and gain-of-function in fibroblast growth factor receptor 3 (FGFR3). Effective therapy for this condition has not as yet been established. We have tested the efficiency of three different small interference RNAs (siRNAs) to abrogate the FGFR3 expression in human immortalized chondrocytes carrying the achondroplasia mutation (G380R). Two siRNA sequences induced markedly decrease of FGFR3 mRNA (up to 75% reduction) and protein levels (up to 61% reduction). Furthemore, siRNA-mediated knockdown of FGFR3 blocked the activation of the downstream signal transduction ERK pathway.
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页码：4 / 11
页数：8
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