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In vitro and in vivo characterization of Recifercept, a soluble fibroblast growth factor receptor 3, as treatment for achondroplasia
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In vitro and in vivo characterization of Recifercept, a soluble fibroblast growth factor receptor 3, as treatment for achondroplasia
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In vitro and in vivo characterization of Recifercept, a soluble fibroblast growth factor receptor 3, as treatment for achondroplasia
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Journal Article
In vitro and in vivo characterization of Recifercept, a soluble fibroblast growth factor receptor 3, as treatment for achondroplasia
2020
Overview
Achondroplasia is a rare genetic disorder caused by mutations in the Fibroblast Growth Factor receptor 3 (FGFR3). These mutations lead to aberrant increase of inhibitory signaling in proliferating chondrocytes at the growth plate. Recifercept is a potential treatment for this disease using a decoy approach to sequester FGFR3 ligands subsequently normalizing activation of the mutated FGFR3 receptor. Recifercept binds to FGF isoforms in vitro and in cellular model systems and reduces FGFR3 signaling. In addition, in a transgenic mouse model of achondroplasia, Recifercept restores reduced body weight and long bone growth in these mice. These data suggest that Recifercept treatment could lead to clinical benefits in children treated with this molecule.
Publisher
Public Library of Science,Public Library of Science (PLoS)
Subject
/ Achondroplasia - drug therapy
/ Animals
/ Bone Development - drug effects
/ Cell Differentiation - drug effects
/ Cell Proliferation - drug effects
/ Female
/ Fibroblast growth factor receptors
/ Fibroblast Growth Factors - metabolism
/ Humans
/ Male
/ Medicine and Health Sciences
/ Mice
/ Mutation
/ Protein Binding - drug effects
/ Receptor, Fibroblast Growth Factor, Type 3 - administration & dosage
/ Receptor, Fibroblast Growth Factor, Type 3 - genetics
/ Receptor, Fibroblast Growth Factor, Type 3 - metabolism
/ Receptor, Fibroblast Growth Factor, Type 3 - pharmacology
