Soluble FGFR-3 as potential therapy for achondroplasia

    French researchers have developed a recombinant protein therapeutic approach, based on a soluble form of human fibroblast growth factor receptor 3 (sFGFR-3), to treat mice with abnormal bone development caused by achondroplasia. This genetic disorder, which is characterized by short stature, is caused by a single point mutation in the gene FGFR3, which leads to prolonged receptor activation upon FGF binding. sFGFR-3 prevents ligand binding to the mutant receptor, thereby preventing excessive intracellular signalling and rescuing the symptoms of achondroplasia.


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      Garcia, S. et al. Postnatal soluble FGFR3 therapy rescues achondroplasia symptoms and restores bone growth in mice. Sci. Transl. Med. 5, 203ra124 (2013)

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    Soluble FGFR-3 as potential therapy for achondroplasia. Nat Rev Endocrinol 9, 692 (2013).

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