The method of our time is to use not one but multiple exploration models.

Marshall McLuhan





Statin treatment rescues FGFR3 skeletal dysplasia phenotypes (Yamashita A et al., 2014) (pdf)

We converted fibroblasts from thanatophoric dysplasia type I (TD1) and ACH patients into iPSCs. The chondrogenic differentiation of TD1 iPSCs and ACH iPSCs resulted in the formation of degraded cartilage. We found that statins could correct the degraded cartilage in both chondrogenically differentiated TD1 and ACH iPSCs. Treatment of ACH model mice with statin led to a significant recovery of bone growth. These results suggest that statins could represent a medical treatment for infants and children with TD1 and ACH.

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Estatinas Nature 2014