Document Detail

Statin treatment rescues FGFR3 skeletal dysplasia phenotypes.
MedLine Citation:
PMID:  25231866     Owner:  NLM     Status:  Publisher    
Gain-of-function mutations in the fibroblast growth factor receptor 3 gene (FGFR3) result in skeletal dysplasias, such as thanatophoric dysplasia and achondroplasia (ACH). The lack of disease models using human cells has hampered the identification of a clinically effective treatment for these diseases. Here we show that statin treatment can rescue patient-specific induced pluripotent stem cell (iPSC) models and a mouse model of FGFR3 skeletal dysplasia. 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.
Akihiro Yamashita; Miho Morioka; Hiromi Kishi; Takeshi Kimura; Yasuhito Yahara; Minoru Okada; Kaori Fujita; Hideaki Sawai; Shiro Ikegawa; Noriyuki Tsumaki
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Publication Detail:
Type:  JOURNAL ARTICLE     Date:  2014-9-17
Journal Detail:
Title:  Nature     Volume:  -     ISSN:  1476-4687     ISO Abbreviation:  Nature     Publication Date:  2014 Sep 
Date Detail:
Created Date:  2014-9-18     Completed Date:  -     Revised Date:  -    
Medline Journal Info:
Nlm Unique ID:  0410462     Medline TA:  Nature     Country:  -    
Other Details:
Languages:  ENG     Pagination:  -     Citation Subset:  -    
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