View in EDS PDF Full Text

Statin treatment rescues FGFR3 skeletal dysplasia phenotypes.

Saved in:
Bibliographic Details
Title: Statin treatment rescues FGFR3 skeletal dysplasia phenotypes.
Authors: Yamashita, Akihiro, Morioka, Miho, Kishi, Hiromi, Yahara, Yasuhito, Okada, Minoru, Fujita, Kaori, Kimura, Takeshi, Sawai, Hideaki, Ikegawa, Shiro, Tsumaki, Noriyuki
Source: Nature. 9/25/2014, Vol. 513 Issue 7519, p507-511. 5p. 4 Graphs.
Subjects: Statins (Cardiovascular agents), Skeletal dysplasia, Achondroplasia, Dysplasia, Fibroblast growth factor receptors, Therapeutics
Abstract: 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. [ABSTRACT FROM AUTHOR]
Copyright of Nature is the property of Springer Nature and its content may not be copied or emailed to multiple sites without the copyright holder's express written permission. Additionally, content may not be used with any artificial intelligence tools or machine learning technologies. However, users may print, download, or email articles for individual use. This abstract may be abridged. No warranty is given about the accuracy of the copy. Users should refer to the original published version of the material for the full abstract. (Copyright applies to all Abstracts.)
Database: Psychology and Behavioral Sciences Collection
FullText Links:
  – Type: pdflink
Text:
  Availability: 0
Header DbId: pbh
DbLabel: Psychology and Behavioral Sciences Collection
An: 98526091
AccessLevel: 6
PubType: Academic Journal
PubTypeId: academicJournal
PreciseRelevancyScore: 0
IllustrationInfo
Items – Name: Title
  Label: Title
  Group: Ti
  Data: Statin treatment rescues FGFR3 skeletal dysplasia phenotypes.
– Name: Author
  Label: Authors
  Group: Au
  Data: <searchLink fieldCode="AR" term="%22Yamashita%2C+Akihiro%22">Yamashita, Akihiro</searchLink><br /><searchLink fieldCode="AR" term="%22Morioka%2C+Miho%22">Morioka, Miho</searchLink><br /><searchLink fieldCode="AR" term="%22Kishi%2C+Hiromi%22">Kishi, Hiromi</searchLink><br /><searchLink fieldCode="AR" term="%22Yahara%2C+Yasuhito%22">Yahara, Yasuhito</searchLink><br /><searchLink fieldCode="AR" term="%22Okada%2C+Minoru%22">Okada, Minoru</searchLink><br /><searchLink fieldCode="AR" term="%22Fujita%2C+Kaori%22">Fujita, Kaori</searchLink><br /><searchLink fieldCode="AR" term="%22Kimura%2C+Takeshi%22">Kimura, Takeshi</searchLink><br /><searchLink fieldCode="AR" term="%22Sawai%2C+Hideaki%22">Sawai, Hideaki</searchLink><br /><searchLink fieldCode="AR" term="%22Ikegawa%2C+Shiro%22">Ikegawa, Shiro</searchLink><br /><searchLink fieldCode="AR" term="%22Tsumaki%2C+Noriyuki%22">Tsumaki, Noriyuki</searchLink>
– Name: TitleSource
  Label: Source
  Group: Src
  Data: <searchLink fieldCode="JN" term="%22Nature%22">Nature</searchLink>. 9/25/2014, Vol. 513 Issue 7519, p507-511. 5p. 4 Graphs.
– Name: Subject
  Label: Subjects
  Group: Su
  Data: <searchLink fieldCode="DE" term="%22Statins+%28Cardiovascular+agents%29%22">Statins (Cardiovascular agents)</searchLink><br /><searchLink fieldCode="DE" term="%22Skeletal+dysplasia%22">Skeletal dysplasia</searchLink><br /><searchLink fieldCode="DE" term="%22Achondroplasia%22">Achondroplasia</searchLink><br /><searchLink fieldCode="DE" term="%22Dysplasia%22">Dysplasia</searchLink><br /><searchLink fieldCode="DE" term="%22Fibroblast+growth+factor+receptors%22">Fibroblast growth factor receptors</searchLink><br /><searchLink fieldCode="DE" term="%22Therapeutics%22">Therapeutics</searchLink>
– Name: Abstract
  Label: Abstract
  Group: Ab
  Data: 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. [ABSTRACT FROM AUTHOR]
– Name: AbstractSuppliedCopyright
  Label:
  Group: Ab
  Data: <i>Copyright of Nature is the property of Springer Nature and its content may not be copied or emailed to multiple sites without the copyright holder's express written permission. Additionally, content may not be used with any artificial intelligence tools or machine learning technologies. However, users may print, download, or email articles for individual use. This abstract may be abridged. No warranty is given about the accuracy of the copy. Users should refer to the original published version of the material for the full abstract.</i> (Copyright applies to all Abstracts.)
PLink https://search.ebscohost.com/login.aspx?direct=true&site=eds-live&db=pbh&AN=98526091
RecordInfo BibRecord:
  BibEntity:
    Identifiers:
      – Type: doi
        Value: 10.1038/nature13775
    Languages:
      – Code: eng
        Text: English
    PhysicalDescription:
      Pagination:
        PageCount: 5
        StartPage: 507
    Subjects:
      – SubjectFull: Statins (Cardiovascular agents)
        Type: general
      – SubjectFull: Skeletal dysplasia
        Type: general
      – SubjectFull: Achondroplasia
        Type: general
      – SubjectFull: Dysplasia
        Type: general
      – SubjectFull: Fibroblast growth factor receptors
        Type: general
      – SubjectFull: Therapeutics
        Type: general
    Titles:
      – TitleFull: Statin treatment rescues FGFR3 skeletal dysplasia phenotypes.
        Type: main
  BibRelationships:
    HasContributorRelationships:
      – PersonEntity:
          Name:
            NameFull: Yamashita, Akihiro
      – PersonEntity:
          Name:
            NameFull: Morioka, Miho
      – PersonEntity:
          Name:
            NameFull: Kishi, Hiromi
      – PersonEntity:
          Name:
            NameFull: Yahara, Yasuhito
      – PersonEntity:
          Name:
            NameFull: Okada, Minoru
      – PersonEntity:
          Name:
            NameFull: Fujita, Kaori
      – PersonEntity:
          Name:
            NameFull: Kimura, Takeshi
      – PersonEntity:
          Name:
            NameFull: Sawai, Hideaki
      – PersonEntity:
          Name:
            NameFull: Ikegawa, Shiro
      – PersonEntity:
          Name:
            NameFull: Tsumaki, Noriyuki
    IsPartOfRelationships:
      – BibEntity:
          Dates:
            – D: 25
              M: 09
              Text: 9/25/2014
              Type: published
              Y: 2014
          Identifiers:
            – Type: issn-print
              Value: 00280836
          Numbering:
            – Type: volume
              Value: 513
            – Type: issue
              Value: 7519
          Titles:
            – TitleFull: Nature
              Type: main
ResultId 1