An RNA aptamer restores defective bone growth in FGFR3-related skeletal dysplasia in mice

Author:

Kimura Takeshi1ORCID,Bosakova Michaela234ORCID,Nonaka Yosuke5ORCID,Hruba Eva4ORCID,Yasuda Kie1ORCID,Futakawa Satoshi5ORCID,Kubota Takuo1ORCID,Fafilek Bohumil234ORCID,Gregor Tomas23ORCID,Abraham Sara P.2,Gomolkova Regina24ORCID,Belaskova Silvie3ORCID,Pesl Martin236ORCID,Csukasi Fabiana78,Duran Ivan78,Fujiwara Masatoshi5ORCID,Kavkova Michaela9ORCID,Zikmund Tomas9ORCID,Kaiser Josef9ORCID,Buchtova Marcela410ORCID,Krakow Deborah7ORCID,Nakamura Yoshikazu511ORCID,Ozono Keiichi1ORCID,Krejci Pavel234ORCID

Affiliation:

1. Department of Pediatrics, Osaka University Graduate School of Medicine, 565-0871 Osaka, Japan.

2. Department of Biology, Faculty of Medicine, Masaryk University, 62500 Brno, Czech Republic.

3. International Clinical Research Center, St. Anne’s University Hospital, 65691 Brno, Czech Republic.

4. Institute of Animal Physiology and Genetics, Czech Academy of Sciences, 60200 Brno, Czech Republic.

5. RIBOMIC Inc., Tokyo 108-0071, Japan.

6. First Department of Internal Medicine-Cardioangiology, St. Anne’s University Hospital, Masaryk University, 65691 Brno, Czech Republic.

7. Department of Orthopaedic Surgery, University of California Los Angeles, Los Angeles, CA 90095, USA.

8. Networking Research Center on Bioengineering, Biomaterials, and Nanomedicine (CIBER-BBN)-LABRET, University of Málaga, IBIMA-BIONAND, 29071 Málaga, Spain.

9. Central European Institute of Technology, Brno University of Technology, 61200 Brno, Czech Republic.

10. Department of Experimental Biology, Faculty of Science, Masaryk University, 62500 Brno, Czech Republic.

11. Institute of Medical Science, University of Tokyo, Tokyo 108-8639, Japan.

Abstract

We report reversal of the FGFR3-related skeletal dysplasia by RNA aptamer designed to neutralize FGFR3 ligand FGF2.

Funder

National Institute of Arthritis and Musculoskeletal and Skin Diseases

Czech Science Foundation

Ministry of Education, Youth and Sports of the Czech Republic

the Japan Agency for Medical Research and Development

Agency for Healthcare Research of the Czech Republic

Publisher

American Association for the Advancement of Science (AAAS)

Subject

General Medicine

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