Gene Therapy for Fibrodysplasia Ossificans Progressiva: Feasibility and Obstacles

Author:

Eekhoff Elisabeth M.W.1ORCID,de Ruiter Ruben D.1,Smilde Bernard J.1,Schoenmaker Ton2,de Vries Teun J.2,Netelenbos Coen1,Hsiao Edward C.3,Scott Christiaan4,Haga Nobuhiko5,Grunwald Zvi6,De Cunto Carmen L.7,di Rocco Maja8,Delai Patricia L. R.9,Diecidue Robert J.10,Madhuri Vrisha11,Cho Tae-Joon12,Morhart Rolf13,Friedman Clive S.14,Zasloff Michael15,Pals Gerard16,Shim Jae-Hyuck17ORCID,Gao Guangping18,Kaplan Frederick19,Pignolo Robert J.20,Micha Dimitra16

Affiliation:

1. Section Endocrinology, Department of Internal Medicine, Amsterdam University Medical Centers, Vrije Universiteit Amsterdam, Amsterdam Rare Bone Disease Center, Amsterdam Bone Center, Amsterdam Movement Sciences, Amsterdam, The Netherlands.

2. Department of Periodontology, Academic Centre for Dentistry Amsterdam (ACTA), University of Amsterdam, Vrije Universiteit, Amsterdam, The Netherlands.

3. Division of Endocrinology and Metabolism, Department of Medicine, Institute for Human Genetics, Program in Craniofacial Biology, the Institute for Regeneration Medicine, University of California, San Francisco, California, USA.

4. Division of Paediatric Rheumatology, Department of Paediatrics and Child Heath, Red Cross War Memorial Children's Hospital, University of Cape Town, Cape Town, South Africa.

5. Rehabilitation Services Bureau, National Rehabilitation Center for Persons with Disabilities, Tokorozawa, Saitama, Japan.

6. Department of Anesthesiology, Sidney Kimmel Medical College at Thomas Jefferson University, Philadelphia, USA.

7. Department of Pediatrics, Section Pediatric Rheumatology, Hospital Italiano Buenos Aires, Argentina.

8. Department of Pediatrics, Unit of Rare Diseases, IRCCS Giannina Gaslini Institute, Genoa, Italy.

9. Teaching and Research Institute of the Hospital Israelita Albert Einstein, Sao Paulo, Brazil.

10. Department of Oral and Maxillofacial Surgery, Sidney Kimmel Medical College, Philadelphia, USA.

11. Department of Paediatric Orthopaedics and Center for Stem Cell Research, Christian Medical College and Hospital, Vellore, India.

12. Department of Orthopaedic Surgery, Seoul National University College of Medicine, Seoul, Republic of Korea.

13. Department of Pediatrics, Garmisch-Partenkichen Medical Center, Garmisch-Partenkirchen, Germany.

14. Schulich School of Medicine and Dentistry, Western University, Clinical Skills Building, London, Ontario, Canada.

15. Surgery and Pediatrics, MedStar Georgetown Transplant Institute, Washington, District of Columbia, USA.

16. Department of Human Genetics, Amsterdam University Medical Centers, Vrije Universiteit Amsterdam, Amsterdam Rare Bone Disease Center, Amsterdam Bone Center, Amsterdam Movement Sciences, Amsterdam, The Netherlands.

17. Department of Medicine/Rheumatology, Horae Gene Therapy Center, Li Weibo Institute for Rare Diseases Research, UMass Chan Medical School, Worcester, Massachusetts, USA.

18. Department of Microbiology and Physiological Systems, Horae Gene Therapy Center, Viral Vector Core, Li Weibo Institute for Rare Diseases Research, UMass Chan Medical School, Worcester, Massachusetts, USA.

19. Department of Orthopaedic Surgery and Medicine, Center for Research in FOP and Related Disorders, The Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania, USA.

20. Department of Medicine, Mayo Clinic, Rochester, Minnesota, USA.

Publisher

Mary Ann Liebert Inc

Subject

Genetics,Molecular Biology,Molecular Medicine

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