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Patients succumb early as they gradually become trapped in a second skeleton of heterotopic bone. Although the underlying genetic defect is long known, the inherent complexity of the disease has hindered the discovery of effective preventions and treatments. New developments in the gene therapy field have motivated its consideration as an attractive therapeutic option for FOP. However, the immune system's role in FOP activation and the as-yet unknown primary causative cell, are crucial issues which must be taken into account in the therapy design. While gene therapy offers a potential therapeutic solution, more knowledge about FOP is needed to enable its optimal and safe application.<\/jats:p>","DOI":"10.1089\/hum.2022.023","type":"journal-article","created":{"date-parts":[[2022,5,3]],"date-time":"2022-05-03T02:25:14Z","timestamp":1651544714000},"page":"782-788","update-policy":"https:\/\/doi.org\/10.1177\/sage-journals-update-policy","source":"Crossref","is-referenced-by-count":13,"title":["Gene Therapy for Fibrodysplasia Ossificans Progressiva: Feasibility and Obstacles"],"prefix":"10.1177","volume":"33","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-5399-676X","authenticated-orcid":false,"given":"Elisabeth M.W.","family":"Eekhoff","sequence":"first","affiliation":[{"name":"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."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ruben D.","family":"de Ruiter","sequence":"additional","affiliation":[{"name":"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."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Bernard J.","family":"Smilde","sequence":"additional","affiliation":[{"name":"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."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ton","family":"Schoenmaker","sequence":"additional","affiliation":[{"name":"Department of Periodontology, Academic Centre for Dentistry Amsterdam (ACTA), University of Amsterdam, Vrije Universiteit, Amsterdam, The Netherlands."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Teun J.","family":"de Vries","sequence":"additional","affiliation":[{"name":"Department of Periodontology, Academic Centre for Dentistry Amsterdam (ACTA), University of Amsterdam, Vrije Universiteit, Amsterdam, The Netherlands."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Coen","family":"Netelenbos","sequence":"additional","affiliation":[{"name":"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."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Edward C.","family":"Hsiao","sequence":"additional","affiliation":[{"name":"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."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Christiaan","family":"Scott","sequence":"additional","affiliation":[{"name":"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."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nobuhiko","family":"Haga","sequence":"additional","affiliation":[{"name":"Rehabilitation Services Bureau, National Rehabilitation Center for Persons with Disabilities, Tokorozawa, Saitama, Japan."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Zvi","family":"Grunwald","sequence":"additional","affiliation":[{"name":"Department of Anesthesiology, Sidney Kimmel Medical College at Thomas Jefferson University, Philadelphia, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Carmen L.","family":"De Cunto","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Section Pediatric Rheumatology, Hospital Italiano Buenos Aires, Argentina."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Maja","family":"di Rocco","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Unit of Rare Diseases, IRCCS Giannina Gaslini Institute, Genoa, Italy."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Patricia L. 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