Eekhoff, Elisabeth M Wde Ruiter, Ruben DSmilde, Bernard JSchoenmaker, Tonde Vries, Teun JNetelenbos, CoenHsiao, Edward CScott, ChristiaanHaga, NobuhikoGrunwald, ZviDe Cunto, Carmen Ldi Rocco, MajaDelai, Patricia L RDiecidue, Robert JMadhuri, VrishaCho, Tae-JoonMorhart, RolfFriedman, Clive SZasloff, MichaelPals, GerardShim, Jae-HyuckGao, GuangpingKaplan, FrederickPignolo, Robert JMicha, Dimitra2024-01-102024-01-102022-08-16Eekhoff EMW, de Ruiter RD, Smilde BJ, Schoenmaker T, de Vries TJ, Netelenbos C, Hsiao EC, Scott C, Haga N, Grunwald Z, De Cunto CL, di Rocco M, Delai PLR, Diecidue RJ, Madhuri V, Cho TJ, Morhart R, Friedman CS, Zasloff M, Pals G, Shim JH, Gao G, Kaplan F, Pignolo RJ, Micha D. Gene Therapy for Fibrodysplasia Ossificans Progressiva: Feasibility and Obstacles. Hum Gene Ther. 2022 Aug;33(15-16):782-788. doi: 10.1089/hum.2022.023. PMID: 35502479; PMCID: PMC9419966.1557-742210.1089/hum.2022.02335502479https://hdl.handle.net/20.500.14038/52933Fibrodysplasia ossificans progressiva (FOP) is a rare and devastating genetic disease, in which soft connective tissue is converted into heterotopic bone through an endochondral ossification process. 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.enCopyright Elisabeth M.W. Eekhoff et al., 2022; Published by Mary Ann Liebert, Inc. This Open Access article is distributed under the terms of the Creative Commons Attribution Noncommercial License [CC-BY-NC] (http://creativecommons.org/licenses/by-nc/4.0/) which permits any noncommercial use, distribution, and reproduction in any medium, provided the original author(s) and the source are cited.Attribution-NonCommercial 4.0 Internationalhttp://creativecommons.org/licenses/by-nc/4.0/ALK2 mutationRNAfibrodysplasia ossificans progressivagene therapyheterotopic ossificationJournal ArticleHuman gene therapy