@article{f79621341815440a9a48dcfe775d4f80,
title = "Gene Therapy for Fibrodysplasia Ossificans Progressiva: Feasibility and Obstacles",
abstract = "Fibrodysplasia 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.",
keywords = "Alk2 mutation, Fibrodysplasia ossificans progressiva, Gene therapy, Heterotopic ossification, Rna",
author = "Eekhoff, \{Elisabeth M. W.\} and \{de Ruiter\}, \{Ruben D.\} and Smilde, \{Bernard J.\} and Ton Schoenmaker and \{de Vries\}, \{Teun J.\} and Coen Netelenbos and Hsiao, \{Edward C.\} and Christiaan Scott and Nobuhiko Haga and Zvi Grunwald and \{de Cunto\}, \{Carmen L.\} and \{di Rocco\}, Maja and Delai, \{Patricia L. R.\} and Diecidue, \{Robert J.\} and Vrisha Madhuri and Tae-Joon Cho and Rolf Morhart and Friedman, \{Clive S.\} and Michael Zasloff and Gerard Pals and Jae-Hyuck Shim and Guangping Gao and Frederick Kaplan and Pignolo, \{Robert J.\} and Dimitra Micha",
note = "Funding Information: E.M.W.E.: PI clinical trials of Regeneron Pharmaceuticals, indirect EU-IMI by Astra Zeneca; Clementia Pharmaceuticals (an Ipsen Company). G.G. and J.-H.S.: funding sources are NIAMS of the NIH under R21AR077557, the AAVAA Therapeutics, IFOPA. F.S.K.: research investigator: Clementia/Ipsen, Regeneron. R.J.P.: R.J.P. is an investigator on FOP clinical trials sponsored by Clementia Pharmaceuticals (an Ipsen Company) and Regeneron Pharmaceuticals. R.J.P is a consultant for Ipsen, Regeneron, Incyte, Biocryst, and Keros, but receives no personal compensation for these activities. N.H.: Past Research Investigator: Clementia/Ipsen. Z.G. consultant and speaker for Ipsen BiopharmaceuticalsInc. Regeneron. C.L.D.C.: Clementia/Ipsen PI, Novartis: speaker, Biogen: speaker, Novo Nordisk: speaker. M.D.R. Disclosures last 2 years: Maja Di Rocco received fees for lectures or participation on advisory board by Sanofi, Takeda, Ultragenix, Orchard. Participating in FOP trials. P.D.: PI for Ipsen Move trial. T.-J.C. Funding source: research grant of the Genome Technology to Business Translation Program from the National Research Foundation (NRF) funded by the Ministry of Science, ICT \& Future Planning of the Republic of Korea Government. E.H.: ECH is an investigator on FOP clinical trials sponsored by Clementia Pharmaceuticals (an Ipsen Company). No other conflict of interest related to this article. Publisher Copyright: {\textcopyright} Elisabeth M.W. Eekhoff et al., 2022; Published by Mary Ann Liebert, Inc. 2022.",
year = "2022",
month = aug,
day = "1",
doi = "10.1089/hum.2022.023",
language = "English",
volume = "33",
pages = "782--788",
journal = "Human gene therapy",
issn = "1043-0342",
publisher = "Mary Ann Liebert Inc.",
number = "15-16",
}