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.
Eekhoff, Elisabeth M W; de Ruiter, Ruben D; Smilde, Bernard J; Schoenmaker, Ton; de Vries, Teun J; Netelenbos, Coen; Hsiao, Edward C; Scott, Christiaan; Haga, Nobuhiko; Grunwald, Zvi; De Cunto, Carmen L; di Rocco, Maja; Delai, Patricia L R; Diecidue, Robert J; Madhuri, Vrisha; Cho, Tae-Joon; Morhart, Rolf; Friedman, Clive S; Zasloff, Michael; Pals, Gerard; Shim, Jae-Hyuck; Gao, Guangping; Kaplan, Frederick; Pignolo, Robert J; and Micha, Dimitra, "Gene Therapy for Fibrodysplasia Ossificans Progressiva: Feasibility and Obstacles" (2022). Department of Anesthesiology Faculty Papers. Paper 84.
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This article is the author's final published version in Human Gene Therapy, Volume 33, Issue 15-16, August 2022, Pages 782 - 788.
The published version is available at https://doi.org/10.1089/hum.2022.023.
Copyright © 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.