Gene therapy for Fibrodysplasia Ossificans Progressiva (FOP): feasibility and obstacles

Ruben D de Ruiter, Bernard J Smilde, Ton Schoenmaker, Teun J de Vries, Coen Netelenbos, Edward C Hsiao, Christiaan Scott, Nobuhiko Haga, Zvi Grunwald, Carmen L De Cunto, Maja Di Rocco, Patricia Delai, Robert Diecidue, Vrisha Madhuri, Tae-Joon Cho, Rolf Morhart, Clive Friedman, Michael Zasloff, Gerard Pals, Jae-Hyuck ShimGuangping Gao, Frederick Kaplan, Robert J Pignolo, Dimitra Micha

Research output: Contribution to journalArticleAcademicpeer-review

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.

Original languageEnglish
JournalHuman Gene Therapy
DOIs
Publication statusE-pub ahead of print - 2 May 2022

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