Gene Therapy for Fibrodysplasia Ossificans Progressiva: Feasibility and Obstacles

Elisabeth M.W. Eekhoff, 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 L.R. Delai, Robert J. Diecidue, Vrisha Madhuri, Tae Joon Cho, Rolf Morhart, Clive S. Friedman, Michael Zasloff, Gerard PalsJae Hyuck Shim, Guangping Gao, Frederick Kaplan, Robert J. Pignolo, Dimitra Micha

Research output: Contribution to journalReview articlepeer-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 (US)
Pages (from-to)782-788
Number of pages7
JournalHuman gene therapy
Volume33
Issue number15-16
DOIs
StatePublished - Aug 1 2022

Keywords

  • ALK2 mutation
  • fibrodysplasia ossificans progressiva
  • gene therapy
  • heterotopic ossification
  • RNA

ASJC Scopus subject areas

  • Molecular Medicine
  • Molecular Biology
  • Genetics

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