{"database":"biostudies-literature","file_versions":[],"scores":null,"additional":{"submitter":["Eekhoff EMW"],"funding":["NIAMS NIH HHS"],"pagination":["782-788"],"full_dataset_link":["https://www.ebi.ac.uk/biostudies/studies/S-EPMC9419966"],"repository":["biostudies-literature"],"omics_type":["Unknown"],"volume":["33(15-16)"],"pubmed_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 therapeu"],"journal":["Human gene therapy"],"pubmed_title":["Gene Therapy for Fibrodysplasia Ossificans Progressiva: Feasibility and Obstacles."],"pmcid":["PMC9419966"],"funding_grant_id":["R21 AR077557","R01 AR078230","R01 AR068983"],"pubmed_authors":["Eekhoff EMW","de Vries TJ","Hsiao EC","Scott C","Haga N","Schoenmaker T","De Cunto CL","Gao G","Delai PLR","Friedman CS","Cho TJ","Diecidue RJ","Micha D","di Rocco M","Zasloff M","de Ruiter RD","Madhuri V","Morhart R","Grunwald Z","Pals G","Smilde BJ","Shim JH","Kaplan F","Netelenbos C","Pignolo RJ"],"additional_accession":[]},"is_claimable":false,"name":"Gene Therapy for Fibrodysplasia Ossificans Progressiva: Feasibility and Obstacles.","description":"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 therapeu","dates":{"release":"2022-01-01T00:00:00Z","publication":"2022 Aug","modification":"2025-04-18T18:43:11.447Z","creation":"2025-02-19T03:28:50.177Z"},"accession":"S-EPMC9419966","cross_references":{"pubmed":["35502479"],"doi":["10.1089/hum.2022.023"]}}