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Gene Therapy for Fibrodysplasia Ossificans Progressiva: Feasibility and Obstacles.
Eekhoff EMW, de Ruiter RD, Smilde BJ, Schoenmaker T, de Vries TJ, Netelenbos C, Hsiao EC, Scott C, Haga N, Grunwald Z, De Cunto CL, di Rocco M, Delai PLR, Diecidue RJ, Madhuri V, Cho TJ, Morhart R, Friedman CS, Zasloff M, Pals G, Shim JH, Gao G, Kaplan F, Pignolo RJ, Micha D. Eekhoff EMW, et al. Among authors: haga n. Hum Gene Ther. 2022 Aug;33(15-16):782-788. doi: 10.1089/hum.2022.023. Hum Gene Ther. 2022. PMID: 35502479 Free PMC article. Review.
A novel mutation of ALK2, L196P, found in the most benign case of fibrodysplasia ossificans progressiva activates BMP-specific intracellular signaling equivalent to a typical mutation, R206H.
Ohte S, Shin M, Sasanuma H, Yoneyama K, Akita M, Ikebuchi K, Jimi E, Maruki Y, Matsuoka M, Namba A, Tomoda H, Okazaki Y, Ohtake A, Oda H, Owan I, Yoda T, Furuya H, Kamizono J, Kitoh H, Nakashima Y, Susami T, Haga N, Komori T, Katagiri T. Ohte S, et al. Among authors: haga n. Biochem Biophys Res Commun. 2011 Apr 1;407(1):213-8. doi: 10.1016/j.bbrc.2011.03.001. Epub 2011 Mar 4. Biochem Biophys Res Commun. 2011. PMID: 21377447
International physician survey on management of FOP: a modified Delphi study.
Di Rocco M, Baujat G, Bertamino M, Brown M, De Cunto CL, Delai PLR, Eekhoff EMW, Haga N, Hsiao E, Keen R, Morhart R, Pignolo RJ, Kaplan FS. Di Rocco M, et al. Among authors: haga n. Orphanet J Rare Dis. 2017 Jun 12;12(1):110. doi: 10.1186/s13023-017-0659-4. Orphanet J Rare Dis. 2017. PMID: 28606101 Free PMC article.
The FOP Connection Registry: Design of an international patient-sponsored registry for Fibrodysplasia Ossificans Progressiva.
Mantick N, Bachman E, Baujat G, Brown M, Collins O, De Cunto C, Delai P, Eekhoff M, Zum Felde R, Grogan DR, Haga N, Hsiao E, Kantanie S, Kaplan F, Keen R, Milosevic J, Morhart R, Pignolo R, Qian X, di Rocco M, Scott C, Sherman A, Wallace M, Williams N, Zhang K, Bogard B. Mantick N, et al. Among authors: haga n. Bone. 2018 Apr;109:285-290. doi: 10.1016/j.bone.2017.08.032. Epub 2017 Sep 1. Bone. 2018. PMID: 28866367
461 results