Gene Therapy for Fibrodysplasia Ossificans Progressiva: Feasibility and Obstacles.
Gene Therapy for Fibrodysplasia Ossificans Progressiva: Feasibility and Obstacles.
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DOI：
10.1089/hum.2022.023
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发表时间：
2022-08
影响因子：
4.2
通讯作者：
Micha, Dimitra
中科院分区：
文献类型：
--
作者：
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.;Micha, Dimitra
进行性纤维发育不良骨化症(FOP)是一种罕见的、毁灭性的遗传性疾病，其特征是软结缔组织通过软骨内骨化过程转化为异位骨。随着患者逐渐被困在异位骨的第二个骨骼中，他们很早就屈服了。虽然潜在的遗传缺陷早已为人所知，但这种疾病固有的复杂性阻碍了有效预防和治疗的发现。基因治疗领域的新发展促使人们考虑将其作为FOP的一种有吸引力的治疗选择。然而，免疫系统在FOP激活中的作用以及迄今未知的主要致病细胞，是治疗设计中必须考虑的关键问题。虽然基因治疗提供了一种潜在的治疗方案，但需要更多关于FOP的知识才能使其获得最佳和安全的应用。
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.
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影响因子：
5.9
作者：
Hino K;Zhao C;Horigome K;Nishio M;Okanishi Y;Nagata S;Komura S;Yamada Y;Toguchida J;Ohta A;Ikeya M
通讯作者：
Ikeya M
影响因子：
--
作者：
Gruntman, Alisha M;Bish, Lawrence T;Mueller, Christian;Sweeney, H Lee;Flotte, Terence R;Gao, Guangping
通讯作者：
Gao, Guangping
DOI：
10.1002/jbmr.2820
发表时间：
2016-09
期刊：
Journal of bone and mineral research : the official journal of the American Society for Bone and Mineral Research
影响因子：
--
作者：
Chakkalakal SA;Uchibe K;Convente MR;Zhang D;Economides AN;Kaplan FS;Pacifici M;Iwamoto M;Shore EM
通讯作者：
Shore EM
影响因子：
3.7
作者：
Baujat G;Choquet R;Bouée S;Jeanbat V;Courouve L;Ruel A;Michot C;Le Quan Sang KH;Lapidus D;Messiaen C;Landais P;Cormier-Daire V
通讯作者：
Cormier-Daire V
影响因子：
5.2
作者：
de Ruiter RD;Smilde BJ;Pals G;Bravenboer N;Knaus P;Schoenmaker T;Botman E;Sánchez-Duffhues G;Pacifici M;Pignolo RJ;Shore EM;van Egmond M;Van Oosterwyck H;Kaplan FS;Hsiao EC;Yu PB;Bocciardi R;De Cunto CL;Longo Ribeiro Delai P;de Vries TJ;Hilderbrandt S;Jaspers RT;Keen R;Koolwijk P;Morhart R;Netelenbos JC;Rustemeyer T;Scott C;Stockklausner C;Ten Dijke P;Triffit J;Ventura F;Ravazzolo R;Micha D;Eekhoff EMW
通讯作者：
Eekhoff EMW