Human gene therapy · 2022
Gene Therapy for Fibrodysplasia Ossificans Progressiva: Feasibility and Obstacles.
СтатьяОбзорПоддержка исследования: вне правительства СШАПоддержка NIH: внешняя программаНазвание и аннотация: Перевод в очереди
Обработка статьи · этапы, ошибки и управление
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Ошибки и ограничения
История · было → стало
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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
Аннотация
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.