Severe murine limb-girdle muscular dystrophy type 2C pathology is diminished by FTY720 treatment.
Severe murine limb-girdle muscular dystrophy type 2C pathology is diminished by FTY720 treatment.
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DOI：
10.1002/mus.25503
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发表时间：
2017-09
期刊：
影响因子：
3.4
通讯作者：
Heydemann A
中科院分区：
文献类型：
--
作者：
Heydemann A
Limb-girdle muscular dystrophy type 2C (LGMD-2C) is caused by mutations in γ-sarcoglycan and is a devastating, progressive, and fully lethal human muscle-wasting disease that has no effective treatment. This study examined the efficacy of the sphingosine-1-phosphate receptor modulator FTY720 in treating Sgcg−/− DBA2/J, a severe mouse model of LGMD-2C. FTY720 treatment was expected to target LGMD-2C disease progression at 2 key positions by reducing chronic inflammation and fibrosis. The treatment protocol was initiated at age 3 weeks and was continued with alternate-day injections for 3 weeks. The treatment produced significant functional benefit by plethysmography and significant reductions of membrane permeability and fibrosis. Furthermore, the protocol elevated protein levels of δ-sarcoglycan, a dystrophin–glycoprotein family member. This study showed that FTY720 is an effective muscular dystrophy treatment when therapy is initiated early in the disease progression.
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影响因子：
3.7
作者：
Loh KC;Leong WI;Carlson ME;Oskouian B;Kumar A;Fyrst H;Zhang M;Proia RL;Hoffman EP;Saba JD
通讯作者：
Saba JD
影响因子：
37.8
作者：
Liu W;Zi M;Naumann R;Ulm S;Jin J;Taglieri DM;Prehar S;Gui J;Tsui H;Xiao RP;Neyses L;Solaro RJ;Ke Y;Cartwright EJ;Lei M;Wang X
通讯作者：
Wang X
DOI：
10.1083/jcb.142.5.1279
发表时间：
1998-09-07
期刊：
The Journal of cell biology
影响因子：
--
作者：
Hack AA;Ly CT;Jiang F;Clendenin CJ;Sigrist KS;Wollmann RL;McNally EM
通讯作者：
McNally EM
影响因子：
4.8
作者：
Dudek, S. M.;Camp, S. M.;Singleton, P. A.
通讯作者：
Singleton, P. A.
影响因子：
15.9
作者：
Heydemann, Ahlke;Ceco, Ermelinda;McNally, Elizabeth M.
通讯作者：
McNally, Elizabeth M.