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FTY720治疗可减轻严重的2C型小鼠肢带型肌营养不良症的病理变化。

Severe murine limb-girdle muscular dystrophy type 2C pathology is diminished by FTY720 treatment.

作者信息

Heydemann Ahlke

机构信息

Department of Physiology and Biophysics, University of Illinois at Chicago, 835 South Wolcott Avenue, COMRB 2035, MC 901, Chicago, Illinois, 60612, USA.

The Center for Cardiovascular Research, University of Illinois at Chicago, Chicago, Illinois, USA.

出版信息

Muscle Nerve. 2017 Sep;56(3):486-494. doi: 10.1002/mus.25503. Epub 2017 Mar 26.

DOI:10.1002/mus.25503
PMID:27935071
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC5511098/
Abstract

INTRODUCTION

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.

METHODS

The treatment protocol was initiated at age 3 weeks and was continued with alternate-day injections for 3 weeks.

RESULTS

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.

CONCLUSION

This study showed that FTY720 is an effective muscular dystrophy treatment when therapy is initiated early in the disease progression. Muscle Nerve 56: 486-494, 2017.

摘要

引言

2C型肢带型肌营养不良症（LGMD-2C）由γ-肌聚糖基因突变引起，是一种严重的、进行性且完全致命的人类肌肉萎缩疾病，目前尚无有效治疗方法。本研究检测了鞘氨醇-1-磷酸受体调节剂FTY720治疗LGMD-2C严重小鼠模型Sgcg DBA2/J的疗效。预计FTY720治疗可通过减轻慢性炎症和纤维化在两个关键环节靶向LGMD-2C疾病进展。

方法

治疗方案于3周龄时开始，隔日注射，持续3周。

结果

该治疗通过体积描记法产生了显著的功能益处，并显著降低了膜通透性和纤维化。此外，该方案提高了肌营养不良蛋白-糖蛋白家族成员δ-肌聚糖的蛋白水平。

结论

本研究表明，在疾病进展早期开始治疗时，FTY720是一种有效的肌营养不良症治疗药物。《肌肉与神经》56: 486 - 494, 2017年。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/23fb/5511098/446421a89179/nihms876835f7.jpg
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https://cdn.ncbi.nlm.nih.gov/pmc/blobs/23fb/5511098/446421a89179/nihms876835f7.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/23fb/5511098/236f3175b3aa/nihms876835f1.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/23fb/5511098/7ec52fab11de/nihms876835f2.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/23fb/5511098/63195b706354/nihms876835f3.jpg
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https://cdn.ncbi.nlm.nih.gov/pmc/blobs/23fb/5511098/8418d040f84b/nihms876835f5.jpg
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