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调节瘦体重的药理学

Pharmacology of manipulating lean body mass.

作者信息

Sepulveda Patricio V, Bush Ernest D, Baar Keith

机构信息

Department of Physiology, Monash University, Melbourne, Vic., Australia.

出版信息

Clin Exp Pharmacol Physiol. 2015 Jan;42(1):1-13. doi: 10.1111/1440-1681.12320.

DOI:10.1111/1440-1681.12320
PMID:25311629
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC4383600/
Abstract

Dysfunction and wasting of skeletal muscle as a consequence of illness decreases the length and quality of life. Currently, there are few, if any, effective treatments available to address these conditions. Hence, the existence of this unmet medical need has fuelled large scientific efforts. Fortunately, these efforts have shown many of the underlying mechanisms adversely affecting skeletal muscle health. With increased understanding have come breakthrough disease-specific and broad spectrum interventions, some progressing through clinical development. The present review focuses its attention on the role of the antagonistic process regulating skeletal muscle mass before branching into prospective promising therapeutic targets and interventions. Special attention is given to therapies in development against cancer cachexia and Duchenne muscular dystrophy before closing remarks on design and conceptualization of future therapies are presented to the reader.

摘要

疾病导致的骨骼肌功能障碍和萎缩会降低生活的长度和质量。目前,针对这些病症的有效治疗方法即便有也很少。因此,这种未满足的医疗需求推动了大量的科学研究。幸运的是,这些研究揭示了许多对骨骼肌健康产生不利影响的潜在机制。随着认识的加深,出现了针对特定疾病和广谱的突破性干预措施,其中一些已进入临床开发阶段。本综述首先关注调节骨骼肌质量的拮抗过程的作用,然后再探讨潜在的有前景的治疗靶点和干预措施。在向读者介绍未来治疗方法的设计和概念之前,特别关注了针对癌症恶病质和杜氏肌营养不良症的正在研发的疗法。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/1b42/4383600/e1e88a5ea9bd/nihms671877f2.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/1b42/4383600/9d9c138ecf26/nihms671877f1.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/1b42/4383600/e1e88a5ea9bd/nihms671877f2.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/1b42/4383600/9d9c138ecf26/nihms671877f1.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/1b42/4383600/e1e88a5ea9bd/nihms671877f2.jpg

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本文引用的文献

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Prevention of muscular dystrophy in mice by CRISPR/Cas9-mediated editing of germline DNA.利用 CRISPR/Cas9 介导的种系 DNA 编辑预防小鼠肌肉萎缩症。
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Current Challenges and Future Directions in Recombinant AAV-Mediated Gene Therapy of Duchenne Muscular Dystrophy.当前 Duchenne 肌营养不良症的重组 AAV 介导基因治疗的挑战和未来方向。
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