期刊
ANNUAL REVIEW OF PHARMACOLOGY AND TOXICOLOGY, VOL 59
卷 59, 期 -, 页码 315-339出版社
ANNUAL REVIEWS
DOI: 10.1146/annurev-pharmtox-010818-021041
关键词
muscle wasting; atrophy; proteostasis; muscular dystrophy; gene therapy; exon skipping
资金
- European Research Council [616830] Funding Source: Medline
- Swiss National Science Foundation [156654] Funding Source: Medline
Adequate skeletal muscle plasticity is an essential element for our well-being, and compromised muscle function can drastically affect quality of life, morbidity, and mortality. Surprisingly, however, skeletal muscle remains one of the most under-medicated organs. Interventions in muscle diseases are scarce, not only in neuromuscular dystrophies, but also in highly prevalent secondary wasting pathologies such as sarcopenia and cachexia. Even in other diseases that exhibit a well-established risk correlation of muscle dysfunction due to a sedentary lifestyle, such as type 2 diabetes or cardiovascular pathologies, current treatments are mostly targeted on non-muscle tissues. In recent years, a renewed focus on skeletal muscle has led to the discovery of various novel drug targets and the design of new pharmacological approaches. This review provides an overview of the current knowledge of the key mechanisms involved in muscle wasting conditions and novel pharmacological avenues that could ameliorate muscle diseases.
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