This article is by Frank J. Burczynski et al. is published in Recent Patents on Drug Delivery & Formulation, Volume 12, Issue 4, 2018.
With an aging population and rising health care costs, clinicians have to be mindful that sarcopenia and related muscle atrophies are serious impediments to healthy aging and have a significant impact on health care budgets. Strong bones, are associated with healthy muscles, reduce the risk of fractures and hospitalization, especially in the elderly. Musculoskeletal disorders, including muscular dystrophies, cause chronic pain and disability. Mechanisms of musculoskeletal diseases are poorly understood and the efficacy of pharmacological treatment of these diseases is in need of improvement.
Frank J. Burczynski et al. review known mechanisms of muscle injury and repair, highlighting some recent patents and research developments for treatment of skeletal muscle disorders. Patents in this area are of a wide spectrum, ranging from pharmacological (small molecule treatment) to molecular biological procedures (gene editing). A novel small molecule, guaifenesin dinitrate, has shown that it can promote satellite cell activation, reduce tissue inflammation and necrosis, and improve muscle regeneration, muscle function, and exercise endurance. Mesenchymal system cells are reported to promote muscle regeneration following muscle damage. Several patents have targeted dystrophin and DMD; these include methods of gene editing (CRISPR/Cas9, vector-mediated gene delivery) to correct dystrophin gene mutations, as well as use of membrane polaxamers, andrographolides to increase efficacy of stem cell therapy, and methods to induce differentiation of pluripotent stem cells into skeletal muscle cells. Other methods of therapy aim to increase muscle regeneration and reduce degeneration; these include use of tetracyclines, VEGF encoded polynucleotides, lowering chondroitin sulfate levels, ursolic acid, and electrical muscle stimulation.
The reviewers emphasize that a myriad of novel inventions endeavorto prevent skeletal muscle degeneration and promote muscle regeneration. These inventions have the potential to enhance healthy aging secondary to beneficial effects on skeletal muscle, reduce hospitalizations and decrease health care costs.
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