Abstract
Muscular dystrophy and dilated cardiomyopathy are intractable diseases and their treatment options are very limited. Transient receptor potential cation channel subfamily V, member 2 (TRPV2), is a stretch-sensitive Ca2+-permeable channel that causes sustained intracellular Ca2+ increase in muscular cells, which is a pathophysiological feature of degenerative muscular disease. Recent reports have clarified that TRPV2 is concentrated and activated in the sarcolemma of cardiomyocytes/myocytes during cardiomyopathy/heart failure and muscular dystrophy. Furthermore, these reports showed that inactivation of TRPV2 ameliorates muscle dysgenesis to improve cardiac function and survival prognosis. Although TRPV2 is a potential therapeutic target for cardiomyopathy, there were no TRPV2 inhibitors available until recently. In this review, we introduce our recent findings and discuss the current progress in the development of TRPV2 inhibitors and their therapeutic applications for cardiomyopathy associated with muscular dystrophy.
Highlights
Muscular dystrophy (MD) and dilated cardiomyopathy (DCM) are intractable diseases and their treatment options are currently very limited
To confirm that TRPV2 is the therapeutic target for cardiomyopathy/heart failure and MD, it is important to determine whether heart failure and muscular dysfunction could be ameliorated by the specific inhibition of TRPV2 activity
To determine how to suppress the accumulation of TRPV2 in the plasma membrane, we found that a part of the amino terminal (NT) domain of TRPV2 protein plays a role in retaining TRPV2 on that a part of the amino terminal (NT) domain of TRPV2 protein plays a role in retaining TRPV2 on the the cell surface
Summary
Muscular dystrophy (MD) and dilated cardiomyopathy (DCM) are intractable diseases and their treatment options are currently very limited. Many muscle diseases are caused by genetic abnormalities in proteins which form complexes with dystrophin (e.g., dystrophin–glycoprotein complexes [1]), and it has been reported that abnormalities in the same responsible genes as those of MD cause DCM [2]. Steroid treatment is a standard treatment for DMD, for other MDs there are few established treatments (treatments based on the evidence) as indications for medical insurance, and only some new drugs at the development stage. In these cases, drugs such as angiotensin-converting enzyme (ACE) inhibitors, angiotensin II receptor blockers (ARBs), beta-blockers, and aldosterone antagonists are prescribed as a standard therapy to treat heart failure in these patients.
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