Research Institute for Aging-Related Diseases, AniMusCure Inc., Suwon 16419, Korea.
Department of Molecular Cell Biology, School of Medicine, Sungkyunkwan University, Suwon 16419, Korea.
Cells. 2022 Jan 21;11(3):364. doi: 10.3390/cells11030364.
Neuromuscular diseases (NMDs) are characterized by progressive loss of muscle mass and strength that leads to impaired body movement. It not only severely diminishes the quality of life of the patients, but also subjects them to increased risk of secondary medical conditions such as fall-induced injuries and various chronic diseases. However, no effective treatment is currently available to prevent or reverse the disease progression. Protein arginine methyltransferases (PRMTs) are emerging as a potential therapeutic target for diverse diseases, such as cancer and cardiovascular diseases. Their expression levels are altered in the patients and molecular mechanisms underlying the association between PRMTs and the diseases are being investigated. PRMTs have been shown to regulate development, homeostasis, and regeneration of both muscle and neurons, and their association to NMDs are emerging as well. Through inhibition of PRMT activities, a few studies have reported suppression of cytotoxic phenotypes observed in NMDs. Here, we review our current understanding of PRMTs' involvement in the pathophysiology of NMDs and potential therapeutic strategies targeting PRMTs to address the unmet medical need.
神经肌肉疾病(NMDs)的特征是肌肉质量和力量逐渐丧失,导致身体运动受损。它不仅严重降低了患者的生活质量,而且使他们面临更高的继发医疗状况风险,如跌倒引起的伤害和各种慢性疾病。然而,目前尚无有效的治疗方法可预防或逆转疾病进展。蛋白质精氨酸甲基转移酶(PRMTs)作为治疗多种疾病(如癌症和心血管疾病)的潜在治疗靶点正在出现。在患者中,它们的表达水平发生改变,并且正在研究 PRMTs 与疾病之间关联的分子机制。已经表明 PRMTs 调节肌肉和神经元的发育、内稳态和再生,它们与 NMDs 的关联也正在出现。通过抑制 PRMT 活性,一些研究报告称抑制了 NMDs 中观察到的细胞毒性表型。在这里,我们回顾了我们对 PRMTs 参与 NMD 病理生理学的理解,以及针对 PRMTs 的潜在治疗策略,以满足未满足的医疗需求。