Re-examination of therapeutic management of muscular dystrophies using a vascular smooth muscle-centered approach

  • Preethy Senthilkumar
    Fujio-Eiji Academic Terrain (FEAT), Nichi-In Centre for Regenerative Medicine (NCRM), B-34, LICET, Loyola College, Nungambakkam, Chennai 600034, India
  • Yamamoto Naoki
    Genome Medical Sciences Project, National Center for Global Health and Medicine (NCGM), 1 Chome-7-1 Kounodai, Ichikawa-shi, Chiba 272-8516, Japan
  • Ozasa Shiro
    Department of Pediatrics, Kumamoto University Hospital, 1 Chome-1-1 Honjo, Chuo-ku, Kumamoto, 860-8556, Japan
  • Raghavan Kadalraja
    Department of Paediatric Neurology, Jesuit Antonyraj Memorial Inter-disciplinary Centre for Advanced Recovery and Education (JAICARE), Mandela Nagar, Madurai, Tamil Nadu 625022, India Department of Paediatric Neurology, Sarvee Integra Private Limited, 61 Bhimasena Garden Street, Mylapore, Chennai 600004, India
  • Dedeepiya Vidyasagar Devaprasad
    Mary-Yoshio Translational Hexagon (MYTH), Nichi-In Centre for Regenerative Medicine (NCRM), C-30 LICET, Loyola College, Nungambakkam, Chennai 600034, Chennai, India
  • Iwasaki Masaru
    Centre for Advancing Clinical Research (CACR), School of Medicine, University of Yamanashi, 1110 Shimokato, Chuo, Yamanashi 409-3898, Japan
  • Abraham Samuel JK
    Mary-Yoshio Translational Hexagon (MYTH), Nichi-In Centre for Regenerative Medicine (NCRM), C-30 LICET, Loyola College, Nungambakkam, Chennai 600034, Chennai, India Centre for Advancing Clinical Research (CACR), School of Medicine, University of Yamanashi, 1110 Shimokato, Chuo, Yamanashi 409-3898, Japan Antony-Xavier Interdisciplinary Scholastics (AXIS), GN Corporation Co. Ltd., 3-8 Wakamatsu, Kofu, Yamanashi 400-0866, Japan R & D, Sophy Inc., 248 Tamura, Niyodogawa, Agawa, Kochi 781-1522, Japan Levy-Jurgen Transdisciplinary Exploratory (LJTE), Global Niche Corp, Wilmington, DE 19805, USA

抄録

<p>In contrast to the long-standing focus on the pathophysiology of skeletal muscles in the hunt for a cure for Duchenne muscular dystrophy (DMD), we opine that the malfunctioning of dystrophin produced by vascular smooth muscle is a major contributor to the pathology of the illness. We believe that a biological response modifier glucan (BRMG), which has been shown in clinical studies of DMD to boost the expression of vascular smooth muscle dystrophin and provide anti-fibrotic and anti-inflammatory effects, may play a key role in reducing the pathogenesis of DMD. According to the evaluation of biomarkers, this BRMG, which is safe and side-effect-free, reduces the pathogenesis of DMD. We describe the possible mechanisms of action by which this BRMG helps in alleviating the symptoms of DMD by targeting smooth muscle dystrophin, in addition to its advantages over other therapeutic modalities, as well as how it can serve as a valuable adjunct to existing therapies. We suggest that using BRMG adjuncts that target smooth muscle dystrophin would be a potential therapeutic approach that prolongs the lifespan and extends the duration of ambulation from the onset of DMD. Further studies are needed to validate this hypothesis.</p>

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