Choose an application

• Reference Manager
• EndNote
• RefWorks (Direct export to RefWorks)

Muscular Dystrophy - Research Update and Therapeutic Strategies is for students, researchers, and clinicians interested in muscular dystrophies who want to improve their knowledge of these complex genetic diseases. The book includes information about the genetics of various types of muscular dystrophies as well as explores new and current therapeutic strategies that aim to ameliorate symptoms and improve patients' quality of life and life expectancy. In addition, this book reviews information on current clinical trials for muscular dystrophies and presents a framework for what to consider during the design of these trials.

Muscular dystrophy.

Choose an application

• Reference Manager
• EndNote
• RefWorks (Direct export to RefWorks)

Muscular dystrophy.

Choose an application

• Reference Manager
• EndNote
• RefWorks (Direct export to RefWorks)

Muscular Dystrophy - Research Update and Therapeutic Strategies is for students, researchers, and clinicians interested in muscular dystrophies who want to improve their knowledge of these complex genetic diseases. The book includes information about the genetics of various types of muscular dystrophies as well as explores new and current therapeutic strategies that aim to ameliorate symptoms and improve patients' quality of life and life expectancy. In addition, this book reviews information on current clinical trials for muscular dystrophies and presents a framework for what to consider during the design of these trials.

Muscular dystrophy.

Choose an application

• Reference Manager
• EndNote
• RefWorks (Direct export to RefWorks)

The book provides a comprehensive overview of the different forms of muscular dystrophy (MD), including potential therapeutic interventions and the advantages associated with repurposing pharmaceuticals for treating these conditions. In addition, the book examines the effects of physical training on improving symptoms as well as patient quality of life and life span. The target audience of this book comprises students, researchers, and doctors with an interest in the field of muscular dystrophy.

Muscular dystrophy.

Choose an application

• Reference Manager
• EndNote
• RefWorks (Direct export to RefWorks)

Duchenne muscular dystrophy (DMD) is the most frequently inherited human myopathy affecting in 3500 boys. DMD stems from X-linked defects in the gene encoding for dystrophin, a key scaffold protein of the dystrophin-associated protein complex (DAPC). Disruption of the DAPC leads to high susceptibility to injury with repeated, eccentric contractions as well as inflammation/oxidative stress, resulting in significant damage and necrosis. Chronic damage and repair cycling lead to fibrosis and weakness. Although dystrophin mutation represents the primary cause of DMD, the secondary process involving persistent inflammation and subsequent impaired regeneration are likely to exacerbate disease progression. Adipnectin (ApN) is an adipokine exclusively secreted by adipocytes under normal conditions. This adipokine is also secreted by skeletal muscle under cellular stress. ApN exhibits insulin-sensiziting and anti-inflammatory properties as well as modulatory effects on oxidative stress. The aim of this work was to test the anti-inflammatory properties of ApN on a muscular disease showing severe inflammation, like DMD. To this end, we used the mdx mice, a model of DMD, and transgenic mice overexpressing ApN (Tg ApN). These mice were crossed to generate mdx Tg ApN mice. We tested the hypothesis that evolution of muscular dystrophy could be lessened by ApN. We searched for several markers of inflammation/oxidative stress, muscle differentiation and performed in vivo functional studies in these mice. Compared to WT, muscles of mdx mice showed acute inflammation and oxidative stress, macrophage and T lymphocytes infiltration and severe cellular damages. These mice’s exhibited significant muscle weakness and high susceptibility to fatigue as well. These abnormalities were markedly reduced in mdx Tg ApN mice. ApN seems to be therefore a powerful anti-inflammatory agent that can exhibit beneficial effects on the skeletal muscle. La dystrophie musculaire de Duchenne (DMD) est une myopathie atteignant un garçon sur 3500. La mutation caractéristique de cette maladie touche le gène codant pour la dystrophine. Cette protéine fait partie du « dystrophin-associated protein complex » présent dans le muscle, qui crée un lien entre la matrice extracellulaire et le cytosquelette. Bien que la mutation soit la cause primaire de la DMD, la réponse inflammatoire et le stress oxydatif accompagnant les altérations cellulaires retrouvées dans cette myopathie en exacerbent le développement. L'adiponectine (ApN) est une protéine sécrétée presque exclusivement par le tissu adipeux en conditions normales. Le muscle en est une de ses principales cibles. L'ApN y accélère le captage du glucose, l'oxydation des acides gras et y exercerait des propriétés anti ­ inflammatoires. Nous avons souhaité étudier la puissance des effets anti-inflammatoires de l'ApN dans un modèle d'inflammation musculaire sévère, la souris myopathe mdx qui est déficiente en dystrophine comme le patient atteint de DMD. Différents modèles de souris ont été utilisés : des souris mdx, des souris transgéniques surexprimant l'ApN de façon précoce et chronique (Tg ApN) et des souris issues du croisement de ces deux premiers modèles, les souris mdx transgéniques pour l'ApN (mdx Tg ApN). Nous y avons analysé dans le muscle différents marqueurs du stress oxydatif, de l'inflammation et de la myogenèse ainsi que la résistance à la fatigue, la force des muscles et les altérations cellulaires. Comparé à celui des souris témoins, le muscle des souris mdx présente une inflammation et un stress oxydatif accrus, un infiltrat de macrophages et de lymphocytes important, une altération de l'intégrité du sarcolemme et une résistance moindre à la fatigue. Ces anomalies sont significativement réduites chez les souris mdx Tg ApN. L'ApN se révèle donc être un puissant agent anti-inflammatoire qui exerce un effet bénéfique sur le muscle dystrophique.

Muscular Dystrophy, Duchenne --- Muscular Dystrophy, Duchenne --- Adiponectin