AdipoRon , a new therapeutic prospect for Duchenne muscular dystrophy
نویسندگان
چکیده
منابع مشابه
Duchenne muscular dystrophy An overview of Duchenne muscular dystrophy
Duchenne muscular dystrophy (DMD) affects approximately 1 in 3,500 live male births [1]. It is caused by a large variety of mutations in the dystrophin gene. Because of these mutations, the body can no longer make dystrophin which is a protein important for stabilisation of the muscle cell during a contraction. Without dystrophin, muscle cells are damaged and slowly replaced by fat and scar tis...
متن کاملP164: Adeno-Associated Viral Vectors in Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (BMD) is an inherited X-link disease. The incidence of this muscle-wasting disease is 1:5000 male live births. Mutation in the gene coding for dystrophin is the main cause of BMD. Most cases of this disease succumb to respiratory and cardiac failure in 3rd to 4th decades. The slow progression of BMD and recent achievement of gene therapies make it as an appropriate c...
متن کاملAdvances in genetic therapeutic strategies for Duchenne muscular dystrophy
NEW FINDINGS What is the topic of this review? This review highlights recent progress in genetically based therapies targeting the primary defect of Duchenne muscular dystrophy. What advances does it highlight? Over the last two decades, considerable progress has been made in understanding the mechanisms underlying Duchenne muscular dystrophy, leading to the development of genetic therapies. Th...
متن کاملLess is more: therapeutic exon skipping for Duchenne muscular dystrophy.
www.thelancet.com/neurology Vol 8 October 2009 873 Duchenne muscular dystrophy (DMD) is a lethal Xlinked progressive muscle-wasting disease caused by premature truncation of the translation of DMD mRNA into dystrophin. Owing to improved respiratory support and treatment with steroids, patients can now survive into early adulthood, which is one or two decades longer than they could survive witho...
متن کاملScreening for Duchenne muscular dystrophy.
Duchenne muscular dystrophy can be diagnosed with confidence before it is clinically apparent, and excluded with certainty where it may have seemed clinically obvious. This has been made possible by the demonstration of a grossly raised level of serum creatine phosphokinase (CPK) (Ebashi et al., 1959; Dreyfus, Schapira, and Demos, 1960). Highest levels (up to 100 times normal) are found early i...
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ژورنال
عنوان ژورنال: Journal of Cachexia, Sarcopenia and Muscle
سال: 2020
ISSN: 2190-5991,2190-6009
DOI: 10.1002/jcsm.12531