Duchenne muscular dystrophy: CRISPR/Cas9 treatment
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 کاملIntegrated Treatment Regime for Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is the most common hereditary neuromuscular disease affecting all races and ethnic groups, with a sex linked inheritance pattern of 1 in 3500 male births. The underlying genetic defect results in the loss of a structural protein called dystrophin that normally serves to provide mechanical stability during muscle contraction. As a result of this loss, there is e...
متن کاملEarly prednisone treatment in Duchenne muscular dystrophy.
The purpose of this long-term, open parallel-group, double-consent study of alternate-day, low-dose prednisone in 2-4-year-old patients with Duchenne muscular dystrophy (DMD) was to determine whether prednisone produces a beneficial effect when given earlier than usual. Muscle function was evaluated by timed tests, and muscle strength with a hand-held myometer. After 55 months of treatment, the...
متن کاملDuchenne muscular dystrophy.
Progress in understanding the role of dystrophin raises promising hopes for a treatment for Duchenne muscular dystrophy. In addition, great improvements have been made in the ability to diagnose this disease using simple molecular methods.
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ژورنال
عنوان ژورنال: Cell Research
سال: 2016
ISSN: 1001-0602,1748-7838
DOI: 10.1038/cr.2016.28