Review article duchenne muscular dystrophy
Recent advances in Duchenne muscular dystrophy
Duchenne muscular dystrophy DMD review article duchenne a lethal progressive pediatric muscle disorder muscular dystrophy genetically inherited as an X-linked disease that caused by mutations in the dystrophin gene.
For decades, scientists tried massively to find an effective therapy method, but there is no absolute cure currently for patients with DMD, nevertheless, recent advanced progressions on the treatment of DMD will be /phd-with-no-thesis-examples.html in the muscular dystrophy.
Muscular dystrophy promising gene therapies are currently learn more here investigation. These include gene replacement, exon skipping, suppression of stop codons. This review intents to briefly describe these methods and comment on their advances. However, there are different review article duchenne muscular dystrophy of mutations in this gene, so such therapeutic approaches are highly mutation specific and thus are personalized.
Therefore, DMD has arisen as a model of review article duchenne muscular dystrophy disorder for understanding and overcoming of the challenges of developing personalized genetic medicines, consequently, the lessons learned from these approaches will be applicable to many other disorders. This muscular dystrophy provides an update on the review article duchenne gene therapies for DMD that aim to compensate for dystrophin deficiency and the related clinical trials.
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Duchenne muscular dystrophy DMD is the most common form of muscular dystrophy in childhood. It is caused by mutations of the DMD gene, leading to progressive muscle weakness, loss of independent ambulation by early teens, and premature death due to cardiorespiratory complications. The diagnosis can usually be made after careful review of the history and examination of affected boys presenting with developmental delay, proximal weakness, and elevated serum creatine kinase, plus confirmation by muscle biopsy or genetic testing.
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