期刊
PEDIATRIC NEUROLOGY
卷 51, 期 5, 页码 607-618出版社
ELSEVIER SCIENCE INC
DOI: 10.1016/j.pediatrneurol.2014.08.002
关键词
gene therapy; muscular dystrophy; exon skipping
资金
- NICHD NIH HHS [T32 HD043003] Funding Source: Medline
Gene therapy for the muscular dystrophies has evolved as a promising treatment for this progressive group of disorders. Although corticosteroids and/or supportive treatments remain the standard of care for Duchenne muscular dystrophy, loss of ambulation, respiratory failure, and compromised cardiac function is the inevitable outcome. Recent developments in genetically mediated therapies have allowed for personalized treatments that strategically target individual muscular dystrophy subtypes based on disease pathomechanism and phenotype. In this review, we highlight the therapeutic progress with emphasis on evolving preclinical data and our own experience in completed clinical trials and others currently underway. We also discuss the lessons we have learned along the way and the strategies developed to overcome limitations and obstacles in this field.
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