期刊
GENES
卷 13, 期 2, 页码 -出版社
MDPI
DOI: 10.3390/genes13020257
关键词
Duchenne muscular dystrophy; exon skipping therapy; antisense oligonucleotide; microdystrophin; adeno-associated virus
资金
- Muscular Dystrophy Canada
- Friends of Garrett Cumming Research Fund
- HM Toupin Neurological Science Research Fund
- Alberta Innovates: Health Solutions (AIHS)
- Jesse's Journey
- Women and Children's Health Research Institute (WCHRI)
This review summarizes the current state of development of N-terminal antisense and gene therapies for Duchenne muscular dystrophy (DMD), mainly focusing on exon-skipping therapy for duplications and deletions, as well as microdystrophin therapy.
Duchenne muscular dystrophy (DMD) is a fatal genetic disease affecting children that is caused by a mutation in the gene encoding for dystrophin. In the absence of functional dystrophin, patients experience progressive muscle deterioration, leaving them wheelchair-bound by age 12 and with few patients surviving beyond their third decade of life as the disease advances and causes cardiac and respiratory difficulties. In recent years, an increasing number of antisense and gene therapies have been studied for the treatment of muscular dystrophy; however, few of these therapies focus on treating mutations arising in the N-terminal encoding region of the dystrophin gene. This review summarizes the current state of development of N-terminal antisense and gene therapies for DMD, mainly focusing on exon-skipping therapy for duplications and deletions, as well as microdystrophin therapy.
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