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Exon skipping therapy for Duchenne muscular dystrophy
- Source :
- Advanced Drug Delivery Reviews. 87:104-107
- Publication Year :
- 2015
- Publisher :
- Elsevier BV, 2015.
-
Abstract
- Duchenne muscular dystrophy (DMD) is caused mostly by internal deletions in the gene for dystrophin, a protein essential for maintaining muscle cell membrane integrity. These deletions abrogate the reading frame and the lack of dystrophin results in progressive muscle deterioration. DMD patients experience progressive loss of ambulation, followed by a need for assisted ventilation, and eventual death in mid-twenties. By the method of exon skipping in dystrophin pre-mRNA the reading frame is restored and the internally deleted but functional dystrophin is produced. Two oligonucleotide drugs that induce desired exon skipping are currently in advanced clinical trials.
- Subjects :
- musculoskeletal diseases
congenital, hereditary, and neonatal diseases and abnormalities
RNA Splicing
Duchenne muscular dystrophy
Oligonucleotides
Pharmaceutical Science
Assisted ventilation
Eteplirsen
Morpholinos
Dystrophin
medicine
Animals
Humans
Myocyte
Drisapersen
Randomized Controlled Trials as Topic
biology
business.industry
Exons
medicine.disease
Exon skipping
Muscular Dystrophy, Duchenne
Mutation
RNA splicing
biology.protein
Cancer research
business
Subjects
Details
- ISSN :
- 0169409X
- Volume :
- 87
- Database :
- OpenAIRE
- Journal :
- Advanced Drug Delivery Reviews
- Accession number :
- edsair.doi.dedup.....78c4880e86c490d5067e50ab6a51b122