Journal article
Current Status of Pharmaceutical and Genetic Therapeutic Approaches to Treat DMD
Molecular Therapy, Vol.19(5), pp.830-840
2011
Abstract
Duchenne muscular dystrophy (DMD) is a genetic disease affecting about one in every 3,500 boys. This X-linked pathology is due to the absence of dystrophin in muscle fibers. This lack of dystrophin leads to the progressive muscle degeneration that is often responsible for the death of the DMD patients during the third decade of their life. There are currently no curative treatments for this disease but different therapeutic approaches are being studied. Gene therapy consists of introducing a transgene coding for full-length or a truncated version of dystrophin complementary DNA (cDNA) in muscles, whereas pharmaceutical therapy includes the use of chemical/biochemical substances to restore dystrophin expression or alleviate the DMD phenotype. Over the past years, many potential drugs were explored. This led to several clinical trials for gentamicin and ataluren (PTC124) allowing stop codon read-through. An alternative approach is to induce the expression of an internally deleted, partially functional dystrophin protein through exon skipping. The vectors and the methods used in gene therapy have been continually improving in order to obtain greater encapsidation capacity and better transduction efficiency. The most promising experimental approaches using pharmaceutical and gene therapies are reviewed in this article.
Details
- Title
- Current Status of Pharmaceutical and Genetic Therapeutic Approaches to Treat DMD
- Authors/Creators
- C. Pichavant (Author/Creator)A. Aartsma-Rus (Author/Creator)P.R. Clemens (Author/Creator)K.E. Davies (Author/Creator)G. Dickson (Author/Creator)S. Takeda (Author/Creator)S.D. Wilton (Author/Creator)J.A. Wolff (Author/Creator)C.I. Wooddell (Author/Creator)X. Xiao (Author/Creator)J.P. Tremblay (Author/Creator)
- Publication Details
- Molecular Therapy, Vol.19(5), pp.830-840
- Publisher
- Nature Publishing Group
- Identifiers
- 991005545310407891
- Copyright
- 2011 The American Society of Gene & Cell Therapy
- Murdoch Affiliation
- Murdoch University
- Language
- English
- Resource Type
- Journal article
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- Collaboration types
- Industry collaboration
- Domestic collaboration
- International collaboration
- Citation topics
- 1 Clinical & Life Sciences
- 1.255 Musculoskeletal Disorders
- 1.255.628 Duchenne Muscular Dystrophy
- Web Of Science research areas
- Biotechnology & Applied Microbiology
- Genetics & Heredity
- Medicine, Research & Experimental
- ESI research areas
- Clinical Medicine