The potential usefulness of viral gene replacement therapy in human disease has been an exciting and widely studied subject in the field of skeletal muscle disease over the past 20 years. Unfortunately, although the strategy of replacing or supplementing copies of the mutant gene is a simple concept, a number of factors complicating the situation have been identified as gene therapy trials progress to clinical studies. Although some of these problems, including the immunological response to viral vectors, are problems encountered with all forms of gene therapy, the use of gene therapy in skeletal muscle also poses additional challenges for which treatment strategies need to be optimized. Despite these challenges, substantial progress has been made in recent years towards optimizing viral gene therapy for skeletal muscle disease in animal models, with a view to optimizing the safety and efficacy of viral gene therapy in humans. Skeletal muscle disorders represent considerable opportunities for researchers developing viral gene therapy strategies, due to the many monogenetic skeletal muscle diseases, clear and quantifiable clinical phenotypes, and the ease of access to muscle tissue. Mutations in the dystrophin gene predispose the myofiber membrane to membrane damage induced by contraction, leading to inflammation, myonecrosis and progressive loss of functional muscle tissue. Several mouse and canine models of dystrophinopathy have been used to study these diseases and evaluate potential therapies, including several approaches to viral gene therapy.
Scientific Tracks Abstracts: Molecular and Genetic Medicine
Scientific Tracks Abstracts: Molecular and Genetic Medicine
Scientific Tracks Abstracts: Molecular and Genetic Medicine
Scientific Tracks Abstracts: Molecular and Genetic Medicine
Posters & Accepted Abstracts: Journal of Molecular Biomarkers & Diagnosis
Posters & Accepted Abstracts: Molecular Biomarkers & Diagnosis
Keynote: Molecular Biomarkers & Diagnosis
Accepted Abstracts: Molecular and Genetic Medicine
Accepted Abstracts: Molecular and Genetic Medicine
Scientific Tracks Abstracts: Journal of Molecular Biomarkers & Diagnosis
Scientific Tracks Abstracts: Molecular Biomarkers & Diagnosis
Scientific Tracks Abstracts: Journal of Tissue Science and Engineering
Scientific Tracks Abstracts: Journal of Tissue Science and Engineering
Scientific Tracks Abstracts: Metabolomics:Open Access
Scientific Tracks Abstracts: Metabolomics:Open Access
Journal of Genetics and DNA Research received 3 citations as per Google Scholar report
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