Translational Medicine: Open Access(TMOA)

Molecular Therapies,

Molecular therapies for neuromuscular disorders are rapidly expanding and hold promise for devastating diseases with few options for treatment. Diseases targeted in this review include Duchenne, Becker, and the limb girdle muscular dystrophies. Strategies to repair defective genes involve exon skipping using antisense oligonucleotides or mutation suppression requiring small molecules that permit dose-dependent readthrough of stop codons.

Journal key Highlights

Animal And Human Studies, Applied Physical Sciences, Bacteriology, Biomedical Data Mining, Bone Marrow Transplantation Cell Culture Cellular And Gene Therapy Clinical Biostatistics Clinical Research, Cloning & Transgenesis, Computational Modeling, Computational Modelling Conservation of Biological Resources Critical Care, Cure Of Disease, Developmental Biology, Disease Biomarkers, Genetic Modification Of Cells And Tissues, Genetics/Chromosome Dynamics Hematology, Hematopoietic Cell Transplantation Hematopoietic Cell Transplantation, Immunobiology And Immunotherapy Immunology, Infectiology, Infectious Disease Medicine Intensive Care Medicine Internal Medicine, Kidney Transplantation And Neonatal Stem Cells Laboratory Medicine, Lipoprotein Translation, Medical Gas Therapy Medical Genome Science Medical Informatics, Mesenchymal Stromal Cells Metabolic Translation, Modern Biotechnology, Molecular Biology, Molecular Therapies, Mycology, Organs Internal Medicine Paediatric Transplantation Pain Science Pediatric Transplantation Pluripotent Stem Cells, Practical Application, Regenerative Medicine, Sampling Techniques In Biology Sanitary Zoology Science In Genetics and Breeding Social And Behavioral Science Stem Cell Therapies Stem-Cell Therapy Structural Biology Techniques System Genome Science Toxicology And Pharmacokinetics Translational Haematology Translational Hematology