Full length dystrophin gene Microgene for dystrophin

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Presentation transcript:

Full length dystrophin gene Microgene for dystrophin Gene therapy for Duchenne Muscular Dystrophy The idea of gene therapy is to genetically alter humans in order to treat a disease. This involves the delivery of functional genes that could ‘correct’ the mutant or disease genes of the recipient. The method uses safe Adeno-associated viruses (AAV) which carry the human gene, in place of one of their own genes, into a cell. In some cells, the gene becomes inserted into a chromosome in the nucleus. However, the dystrophin gene is too large to be packaged inside AVV so dystrophin microgenes, excluding domains dispensable for the functionality of the protein, were designed. Full length dystrophin gene ABD H1 1 2 3 H3 CR 24 H4 CT H2 4 5 6 7 9 10 11 12 13 14 15 16 17 18 19 20 21 22 23 8 Microgene for dystrophin Duchenne Muscular Dystrophy is a neuromuscular disease caused by a defect in the gene coding for the protein dystrophin. This leads to a progressive loss of muscle fibres. Progressive loss of muscle fibres Micro-dystrophin genes are small enough to be carried inside the virus. Tibialis Anterior (shin) muscles injected with AAV vector expressing microdystrophin. Recombinant microdystrophin is stained red. Royal Holloway is part of a worldwide collaboration to develop a treatment for this devastating condition. Research there has led to the development of a gene therapy solution which is now at the clinical trial stage. Muscle fibres damaged by dystrophin deficiency are stained green. royalholloway.ac.uk/breakthrough Micro-dystrophins are functional, re-activate the dystrophin complex at the muscle fibre membrane and yield sustained improvements.