Duchenne muscle dystrophy (DMD) is an immobilizing disease, which implies a shortened life expectation due to heart and breathing disorders. A complete lack of dystrophin, which usually connects the cytoskeleton to anchors in the cell wall, causes this disease. In preclinical experiments in a DMD pig cohort, we will proceed to solve approach- and model specific details which would also occur in human application in order to be able to provide efficient and ubiquitous (including heart-specific) therapy of a devastating muscular disease which has proven as in principle amenable to AAV.Cas9-gRNA therapy.
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Prof. Dr. med. vet. Eckhard Wolf
Molekulare Tierzucht, Genzentrum
Prof. Dr. Wolfgang Wurst
Helmholtz Zentrum München,
Deutsches Forschungszentrum für Gesundheit und Umwelt (GmbH)
Ingolstädter Landstraße 1