Journal article

Immunity to adeno-associated virus-mediated gene transfer in a random-bred canine model of Duchenne muscular dystrophy

Zejing Wang, James M Allen, Stanley R Riddell, Paul Gregorevic, Rainer Storb, Stephen J Tapscott, Jeffrey S Chamberlain, Christian S Kuhr

HUMAN GENE THERAPY | MARY ANN LIEBERT INC | Published : 2007

Abstract

Recombinant adeno-associated virus (rAAV)-mediated gene transfer has shown promise for treating diseases in various animal models including the mdx mouse model of Duchenne muscular dystrophy (DMD). In many cases, however, preclinical studies in inbred mice have not successfully predicted human clinical responses. To assess the potential clinical utility of treating human DMD patients by AAV-mediated gene delivery, we performed a series of direct intramuscular injections in random-bred wild-type dogs. AAV serotypes 2 and 6 carrying different promoter-transgene cassettes were produced as previously described for murine studies and administered intramuscularly. The injection sites were biopsied..

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University of Melbourne Researchers