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Early adenovirus-mediated gene transfer effectively prevents muscular dystrophy in alpha-sarcoglycan-deficient mice

Lookup NU author(s): Professor Volker Straub

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Abstract

Limb-girdle muscular dystrophy type 2D (LGMD 2D) is the most common cause of LGMD with a sarcoglycan defect. We recently engineered a murine model for this progressive disease and we investigated the possibility of preventing the development of muscular dystrophy in these animals by adenovirus-mediated gene transfer of human alpha-sarcoglycan. Here we report that a single intramuscular injection of a first generation adenovirus into the skeletal muscle of neonate mice led to sustained expression of alpha-sarcoglycan at the sarcolemma of transduced myofibers for at least 7 months. The morphology of transduced muscles was consequently preserved. In addition, we have used contrast agent-enhanced magnetic resonance imaging (MRI) to investigate sarcolemmal integrity in adenovirus-injected animals and have thereby demonstrated maintenance of sarcolemmal function. In conclusion, we provide evidence that early virus-mediated gene transfer of a sarcoglycan protein constitutes a promising therapeutic strategy for LGMDs and that the benefits of this approach can easily and effectively be monitored by noninvasive methodologies such as MRI.


Publication metadata

Author(s): Straub V; Allamand V; Donahue KM; Davisson RL; Davidson BL; Campbell KP

Publication type: Article

Publication status: Published

Journal: Gene Therapy

Year: 2000

Volume: 7

Issue: 16

Pages: 1385-1391

ISSN (print): 0969-7128

ISSN (electronic): 1476-5462

Publisher: Nature Publishing Group

URL: http://dx.doi.org/10.1038/sj.gt.3301247

DOI: 10.1038/sj.gt.3301247


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