Failure to correct murine muscular dystrophy

Giuliana Ferrari, Anna Stornaiuolo and Fulvio Mavilio ()
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Giuliana Ferrari: H. San Raffaele-Telethon Institute for Gene Therapy
Anna Stornaiuolo: GenEra SpA
Fulvio Mavilio: H. San Raffaele-Telethon Institute for Gene Therapy

Nature, 2001, vol. 411, issue 6841, 1014-1015

Abstract: Abstract Bone-marrow cells have the potential to differentiate into other cell types such as muscle fibres, and can be transplanted into acutely1 or chronically2 damaged muscle as a way of delivering normal dystrophin (the protein that is defective or missing in Duchenne's muscular dystrophy) to the skeletal and heart muscle of mdx mice2,3, an animal model for this disease. But the corrective potential of this approach has been hard to estimate against the high background of muscle fibres that spontaneously revert to synthesizing dystrophin, a feature of the original mdx mutation4. Here we test the long-term efficacy of bone-marrow transplantation in a different mdx mutant which is free of this problem and find that it has no impact on murine muscular dystrophy.

Date: 2001
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DOI: 10.1038/35082631

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