Article
Adenovirus-mediated utrophin gene transfer mitigates the dystrophic phenotype of mdx mouse muscles.
Human gene therapy - 20 May 1999
Gilbert R, Nalbantoglu J, Petrof B J, Ebihara S, Guibinga G H, Tinsley J M, Kamen A, Massie B, Davies K E, Karpati G
Abstract excerpt
Utrophin is a close homolog of dystrophin, the protein whose mutations cause Duchenne muscular dystrophy (DMD). Utrophin is present at low levels in normal and dystrophic muscle, whereas dystrophin is largely absent in DMD. In such cases, the replacement of dystrophin using a utrophin gene transfer strategy could be more advantageous because utrophin would not be a neoantigen. To establish if adenovirus...
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