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Efficient long-term gene transfer into muscle tissue of immunocompetent mice by adeno-associated virus vector

1 Nov 1996

Abstract excerpt

Muscle-directed gene transfer is being considered for the treatment of several metabolic diseases, including hemophilia and Duchene's muscular dystrophy. Previous efforts to target this tissue for somatic delivery with various vector systems have resulted in transient expression due to silencing of the transgene or to an immune response against the vector-transduced cells. We introduced recombinant...

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PMID: 8892935
DOI: 10.1128/jvi.70.11.8098-8108.1996

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