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Article

Recombinant Adeno-Associated Viral Vectors Mediate Long-Term Transgene Expression in Muscle

10 Apr 1997

Abstract excerpt

Gene transfer to muscle holds overt promise for the treatment of inherited myopathies, lysosomal storage disorders, and serum protein deficiencies. In addition, muscle could provide a reservoir for delivery of therapeutic molecules like blood clotting factors, erythropoietin, or insulin. To date, successful gene transfer to muscle has been limited by the inefficiency of the vector delivery systems and the...

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PMID: 9113506
DOI: 10.1089/hum.1997.8.6-659

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