PMID: 16629054Apr 25, 2006Paper

Non-viral approaches for gene transfer

Acta Myologica : Myopathies and Cardiomyopathies : Official Journal of the Mediterranean Society of Myology
J A WolffJames E Hagstrom

Abstract

Gene therapy has great potential to treat or prevent a variety of both genetic and acquired conditions that include neuromuscular disorders, cardiovascular disease, cancer, and infectious diseases. For recessive genetic disorders such as Duchenne muscular dystrophy, delivery of the normal dystrophin gene to muscle should prevent the myofibers from dying. Despite the great promise and sound principles of gene therapy, its application to humans have been hampered by the inability to safely and effectively deliver genes to the target tissues. Among the several gene transfer methods under development, the use of non-viral delivery methods and specifically naked DNA is particularly attractive in that many of the concerns over the use of viral-mediated methods, such as immunogenicity of viral packaging proteins and cost of viral vector production can be avoided. Recently we used limb veins for efficient, repeatable, and safe delivery of nucleic acids to skeletal myofibers throughout the limb muscles of mammals in vivo. Promising results have been obtained in both rodents and larger animals including non-human primates. Studies in the mdx mouse model indicate that the approach should be of use for patients with Duchenne muscular dystr...Continue Reading

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