Gene Therapy for the Treatment of Hemophilia B Using PINC-Formulated Plasmid Delivered to Muscle with Electroporation

Gene therapy, as a safe and efficacious treatment or prevention of diseases, is one of the next fundamental medical innovations. Direct injection of plasmid into skeletal muscle is still a relatively inefficient and highly variable method of gene transfer. However, published reports have shown that...

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Veröffentlicht in:	Molecular therapy 2001-04, Vol.3 (4), p.574-583
Hauptverfasser:	Fewell, Jason G., MacLaughlin, Fiona, Mehta, Vidya, Gondo, Margaret, Nicol, Francois, Wilson, Elizabeth, Smith, Louis C.
Format:	Artikel
Sprache:	eng
Schlagworte:	Animals Blotting, Western Creatine Kinase - metabolism Dogs Dose-Response Relationship, Drug electroporation Electroporation - methods factor IX Factor IX - genetics Gene expression Gene therapy Genetic Therapy - methods Hemophilia Hemophilia B - therapy Humans Immunohistochemistry Kinases Laboratories Male Mice Mice, Inbred C57BL Mice, SCID muscle Muscle, Skeletal - metabolism Muscles - metabolism Musculoskeletal system PINC Plasma plasmid Plasmids Plasmids - genetics Plasmids - metabolism Polymers - chemistry Time Factors Vectors (Biology)
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