Titlebook: Gene Therapy Protocols; Jeffrey R. Morgan Book 20022nd edition Humana Press 2002

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Poly-,-Lysine-Based Gene Delivery Systems,vement over existing therapies because of putative advantages in dosing schedule, patient compliance, toxicity, immunogenicity, and cost. Development of a nonviral gene delivery vehicle capable of efficient, cell-specific delivery will be a valuable addition to the clinical armamentarium.

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Targeted Gene Transfer to Liver Using Protein-DNA Complexes,lso are disadvantages, including generally low efficiency and transience of transgene expression. To create more efficient systems, the use of approaches present in natural pathogens has been shown to be helpful. Based on an understanding of these natural components, ligand-polycation DNA delivery s

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Receptor-Directed Molecular Conjugates for Gene Transfer,lecular conjugates to direct gene transfer into mammalian cells in vitro (.–.), and in vivo (.–.). This method has potential for human gene therapy, once it is perfected in animal models. DNA, noncovalently bound to a poly cation polymer that is chemically conjugated to a ligand, can be bound to the

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Gene Transfer into Muscle by Electroporation In Vivo,However, applications of this method have been limited by the relatively low expression levels of the transferred gene. Recently, we investigated the applicability of in vivo electroporation for gene transfer into muscle, using plasmid DNA expressing a cytokine as the vector. The results demonstrate

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Solvoplex Synthetic Vector for Intrapulmonary Gene Delivery,size DNA molecules (therapeutic genes including their endogenous regulatory regions), be used under reduced confinement conditions, be administered repeatedly, and be modified with appropriate ligands that allow specific cell targeting. Until now, many different types of organic compounds have been

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incision

Regulated Expression of Plasmid-Based Gene Therapies,proximately 1 month, and then decline to trace levels that persist for 1 year or more (.,.). However, even during the first month, transgene expression levels are too variable and too low to provide consistent therapeutic levels of circulating proteins. Thus, most intramuscular plasmid-based gene th