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- W186401693 abstract "In the last few decades, the field of gene therapy has gained increasing interest, as it has moved from a theoretical concept toward a practical reality. Clinical barriers to patient treatment are still substantial and there remain many obstacles to overcome before gene therapies could become routine treatments. Selection of the gene carrier is very important, as the success of the therapeutic outcome is critically dependent on the transport of the nucleic acid to the correct target in the body at the right time and in the right dose. An ideal gene delivery system should display properties such as prolonged blood circulation, protection of the biopolymer from degradation, ability to target the specific sites of disease, correct intracellular trafficking, controlled release of the nucleic acid payload, no immunoreactions, and no toxicity. In this chapter, we consider the classes of nanoparticle (NP) gene carriers, including viral vectors, lipids, peptides, natural and synthetic polymers, and hybrids of viruses and lipids. The general definitions and therapeutic opportunities in nucleic acid delivery are outlined, and the formulation issues for gene-based therapeutics are evaluated with reference to the biological barriers to the introduction of exogenous genetic material. The last section of the chapter looks at the opportunities and the challenges in NP therapeutics." @default.
- W186401693 created "2016-06-24" @default.
- W186401693 creator A5018262535 @default.
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- W186401693 creator A5070106608 @default.
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- W186401693 date "2011-01-01" @default.
- W186401693 modified "2023-10-13" @default.
- W186401693 title "Nanoparticles for Nucleic Acid Delivery" @default.
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