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• W2306207712 abstract "Gene therapy has drawn significant attention over the past two decades for the treatment of inherited and acquired diseases. Major research efforts have focused on designing suitable carrier vectors that compact and protect oligonucleotides for gene therapy. DNA or RNA is large, hydrophilic macromolecule with a negative charge, very unstable in the physiological environment (free oligonucleotides and DNA are rapidly degraded by serum nucleases in the blood when injected intravenously) and do not cross biological membranes effectively. Since both the genes and cell surfaces are negatively charged, spontaneous entry of unprotected genes inside cells is difficult. Nucleic acids can be locally injected into specific organs such as muscle or liver, producing high gene expression. However, this strategy is limited to tissues that are easily accessible by direct injection, such as skin, whereas it is not applicable to systemic gene delivery or is unrealistic for a commercial gene therapy. Therefore, the clinical success of gene therapy is heavily dependent on the development of safe and efficient gene delivery systems, known as vectors. Research was initially focused on viral vectors, including both retroviruses and adenoviruses, as these vectors exhibited high efficiency at delivering both DNA and RNA to numerous cell lines. However, several problems associated with viral vector systems, such as toxicity, immunogenicity, and limitations with respect to scale-up procedures, encouraged the investigation of other potential DNA or RNA carriers into targeted tissue. Non-viral vector systems, including cationic lipids, polymers, dendrimers, peptides, and nanoparticles, are other means of compacting DNA for systemic delivery, but, unlike viral analogues, non-viral gene carriers consistently demonstrate significant reduced transfection efficiency due to numerous extra- and intracellular obstacles. Their biocompatibility and facilitated production make them potentially useful and attractive for gene therapy. The mechanism, current challenges, and different approaches are discussed in this chapter." @default.
• W2306207712 created "2016-06-24" @default.
• W2306207712 creator A5027980410 @default.
• W2306207712 creator A5066547166 @default.
• W2306207712 date "2016-01-01" @default.
• W2306207712 modified "2023-10-12" @default.
• W2306207712 title "Mechanism, current challenges and new approaches for non viral gene delivery" @default.
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