Polymer Design for Nonviral Gene Delivery
Identifieur interne : 001C83 ( Main/Exploration ); précédent : 001C82; suivant : 001C84Polymer Design for Nonviral Gene Delivery
Auteurs : Kam W. Leong [États-Unis]Source :
Abstract
Abstract: Gene therapy continues to hold promise in treating a variety of inherited and acquired diseases. The great majority of gene therapy trials rely on viral vectors for gene transduction because of their high efficiency. Viruses remain the vectors of choice in achieving high efficiency of gene transfer in vivo. Viral vectors, however, pose safety concerns unlikely to abate in the near future [1–3]. Issues of immunogenicity and toxicity remain a challenge. Limitations of cell mitosis for retrovirus, contamination of adenovirus, and packaging constraints of adeno-associated virus (AAV) also lessen their appeal. Non-viral vectors, although achieving only transient and lower gene expression level, may be able to compete on potential advantages of ease of synthesis, low immune response, and unrestricted plasmid size [4–9]. They have the potential to be administered repeatedly with minimal host immune response. They can also satisfy many of the pharmaceutical issues better than the viral vectors, such as scale-up, storage stability, and quality control. However, non-viral gene delivery is still too inefficient to be therapeutic for many applications. Development of safe and effective non-viral gene carriers is still critical to the ultimate success of gene therapy.
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DOI: 10.1007/978-0-387-25842-3_9
Affiliations:
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