Cationic liposome-nucleic acid complexes for gene delivery and silencing

Pathways and mechanisms for plasmid DNA and siRNA

Kai K. Ewert, Alexandra Zidovska, Ayesha Ahmad, Nathan F. Bouxsein, Heather M. Evans, Christopher S. McAllister, Charles E. Samuel, Cyrus R. Safinya

    Research output: Chapter in Book/Report/Conference proceedingChapter

    Abstract

    Motivated by the promises of gene therapy, there is great interest in developing non-viral lipid-based vectors for therapeutic applications due to their low immunogenicity, low toxicity, ease of production, and the potential of transferring large pieces of DNA into cells. In fact, cationic liposome (CL) based vectors are among the prevalent synthetic carriers of nucleic acids (NAs) currently used in gene therapy clinical trials worldwide. These vectors are studied both for gene delivery with CL-DNA complexes and gene silencing with CL-siRNA (short interfering RNA) complexes. However, their transfection efficiencies and silencing efficiencies remain low compared to those of engineered viral vectors. This reflects the currently poor understanding of transfection-related mechanisms at the molecular and self-assembled levels, including a lack of knowledge about interactions between membranes and double stranded NAs and between CL-NA complexes and cellular components. In this review we describe our recent efforts to improve the mechanistic understanding of transfection by CL-NA complexes, which will help to design optimal lipid-based carriers of DNA and siRNA for therapeutic gene delivery and gene silencing.

    Original languageEnglish (US)
    Title of host publicationNucleic Acid Transfection
    Pages191-226
    Number of pages36
    Volume296
    DOIs
    StatePublished - 2010

    Publication series

    NameTopics in Current Chemistry
    Volume296
    ISSN (Print)03401022

    Fingerprint

    Liposomes
    Nucleic Acids
    Small Interfering RNA
    Plasmids
    Genes
    DNA
    Gene therapy
    Lipids
    Toxicity
    Membranes

    Keywords

    • Cholesterol
    • Gene delivery
    • Multivalent cationic lipid
    • siRNA
    • Small angle X-ray scattering

    ASJC Scopus subject areas

    • Chemistry(all)

    Cite this

    Ewert, K. K., Zidovska, A., Ahmad, A., Bouxsein, N. F., Evans, H. M., McAllister, C. S., ... Safinya, C. R. (2010). Cationic liposome-nucleic acid complexes for gene delivery and silencing: Pathways and mechanisms for plasmid DNA and siRNA. In Nucleic Acid Transfection (Vol. 296, pp. 191-226). (Topics in Current Chemistry; Vol. 296). https://doi.org/10.1007/128_2010_70

    Cationic liposome-nucleic acid complexes for gene delivery and silencing : Pathways and mechanisms for plasmid DNA and siRNA. / Ewert, Kai K.; Zidovska, Alexandra; Ahmad, Ayesha; Bouxsein, Nathan F.; Evans, Heather M.; McAllister, Christopher S.; Samuel, Charles E.; Safinya, Cyrus R.

    Nucleic Acid Transfection. Vol. 296 2010. p. 191-226 (Topics in Current Chemistry; Vol. 296).

    Research output: Chapter in Book/Report/Conference proceedingChapter

    Ewert, KK, Zidovska, A, Ahmad, A, Bouxsein, NF, Evans, HM, McAllister, CS, Samuel, CE & Safinya, CR 2010, Cationic liposome-nucleic acid complexes for gene delivery and silencing: Pathways and mechanisms for plasmid DNA and siRNA. in Nucleic Acid Transfection. vol. 296, Topics in Current Chemistry, vol. 296, pp. 191-226. https://doi.org/10.1007/128_2010_70
    Ewert KK, Zidovska A, Ahmad A, Bouxsein NF, Evans HM, McAllister CS et al. Cationic liposome-nucleic acid complexes for gene delivery and silencing: Pathways and mechanisms for plasmid DNA and siRNA. In Nucleic Acid Transfection. Vol. 296. 2010. p. 191-226. (Topics in Current Chemistry). https://doi.org/10.1007/128_2010_70
    Ewert, Kai K. ; Zidovska, Alexandra ; Ahmad, Ayesha ; Bouxsein, Nathan F. ; Evans, Heather M. ; McAllister, Christopher S. ; Samuel, Charles E. ; Safinya, Cyrus R. / Cationic liposome-nucleic acid complexes for gene delivery and silencing : Pathways and mechanisms for plasmid DNA and siRNA. Nucleic Acid Transfection. Vol. 296 2010. pp. 191-226 (Topics in Current Chemistry).
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