Charge-Conversion Ternary Polyplex with Endosome Disruption Moiety: A Technique for Efficient and Safe Gene Delivery

Authors

  • Yan Lee Dr.,

    1. Division of Clinical Biotechnology, Center for Disease Biology and Integrative Medicine, Graduate School of Medicine, University of Tokyo, 7-3-1 Hongo, Bunkyo-ku, Tokyo 113-0033 (Japan), Fax: (+81) 3-5841-7139
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  • Kanjiro Miyata Dr.,

    1. Department of Bioengineering, University of Tokyo, 7-3-1 Hongo, Bunkyo-ku, Tokyo 113-0033 (Japan)
    2. Center for Nanobio Integration, University of Tokyo, 7-3-1 Hongo, Bunkyo-ku, Tokyo 113-8656 (Japan)
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  • Makoto Oba Dr.,

    1. Department of Clinical Vascular Regeneration, University of Tokyo, 7-3-1 Hongo, Bunkyo-ku, Tokyo 113-0033 (Japan)
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  • Takehiko Ishii Dr.,

    1. Department of Bioengineering, University of Tokyo, 7-3-1 Hongo, Bunkyo-ku, Tokyo 113-0033 (Japan)
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  • Shigeto Fukushima,

    1. Division of Clinical Biotechnology, Center for Disease Biology and Integrative Medicine, Graduate School of Medicine, University of Tokyo, 7-3-1 Hongo, Bunkyo-ku, Tokyo 113-0033 (Japan), Fax: (+81) 3-5841-7139
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  • Muri Han Dr.,

    1. Department of Materials Engineering, University of Tokyo, 7-3-1 Hongo, Bunkyo-ku, Tokyo 113-8656 (Japan)
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  • Hiroyuki Koyama Prof. Dr.,

    1. Department of Clinical Vascular Regeneration, University of Tokyo, 7-3-1 Hongo, Bunkyo-ku, Tokyo 113-0033 (Japan)
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  • Nobuhiro Nishiyama Dr.,

    1. Division of Clinical Biotechnology, Center for Disease Biology and Integrative Medicine, Graduate School of Medicine, University of Tokyo, 7-3-1 Hongo, Bunkyo-ku, Tokyo 113-0033 (Japan), Fax: (+81) 3-5841-7139
    2. Center for Nanobio Integration, University of Tokyo, 7-3-1 Hongo, Bunkyo-ku, Tokyo 113-8656 (Japan)
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  • Kazunori Kataoka Prof. Dr.

    1. Division of Clinical Biotechnology, Center for Disease Biology and Integrative Medicine, Graduate School of Medicine, University of Tokyo, 7-3-1 Hongo, Bunkyo-ku, Tokyo 113-0033 (Japan), Fax: (+81) 3-5841-7139
    2. Department of Bioengineering, University of Tokyo, 7-3-1 Hongo, Bunkyo-ku, Tokyo 113-0033 (Japan)
    3. Center for Nanobio Integration, University of Tokyo, 7-3-1 Hongo, Bunkyo-ku, Tokyo 113-8656 (Japan)
    4. Department of Materials Engineering, University of Tokyo, 7-3-1 Hongo, Bunkyo-ku, Tokyo 113-8656 (Japan)
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  • This work was supported by a Core Research for Evolutional Science and Technology (CREST) grant from the Japan Science and Technology Agency (JST).

Abstract

original image

Wrapped for special delivery: A ternary polyplex, with an endosomal disruption moiety based on the charge-conversion polymer pAsp(DET-Aco), showed negative charges for serum stability and low cytotoxicity, but the charges became positive and the endosome disruption moiety was exposed (see picture). High transfection efficiency and minimal cytotoxicity were observed with primary cells.

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