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α-dystroglycan结合肽A2G80修饰的隐形脂质体作为杜兴肌营养不良症的肌肉靶向载体
Journal of Controlled Release ( IF 10.5 ) Pub Date : 2020-10-17 , DOI: 10.1016/j.jconrel.2020.10.032 Eri Sasaki 1 , Yoshihiro Hayashi 1 , Yuka Kimura 1 , Sanae Sashida 1 , Nobuhito Hamano 1 , Kei Nirasawa 1 , Keisuke Hamada 2 , Fumihiko Katagiri 2 , Yamato Kikkawa 2 , Takaaki Sakai 3 , Akihiro Yoshida 3 , Masahiro Kawada 3 , Shin-Ichi Hirashima 3 , Tsuyoshi Miura 3 , Yoko Endo-Takahashi 1 , Motoyoshi Nomizu 2 , Yoichi Negishi 1
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更新日期:2020-10-17
Journal of Controlled Release ( IF 10.5 ) Pub Date : 2020-10-17 , DOI: 10.1016/j.jconrel.2020.10.032 Eri Sasaki 1 , Yoshihiro Hayashi 1 , Yuka Kimura 1 , Sanae Sashida 1 , Nobuhito Hamano 1 , Kei Nirasawa 1 , Keisuke Hamada 2 , Fumihiko Katagiri 2 , Yamato Kikkawa 2 , Takaaki Sakai 3 , Akihiro Yoshida 3 , Masahiro Kawada 3 , Shin-Ichi Hirashima 3 , Tsuyoshi Miura 3 , Yoko Endo-Takahashi 1 , Motoyoshi Nomizu 2 , Yoichi Negishi 1
Affiliation
需要安全有效的基因疗法来治疗遗传性疾病杜兴氏肌营养不良(DMD)。为此,基因和核酸的肌肉靶向递送系统是理想的。在这项研究中,我们专注于A2G80肽,它对在肌肉细胞膜上表达的α-dystroglycan有亲和力,作为DMD的肌肉靶向纳米载体,并开发了A2G80修饰的脂质体。我们还制备了包覆有长链和短链PEG的A2G80修饰的脂质体,称为A2G80-LSP-Lip,以使用微流控技术改善脂质体的血液循环。
脂质体的粒径约为80nm。通过覆盖试验,A2G80-LSP-Lip对小鼠的肌肉组织切片显示出亲和力。当通过尾静脉将脂质体施用于DMD模型小鼠(mdx小鼠)时,与对照脂质体相比,A2G80-LSP-Lip在肌肉组织中有效积累。这些结果表明,A2G80-LSP-Lip可以通过全身给药作为DMD的肌肉靶向脂质体,并且可能是DMD治疗的有用工具。
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