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Updated: Aug 18, 2026

Generation of Cationic Nanoliposomes for the Efficient Delivery of In Vitro Transcribed Messenger RNA
08:29

Generation of Cationic Nanoliposomes for the Efficient Delivery of In Vitro Transcribed Messenger RNA

Published on: February 1, 2019

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脂纳米颗粒向mRNA配方作为一种治疗小鼠模型中的ornithine-transcarbamylase缺乏症的治疗方法

Kazuto Yamazaki1, Kenji Kubara1, Satoko Ishii1

  • 1Tsukuba Research Laboratories, Eisai Co., Ltd., 5-1-3, Tokodai, Tsukuba, Ibaraki 300-2635, Japan.

Molecular therapy. Nucleic acids
|July 31, 2023
PubMed
概括

脂质纳米颗粒输送的人类甲氨酸转糖酶 (OTC) mRNA通过恢复肝功能有效治疗尿素循环障碍 (UCD). 这种创新的mRNA疗法显示了对甲素转糖酶缺乏症 (OTCD) 的治疗潜力.

关键词:
MT: 交付策略: 甲氨酸转糖氨酸酶.一个OTCSPF灰小鼠.冷电子显微镜的使用方法同型三聚体的构造结构.脂质纳米颗粒的使用方法消息传递 RNA 疗法线粒体的局部化定位

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