Guangdong Provincial Key Laboratory of Malignant Tumor Epigenetics and Gene Regulation, Sun Yat-sen Memorial Hospital, Sun Yat-sen University, Guangzhou 510120, China.
Medical Research Center, Sun Yat-sen Memorial Hospital, Sun Yat-sen University, Guangzhou 510120, China.
STAR Protoc. 2021 Jan 15;2(1):100275. doi: 10.1016/j.xpro.2020.100275. eCollection 2021 Mar 19.
Targeted regulation of mitochondrial gene expression is challenging due to the lack of a mitochondria-specific delivery system. We have previously developed various stimuli-responsive nanoparticle (NP)-based delivery systems to transport nucleic acids for regulation of target gene expression. This protocol describes the design and preparation of an NP platform for mitochondria-specific gene delivery (mito-NP). We use mito-NP in primary liver fibroblasts that are transplanted into mice. Mito-NP can be used to deliver various nucleic acid therapeutics and to treat mitochondria-regulated diseases. For complete details on the use and execution of this protocol, please refer to Zhao et al. (2020).
由于缺乏线粒体特异性递药系统，靶向调控线粒体基因表达具有挑战性。我们先前开发了各种基于刺激响应性纳米颗粒（NP）的递药系统，以输送核酸来调控靶基因表达。本方案描述了用于线粒体特异性基因递药（mito-NP）的 NP 平台的设计和制备。我们在移植到小鼠体内的原代肝成纤维细胞中使用 mito-NP。mito-NP 可用于递送各种核酸治疗药物，并治疗受线粒体调控的疾病。如需了解本方案使用和实施的完整详细信息，请参考 Zhao 等人（2020 年）。
