4.1 Article

Mitochondria-specific delivery system for targeted regulation of mitochondrial gene expression

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STAR PROTOCOLS
卷 2, 期 1, 页码 -

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ELSEVIER
DOI: 10.1016/j.xpro.2020.100275

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资金

  1. National Key Research and Development Program of China [2017YFA0106300]
  2. National Natural Science Foundation of China [81874226, 81803020, 91942309, 92057210]
  3. Thousand Talents Program for Distinguished Young Scholars
  4. International Scientific and Technological Cooperation Program from Guangdong Science and Technology Department [2018A050506033]
  5. Fundamental Research Funds for the Central Universities of China
  6. Three million for Three Years Project of the High-level Talent Special Funding Scheme of Sun Yat-sen Memorial Hospital
  7. Guangzhou Science and Technology Bureau [201902020015]

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This protocol describes the design and preparation of an NP platform for mitochondria-specific gene delivery, which can be used in mice to deliver nucleic acid therapeutics and treat mitochondria-regulated diseases.
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 de-livery (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).

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