4.8 Article

Non-viral vectors for RNA delivery

期刊

JOURNAL OF CONTROLLED RELEASE
卷 342, 期 -, 页码 241-279

出版社

ELSEVIER
DOI: 10.1016/j.jconrel.2022.01.008

关键词

RNA drugs; Non-viral vector; Biological barrier; Control release; Gene therapy

资金

  1. National Natural Science Foundation of China [81773650, 81690264, 81973259]
  2. Beijing Natural Science Foundation [J200018]
  3. National Key New Drug Creation and Manufacturing Program, Ministry of Science and Technology of China [2018ZX09721003-004]

向作者/读者索取更多资源

RNA-based therapy is a promising strategy for disease treatment, and recent advances in non-viral delivery systems have shown potential in protecting RNA, facilitating cell internalization, and controlled release of therapeutics.
RNA-based therapy is a promising and potential strategy for disease treatment by introducing exogenous nucleic acids such as messenger RNA (mRNA), small interfering RNA (siRNA), microRNA (miRNA) or antisense oligonucleotides (ASO) to modulate gene expression in specific cells. It is exciting that mRNA encoding the spike protein of COVID-19 (coronavirus disease 2019) delivered by lipid nanoparticles (LNPs) exhibits the efficient protection of lungs infection against the virus. In this review, we introduce the biological barriers to RNA delivery in vivo and discuss recent advances in non-viral delivery systems, such as lipid-based nanoparticles, polymeric nanoparticles, N-acetylgalactosamine (GalNAc)-siRNA conjugate, and biomimetic nanovectors, which can protect RNAs against degradation by ribonucleases, accumulate in specific tissue, facilitate cell internalization, and allow for the controlled release of the encapsulated therapeutics.

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