2. Academic Validation
  3. Lipid Nanoparticles: A Novel Gene Delivery Technique for Clinical Application

Lipid Nanoparticles: A Novel Gene Delivery Technique for Clinical Application

  • Curr Issues Mol Biol. 2022 Oct 19;44(10):5013-5027. doi: 10.3390/cimb44100341.
Ryuichi Mashima 1,  Shuji Takada 2
Affiliations

Affiliations

  • 1 Department of Clinical Laboratory Medicine, National Center for Child Health and Development, 2-10-1 Okura, Setagaya-ku, Tokyo 157-8535, Japan.
  • 2 Department of Systems BioMedicine, National Research Institute for Child Health and Development, 2-10-1 Okura, Setagaya-ku, Tokyo 157-8535, Japan.
PMID: 36286056 DOI: 10.3390/cimb44100341
Abstract

Lipid nanoparticles (LNPs) are an emerging vehicle for gene delivery that accommodate both nucleic acid and protein. Based on the experience of therapeutic liposomes, current LNPs have been developed based on the chemistry of lipids and RNA and on the biology of human disease. LNPs have been used for the development of Onpattro, an siRNA drug for transthyretin-mediated amyloidosis, in 2018. The subsequent outbreak of COVID-19 required a vaccine for its suppression. LNP-based vaccine production received much attention for this and resulted in great success. In this review, the essential technology of LNP gene delivery has been described according to the chemistry for LNP production and biology for its clinical application.

Keywords

RNA modification; genome editing; lipid nanoparticle; lipids.

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