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Delivery of Oligonucleotides Using a Self-Degradable Lipid-Like Material

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Title: Delivery of Oligonucleotides Using a Self-Degradable Lipid-Like Material
Authors: Tanaka, Hiroki Browse this author
Takata, Nae Browse this author
Sakurai, Yu Browse this author
Yoshida, Tokuyuki Browse this author
Inoue, Takao Browse this author
Tamagawa, Shinya Browse this author
Nakai, Yuta Browse this author
Tange, Kota Browse this author
Yoshioka, Hiroki Browse this author
Maeki, Masatoshi Browse this author
Tokeshi, Manabu Browse this author →KAKEN DB
Akita, Hidetaka Browse this author
Keywords: siRNA
antisense oligonucleotide
lipid nanoparticle
Issue Date: Apr-2021
Publisher: MDPI
Journal Title: Pharmaceutics
Volume: 13
Issue: 4
Start Page: 544
Publisher DOI: 10.3390/pharmaceutics13040544
Abstract: The world-first success of lipid nanoparticle (LNP)-based siRNA therapeutics (ONPATTRO(R)) promises to accelerate developments in siRNA therapeutics/gene therapy using LNP-type drug delivery systems (DDS). In this study, we explore the optimal composition of an LNP containing a self-degradable material (ssPalmO-Phe) for the delivery of oligonucleotides. siRNA or antisense oligonucleotides (ASO) were encapsulated in LNP with different lipid compositions. The hepatic knockdown efficiency of the target genes and liver toxicity were evaluated. The optimal compositions for the siRNA were different from those for ASO, and different from those for mRNA that were reported in a previous study. Extracellular stability, endosomal escape and cellular uptake appear to be the key processes for the successful delivery of mRNA, siRNA and ASO, respectively. Moreover, the compositions of the LNPs likely contribute to their toxicity. The lipid composition of the LNP needs to be optimized depending on the type of nucleic acids under consideration if the applications of LNPs are to be further expanded.
Type: article
Appears in Collections:工学院・工学研究院 (Graduate School of Engineering / Faculty of Engineering) > 雑誌発表論文等 (Peer-reviewed Journal Articles, etc)

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