Oligonucleotide-Based Delivery Systems

A special issue of Pharmaceutics (ISSN 1999-4923). This special issue belongs to the section "Drug Delivery and Controlled Release".

Deadline for manuscript submissions: closed (31 January 2023) | Viewed by 7207

Special Issue Editors


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Guest Editor
Department of Pharmaceutical Informatics, Graduate School of Biomedical Sciences, Nagasaki University, 1-7-1 Sakamoto, Nagasaki 852-8588, Japan
Interests: drug delivery systems; lipid nanoparticles; liposomes; targeting

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Guest Editor
Laboratory of Biofunctional Molecules, Graduate School of Biomedical Sciences, Nagasaki University, 1-14 Bunkyo-machi, Nagasaki 852-8521, Japan
Interests: antisense therapeutics; nucleic acid chemistry; ligand-targeted oligonucleotide delivery
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Special Issue Information

Dear Colleagues,

In recent years, delivery studies of oligonucleotides including messenger RNA, small interfering RNA, microRNA, and antisense oligonucleotides have been intensively conducted. Currently, oligonucleotide drug delivery research can be broadly classified into 1) chemical modification and ligand conjugation of oligonucleotides, 2) nano-particulation such as lipid nanoparticles, and 3) the use of external stimuli, with attempts to combine each method. It is also important to properly evaluate and understand the pharmacokinetics and toxicity of the oligonucleotides compositions, and to justify their delivery strategies at the body, tissue, and cellular levels.

In this Special Issue on "Oligonucleotide-Based Delivery Systems," we will focus on the development of new delivery systems of therapeutic oligonucleotides to be able to target various organs, tissues, and cells with a systematic evaluation of the pharmacokinetics and toxicity of the formulations. We would like to discuss the therapeutic possibilities of novel oligonucleotide-based drugs with specialized delivery systems for refractory diseases, and to consider their potential problems to be solved.

Prof. Dr. Shigeru Kawakami
Dr. Tsuyoshi Yamamoto
Guest Editors

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Keywords

  • drug delivery systems 
  • oligonucleotides 
  • lipid nanoparticles (LNPs) 
  • ligand conjugation 
  • pharmacokinetics 
  • toxicity

Published Papers (1 paper)

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Research

13 pages, 2339 KiB  
Article
Stability Study of mRNA-Lipid Nanoparticles Exposed to Various Conditions Based on the Evaluation between Physicochemical Properties and Their Relation with Protein Expression Ability
by Mariko Kamiya, Makoto Matsumoto, Kazuma Yamashita, Tatsunori Izumi, Maho Kawaguchi, Shusaku Mizukami, Masako Tsurumaru, Hidefumi Mukai and Shigeru Kawakami
Pharmaceutics 2022, 14(11), 2357; https://doi.org/10.3390/pharmaceutics14112357 - 31 Oct 2022
Cited by 15 | Viewed by 6795
Abstract
Lipid nanoparticles (LNPs) are currently in the spotlight as delivery systems for mRNA therapeutics and have been used in the Pfizer/BioNTech and Moderna COVID-19 vaccines. mRNA-LNP formulations have been indicated to require strict control, including maintenance at fairly low temperatures during their transport [...] Read more.
Lipid nanoparticles (LNPs) are currently in the spotlight as delivery systems for mRNA therapeutics and have been used in the Pfizer/BioNTech and Moderna COVID-19 vaccines. mRNA-LNP formulations have been indicated to require strict control, including maintenance at fairly low temperatures during their transport and storage. Since it is a new pharmaceutical modality, there is a lack of information on the systematic investigation of how storage and handling conditions affect the physicochemical properties of mRNA-LNPs and their protein expression ability. In this study, using the mRNA-LNPs with standard composition, we evaluated the effects of temperature, cryoprotectants, vibration, light exposure, and syringe aspiration from the vials on the physicochemical properties of nanoparticles in relation to their in vitro/in vivo protein expression ability. Among these factors, storage at −80 °C without a cryoprotectant caused a decrease in protein expression, which may be attributed to particle aggregation. Exposure to vibration and light also caused similar changes under certain conditions. Exposure to these factors can occur during laboratory and hospital handling. It is essential to have sufficient knowledge of the stability of mRNA-LNPs in terms of their physical properties and protein expression ability at an early stage to ensure reproducible research and development and medical care. Full article
(This article belongs to the Special Issue Oligonucleotide-Based Delivery Systems)
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