Advanced Nanotechnology-Based Nucleic Acid Medicines

Nucleic acid medicines are a highly attractive modality that act in a sequence-specific manner on target molecules. To date, 21 such products have been approved by the Food and Drug Administration. However, the development of nucleic acid medicines continues to face various challenges, including tis...

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Main Authors: Noriko Miyamoto, Mina Sakuragi, Yukio Kitade
Format: Article
Language:English
Published: MDPI AG 2024-10-01
Series:Pharmaceutics
Subjects:
Online Access:https://www.mdpi.com/1999-4923/16/11/1367
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author Noriko Miyamoto
Mina Sakuragi
Yukio Kitade
author_facet Noriko Miyamoto
Mina Sakuragi
Yukio Kitade
author_sort Noriko Miyamoto
collection DOAJ
description Nucleic acid medicines are a highly attractive modality that act in a sequence-specific manner on target molecules. To date, 21 such products have been approved by the Food and Drug Administration. However, the development of nucleic acid medicines continues to face various challenges, including tissue and cell targeting as well as intracellular delivery. Numerous research groups are addressing these issues by advancing the development of nucleic acid medicines through nanotechnology. In countries other than Japan (including Europe and the USA), >40 nanotechnology-based nucleic acid medicines have been tested in clinical trials, and 15 clinical trials are ongoing. In Japan, three phase I trials are ongoing, and future results are awaited. The review summarizes the latest research in the nanotechnology of nucleic acid medicines and statuses of clinical trials in Japan, with expectations of further evolutions.
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spelling doaj-art-716d414a46f4491fb9f3ea76834af2ab2025-08-20T01:54:03ZengMDPI AGPharmaceutics1999-49232024-10-011611136710.3390/pharmaceutics16111367Advanced Nanotechnology-Based Nucleic Acid MedicinesNoriko Miyamoto0Mina Sakuragi1Yukio Kitade2Department of Applied Chemistry, Faculty of Engineering, Aichi Institute of Technology, 1247 Yachigusa, Yakusa-cho, Toyota 470-0392, JapanDepartment of Nanoscience, Faculty of Engineering, Sojo University, 4-22-1, Ikeda, Nishi, Kumamoto 860-0082, JapanDepartment of Applied Chemistry, Faculty of Engineering, Aichi Institute of Technology, 1247 Yachigusa, Yakusa-cho, Toyota 470-0392, JapanNucleic acid medicines are a highly attractive modality that act in a sequence-specific manner on target molecules. To date, 21 such products have been approved by the Food and Drug Administration. However, the development of nucleic acid medicines continues to face various challenges, including tissue and cell targeting as well as intracellular delivery. Numerous research groups are addressing these issues by advancing the development of nucleic acid medicines through nanotechnology. In countries other than Japan (including Europe and the USA), >40 nanotechnology-based nucleic acid medicines have been tested in clinical trials, and 15 clinical trials are ongoing. In Japan, three phase I trials are ongoing, and future results are awaited. The review summarizes the latest research in the nanotechnology of nucleic acid medicines and statuses of clinical trials in Japan, with expectations of further evolutions.https://www.mdpi.com/1999-4923/16/11/1367nucleic acid medicinesiRNAmiRNAmRNADNAASO
spellingShingle Noriko Miyamoto
Mina Sakuragi
Yukio Kitade
Advanced Nanotechnology-Based Nucleic Acid Medicines
Pharmaceutics
nucleic acid medicine
siRNA
miRNA
mRNA
DNA
ASO
title Advanced Nanotechnology-Based Nucleic Acid Medicines
title_full Advanced Nanotechnology-Based Nucleic Acid Medicines
title_fullStr Advanced Nanotechnology-Based Nucleic Acid Medicines
title_full_unstemmed Advanced Nanotechnology-Based Nucleic Acid Medicines
title_short Advanced Nanotechnology-Based Nucleic Acid Medicines
title_sort advanced nanotechnology based nucleic acid medicines
topic nucleic acid medicine
siRNA
miRNA
mRNA
DNA
ASO
url https://www.mdpi.com/1999-4923/16/11/1367
work_keys_str_mv AT norikomiyamoto advancednanotechnologybasednucleicacidmedicines
AT minasakuragi advancednanotechnologybasednucleicacidmedicines
AT yukiokitade advancednanotechnologybasednucleicacidmedicines