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Nonviral delivery systems for antisense oligonucleotide therapeutics

Authors :
Si Huang
Xin-Yan Hao
Yong-Jiang Li
Jun‑Yong Wu
Da-Xiong Xiang
Shilin Luo
Source :
Biomaterials Research, Vol 26, Iss 1, Pp 1-23 (2022)
Publication Year :
2022
Publisher :
American Association for the Advancement of Science (AAAS), 2022.

Abstract

Abstract Antisense oligonucleotides (ASOs) are an important tool for the treatment of many genetic disorders. However, similar to other gene drugs, vectors are often required to protect them from degradation and clearance, and to accomplish their transport in vivo. Compared with viral vectors, artificial nonviral nanoparticles have a variety of design, synthesis, and formulation possibilities that can be selected to accomplish protection and delivery for specific applications, and they have served critical therapeutic purposes in animal model research and clinical applications, allowing safe and efficient gene delivery processes into the target cells. We believe that as new ASO drugs develop, the exploration for corresponding nonviral vectors is inevitable. Intensive development of nonviral vectors with improved delivery strategies based on specific targets can continue to expand the value of ASO therapeutic approaches. Here, we provide an overview of current nonviral delivery strategies, including ASOs modifications, action mechanisms, and multi-carrier methods, which aim to address the irreplaceable role of nonviral vectors in the progressive development of ASOs delivery.

Details

Language :
English
ISSN :
20557124
Volume :
26
Issue :
1
Database :
Directory of Open Access Journals
Journal :
Biomaterials Research
Publication Type :
Academic Journal
Accession number :
edsdoj.5d02ddd4fb8422ba5cd0eef2ea0aa69
Document Type :
article
Full Text :
https://doi.org/10.1186/s40824-022-00292-4