4.5 Review

Nonviral delivery systems for antisense oligonucleotide therapeutics

Journal

BIOMATERIALS RESEARCH
Volume 26, Issue 1, Pages -

Publisher

SPRINGERNATURE
DOI: 10.1186/s40824-022-00292-4

Keywords

Antisense oligonucleotides; Nonviral delivery; Gene drugs; Nanoparticles

Funding

  1. Research funds of Natural Science Foundation of Hunan Province [2020JJ9009]
  2. Hunan Provincial Science and Technology Plan [2016TP2002]

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In this article, a review of current nonviral delivery strategies for antisense oligonucleotides (ASOs) is provided, including modifications, action mechanisms, and multi-carrier methods. The irreplaceable role of nonviral vectors in the progressive development of ASOs delivery is emphasized.
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.

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