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Review
. 2020 Mar 6:19:581-601.
doi: 10.1016/j.omtn.2019.12.004. Epub 2019 Dec 17.

Improved Nucleic Acid Therapy with Advanced Nanoscale Biotechnology

Yuhua Weng  1 , Qianqian Huang  2 , Chunhui Li  1 , Yongfeng Yang  3 , Xiaoxia Wang  4 , Jie Yu  3 , Yuanyu Huang  5 , Xing-Jie Liang  6
Affiliations
Review

Improved Nucleic Acid Therapy with Advanced Nanoscale Biotechnology

Yuhua Weng et al. Mol Ther Nucleic Acids. .

Abstract

Due to a series of systemic and intracellular obstacles in nucleic acid (NA) therapy, including fast degradation in blood, renal clearance, poor cellular uptake, and inefficient endosomal escape, NAs may need delivery methods to transport to the cell nucleus or cytosol to be effective. Advanced nanoscale biotechnology-associated strategies, such as controlling the particle size, charge, drug loading, response to environmental signals, or other physical/chemical properties of delivery carriers, have provided great help for the in vivo and in vitro delivery of NA therapeutics. In this review, we introduce the characteristics of different NA modalities and illustrate how advanced nanoscale biotechnology assists NA therapy. The specific features and challenges of various nanocarriers in clinical and preclinical studies are summarized and discussed. With the help of advanced nanoscale biotechnology, some of the major barriers to the development of NA therapy will eventually be overcome in the near future.

Keywords: ASO; CRISPR/Cas; RNAi; drug delivery; gene editing; nucleic acid therapy.

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Figures

Figure 1
Figure 1
Challenges and Strategies for Efficient Intracellular NA Delivery Reproduced from Lostalé-Seijo et al. with permission.
Figure 2
Figure 2
Scheme of Representative Nanocarriers Used for NA Therapy (A) Liposome; (B) exosome; (C) bacterial-derived nanocell; (D) polymeric NP; (E) DNA nanostructure; (F) inorganic NP; (G) dendrimer-based NP; and (H) RNA nanostructure.
Figure 3
Figure 3
Structure of a pH-Sensitive Nanoformulation and the Schematic Illustration of In Vivo Prostate Cancer-Targeted RNAi Therapy Reproduced from Xu et al. with permission.
Figure 4
Figure 4
A Light-Responsive Nanostructure for Efficient siRNA Delivery and Sensitizing Photothermal Therapy Reproduced from Wang et al. with permission.
See this image and copyright information in PMC

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