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Review
. 2021 Mar 24;22(7):3295.
doi: 10.3390/ijms22073295.

Recent Advances in Oligonucleotide Therapeutics in Oncology

Haoyu Xiong  1 , Rakesh N Veedu  2   3 , Sarah D Diermeier  1
Affiliations
Review

Recent Advances in Oligonucleotide Therapeutics in Oncology

Haoyu Xiong et al. Int J Mol Sci. .

Abstract

Cancer is one of the leading causes of death worldwide. Conventional therapies, including surgery, radiation, and chemotherapy have achieved increased survival rates for many types of cancer over the past decades. However, cancer recurrence and/or metastasis to distant organs remain major challenges, resulting in a large, unmet clinical need. Oligonucleotide therapeutics, which include antisense oligonucleotides, small interfering RNAs, and aptamers, show promising clinical outcomes for disease indications such as Duchenne muscular dystrophy, familial amyloid neuropathies, and macular degeneration. While no approved oligonucleotide drug currently exists for any type of cancer, results obtained in preclinical studies and clinical trials are encouraging. Here, we provide an overview of recent developments in the field of oligonucleotide therapeutics in oncology, review current clinical trials, and discuss associated challenges.

Keywords: DNAzymes; antisense oligonucleotides; aptamers; cancers; siRNA.

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Conflict of interest statement

The authors declare no conflict of interest.

Figures

Figure 1
Figure 1
Summary of clinical trials for oligonucleotide therapeutics in oncology. (a) Numbers of trials for different types of oligonucleotide therapeutics; (b) Trials status. Number of trials were retrieved from Supplementary Materials.
Figure 2
Figure 2
Summary of mechanisms of different types of oligonucleotide therapeutics. ASOs inhibit protein expression by sterically blocking translation (A) or RNase H dependent RNA degradation (B), or alter splicing (C); siRNAs degrade mRNA through RISC (D); miR mimetics degrade mRNA through RISC (E); antagomiRs block endogenous miRNA (F); DNAzymes specifically bind and cleave RNA (G); aptamers block cell receptors (H), block interactions between proteins (I), or act as drug delivery vectors (J). RISC, RNA-induced silencing complex. ASOs, aptamers and DNAzymes can also be delivered through vectors and cell endocytosis (not shown in the figure). Figure created with BioRender.com. Adapted from https://app.biorender.com/biorender-templates (accessed on 15 March 2021).
Figure 3
Figure 3
Overview of different chemical modification of antisense oligonucleotides.
See this image and copyright information in PMC

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