2021
DOI: 10.3390/cancers13195002
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Nucleic Acid Drugs—Current Status, Issues, and Expectations for Exosomes

Abstract: Nucleic acid drugs are being developed as novel therapeutic modalities. They have great potential to treat human diseases such as cancers, viral infections, and genetic disorders due to unique characteristics that make it possible to approach undruggable targets using classical small molecule or protein/antibody-based biologics. In this review, I describe the advantages, classification, and clinical status of nucleic acid therapeutics. To date, more than 10 products have been launched, and many products have b… Show more

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Cited by 45 publications
(27 citation statements)
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References 143 publications
(156 reference statements)
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“…The expansion of drug-discovery modalities has been remarkable in recent years. In particular, nucleic-acid drugs can directly act on gene regulation [87][88][89]. Compared with conventional small-molecule drugs, biopharmacology makes it easier to design drugs that specifically act on various molecules.…”
Section: Micrornas In Ev-mediated Ddsmentioning
confidence: 99%
“…The expansion of drug-discovery modalities has been remarkable in recent years. In particular, nucleic-acid drugs can directly act on gene regulation [87][88][89]. Compared with conventional small-molecule drugs, biopharmacology makes it easier to design drugs that specifically act on various molecules.…”
Section: Micrornas In Ev-mediated Ddsmentioning
confidence: 99%
“…Under physiological and pathological conditions, exosomes can be actively secreted extracellularly by various cells including immune cells, stem cells and tumor cells, and can transport a large number of biomolecules from the parent cell to other cells, which is closely related to the pathogenesis of tumors ( Claridge et al, 2021 ; Oey et al, 2021 ; Yamada, 2021 ). Because of their small size, exosomes are able to avoid phagocytosis by mononuclear macrophages and cross the vascular wall to the extracellular matrix, they are widely present in various body fluids including blood, saliva, urine, cerebrospinal fluid, and thoracoabdominal fluid ( Clark et al, 2021 ; Leggio et al, 2021 ; Mao et al, 2021 ; Prieto-Vila et al, 2021 ).…”
Section: Detection Of Exosomesmentioning
confidence: 99%
“…Due to the arduous nature of the causation and phenotype of cancer, it is evident that nucleic acid-based therapeutics must implement a plethora of strategies via different modes of action to target relevant genes and their products in cancer cells or stimulate an immune response against them[ 78 ]. Table 3 summarizes the current status of nucleic acid nanomedicines available for cancer treatment.…”
Section: Nucleic Acidsmentioning
confidence: 99%
“…ASOs are synthetic oligonucleotides complementary to a gene of interest that bind on the pre-mRNA or mRNA of the target gene, hindering cellular post-transcriptional and translational machinery and eventually leading to altered splicing patterns or gene silencing, respectively[ 79 , 80 ]. Lipid nanoparticle (LNP)-based ASOs are under clinical evaluation to treat leukemia[ 78 , 81 ] and solid tumors[ 81 ] via targeting Grb2. Furthermore, targeting the anti-apoptotic gene Bcl-2 is also being examined as a possible target in patients with advanced lymphoid malignancies[ 82 ].…”
Section: Nucleic Acidsmentioning
confidence: 99%