Results 271 to 280 of about 732,131 (343)
Advances in oligonucleotide drug delivery
Nature Reviews Drug Discovery, 2020 Oligonucleotides can be used to modulate gene expression via a range of processes including RNAi, target degradation by RNase H-mediated cleavage, splicing modulation, non-coding RNA inhibition, gene activation and programmed gene editing. As such, these
Thomas C Roberts +2 more
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Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS
New England Journal of Medicine, 2022 BACKGROUND The intrathecally administered antisense oligonucleotide tofersen reduces synthesis of the superoxide dismutase 1 (SOD1) protein and is being studied in patients with amyotrophic lateral sclerosis (ALS) associated with mutations in SOD1 (SOD1 ...
Gen Sobue +2 more
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The chemical evolution of oligonucleotide therapies of clinical utility
Nature Biotechnology, 2017 Anastasia Khvorova, Jonathan K Watts
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Nephron Experimental Nephrology, 1998
Antisense technology was developed to inhibit gene expression by utilizing an oligonucleotide complementary to the mRNA which encodes the target gene. There are a few possible mechanisms for the inhibitory effects of antisense oligonucleotides. Among them, degradation of mRNA by RNase H is considered to be the major mechanism of action for antisense ...
N, Kashihara, Y, Maeshima, H, Makino
openaire +2 more sources
Antisense technology was developed to inhibit gene expression by utilizing an oligonucleotide complementary to the mRNA which encodes the target gene. There are a few possible mechanisms for the inhibitory effects of antisense oligonucleotides. Among them, degradation of mRNA by RNase H is considered to be the major mechanism of action for antisense ...
N, Kashihara, Y, Maeshima, H, Makino
openaire +2 more sources
Expert Opinion on Drug Discovery, 2008
Numerous oligonucleotide-based biopharmaceuticals have been tested to suppress disease progression. These potent therapeutics include plasmids containing transgenes, antisense oligonucleotides, ribozymes, DNAzymes, decoys, aptamers and small interfering RNAs.
Yoshitaka, Isaka +3 more
openaire +2 more sources
Numerous oligonucleotide-based biopharmaceuticals have been tested to suppress disease progression. These potent therapeutics include plasmids containing transgenes, antisense oligonucleotides, ribozymes, DNAzymes, decoys, aptamers and small interfering RNAs.
Yoshitaka, Isaka +3 more
openaire +2 more sources