A Tandem Oligonucleotide Approach for SNP-Selective RNA Degradation Using Modified Antisense Oligonucleotides. [PDF]
PLoS ONE, 2015 Antisense oligonucleotides have been studied for many years as a tool for gene silencing. One of the most difficult cases of selective RNA silencing involves the alleles of single nucleotide polymorphisms, in which the allele sequence is differentiated ...
Dorota Magner +4 more
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Antisense Therapy for Infectious Diseases
Cells, 2023 Infectious diseases, particularly Tuberculosis (TB) caused by Mycobacterium tuberculosis, pose a significant global health challenge, with 1.6 million reported deaths in 2021, making it the most fatal disease caused by a single infectious agent. The rise
Lwanda Abonga Buthelezi +4 more
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Antisense oligonucleotide therapeutic approach for Timothy syndrome
NatureTimothy syndrome (TS) is a severe, multisystem disorder characterized by autism, epilepsy, long-QT syndrome and other neuropsychiatric conditions1. TS type 1 (TS1) is caused by a gain-of-function variant in the alternatively spliced and developmentally ...
Xiaoyu Chen +15 more
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Antisense Oligonucleotide Reverses Leukodystrophy in Canavan Disease Mice
Annals of Neurology, 2020 Marked elevation in the brain concentration of N‐acetyl‐L‐aspartate (NAA) is a characteristic feature of Canavan disease, a vacuolar leukodystrophy resulting from deficiency of the oligodendroglial NAA‐cleaving enzyme aspartoacylase.
V. Hull +7 more
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CyclinD1 antisense oligonucleotide induces apoptosis of lung adenocarcinoma cell A549
, 2020 To investigate the role of CyclinD1 antisense oligonucleotide in lung cancer gene therapy, an expression vector containing CyclinD1 antisense oligonucleotide, named pcDNA3.1-CyclinD1, was designed, and this vector was transfected into lung adenocarcinoma
Tahama Sharma
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Antisense oligonucleotide based therapeutics and its applications against bacterial infections
Medicine in Drug Discovery, 2023 The transcriptome of a cell governs the overall cellular expression of proteins, hence, targeting mRNA holds promise as a therapeutic strategy in the field of drug discovery.
Nupur Angrish, Garima Khare
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Silencing disease genes in the laboratory and the clinic
, 2011 Synthetic nucleic acids are commonly used laboratory tools for modulating gene expression and have the potential to be widely used in the clinic. Progress towards nucleic acid drugs, however, has been slow and many challenges remain to be overcome before
Jonathan K Watts +3 more
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Antisense Oligonucleotide Therapies for Neurodegenerative Diseases
Annual Review of Neuroscience, 2019 Antisense oligonucleotides represent a novel therapeutic platform for the discovery of medicines that have the potential to treat most neurodegenerative diseases.
C. Bennett, A. Krainer, D. Cleveland
semanticscholar +1 more source
Nusinersen: A Novel Antisense Oligonucleotide for the Treatment of Spinal Muscular Atrophy.
The Journal of Pediatric Pharmacology and Therapeutics, 2019 Spinal muscular atrophy (SMA) encompasses a group of autosomal recessively inherited degenerative neuromuscular disorders. They range in severity from neonatal onset with rapidly progressive weakness and early mortality (SMA-1), to onset in infancy (SMA ...
E. Neil, E. Bisaccia
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CD4 expression decrease by antisense oligonucleotides. Inhibition of rat T CD4+ cell reactivity
, 2012 In previous studies, we have demonstrated the inhibition of CD4 expression in rat lymphocytes treated with phorbol myristate acetate (PMA) by antisense oligonucleotides (AS-ODNs) directed against the AUG start region of the cd4 gene.
Franch i Masferrer, Àngels +6 more
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