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Antisense oligonucleotides complementary to RNA targets promise generality and ease of drug design. The first systemically administered antisense drug was recently approved for treatment and others are in clinical development. Chemical modifications that
Lykke Pedersen+3 more
doaj +1 more source
Mutations in the human ether-a-go-go-related gene (hERG) cause long-QT syndrome type 2 (LQT2). We previously described a homozygous LQT2 nonsense mutation Q1070X in which the mutant mRNA is degraded by nonsense-mediated mRNA decay (NMD) leading to a ...
Bhuiyan+39 more
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Selection of prebiotic oligonucleotides by cyclic phase separation [PDF]
The emergence of functional oligonucleotides on early Earth required a molecular selection mechanism to screen for specific sequences with prebiotic functions. Cyclic processes such as daily temperature oscillations were ubiquitous in this environment and could trigger oligonucleotide phase separation. Here, we propose sequence selection based on phase
arxiv
Antisense Oligonucleotides: Concepts and Pharmaceutical Applications
Antisense oligonucleotides are drugs whose mechanism is based on binding to RNA target sequences. For this purpose, they modify the protein expression through steric hindrance and exon omission.
Ariana Araya+5 more
doaj +1 more source
Resveratrol mediated modulation of Sirt-1/Runx2 promotes osteogenic differentiation of mesenchymal stem cells: potential role of Runx2 deacetylation. [PDF]
Osteogenic repair in response to bone injury is characterized by activation and differentiation of mesenchymal stem cells (MSCs) to osteoblasts.
Aldinger, Constance+6 more
core +9 more sources
Liver and kidney uptake and antisense activity is studied for a series of Locked Nucleic Acid (LNA) oligonucleotides with fully stereo-defined, internucleoside linkages.
Henrik Frydenlund Hansen+5 more
doaj +1 more source
Silencing Antibiotic Resistance with Antisense Oligonucleotides
Antisense technologies consist of the utilization of oligonucleotides or oligonucleotide analogs to interfere with undesirable biological processes, commonly through inhibition of expression of selected genes.
Saumya Jani+2 more
doaj +1 more source
Targeted delivery of antisense oligonucleotides to pancreatic β-cells
Receptor-dependent productive uptake of GLP1-conjugated antisense oligonucleotides occurs selectively in pancreatic β-cells. Antisense oligonucleotide (ASO) silencing of the expression of disease-associated genes is an attractive novel therapeutic ...
Carina Ämmälä+19 more
semanticscholar +1 more source
Silencing disease genes in the laboratory and the clinic
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
Corey, David R., Watts, Jonathan K.
core +1 more source
Effect of Terminal Groups of Dendrimers in the Complexation with Antisense Oligonucleotides and Cell Uptake [PDF]
Indexación: Web of Science.Poly(amidoamine) dendrimers are the most recognized class of dendrimer. Amino-terminated (PAMAM-NH2) and hydroxyl-terminated (PAMAM-OH) dendrimers of generation 4 are widely used, since they are commercially available.
Araya-Durán, Ingrid+10 more
core +1 more source