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History of Research on Antisense Oligonucleotide Analogs
Substantia, 2021 In the search for novel therapeutics, antisense oligonucleotide (ASO) analogs have been a major focus of research for over 40 years. They use the antisense strategy, namely they have a nucleic acid base sequence that is complementary to a portion of a ...
Jack S. Cohen
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RNA Interference-Guided Targeting of Hepatitis C Virus Replication with Antisense Locked Nucleic Acid-Based Oligonucleotides Containing 8-oxo-dG Modifications. [PDF]
PLoS ONE, 2015 The inhibitory potency of an antisense oligonucleotide depends critically on its design and the accessibility of its target site. Here, we used an RNA interference-guided approach to select antisense oligonucleotide target sites in the coding region of ...
Margit Mutso +10 more
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Challenges and future perspective of antisense therapy for spinal muscular atrophy: A review
European Journal of Cell Biology, 2023 Spinal muscular atrophy (SMA), the most common genetic cause of infantile death, is caused by a mutation in the survival of motor neuron 1 gene (SMN1), leading to the death of motor neurons and progressive muscle weakness.
Zorica Nakevska, Toshifumi Yokota
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Pharmaceutics, 2023 The aim of this study was to develop antisense oligonucleotide tablet formulations using high-speed electrospinning. Hydroxypropyl-beta-cyclodextrin (HPβCD) was used as a stabilizer and as an electrospinning matrix. In order to optimize the morphology of
Edit Hirsch +13 more
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Molecular Therapy: Nucleic Acids, 2018 Duchenne muscular dystrophy (DMD), the most common lethal heritable childhood disease, is caused by mutations in the DMD gene that result in the absence of functional dystrophin protein.
Naoki Watanabe +8 more
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Regulation of metabolism by miR-378 [PDF]
, 2014 The present invention provides a method of regulating fatty acid metabolism in a cell by contacting the cell with a modulator of miR-378 and/or miR-378* activity or expression.
Carrer, Michele, Olson, Eric N.
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Locked nucleic acid: tighter is different [PDF]
, 2013 This viewpoint briefly reviews the impact of Locked Nucleic Acid (LNA) oligonucleotides, first described in a ChemComm paper in 1998. A number of unique applications in oligonucleotide biotechnology have been made possible by the high binding affinity ...
Watts, Jonathan
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Preparation of 5'-O-(1-Thiotriphosphate)-modified oligonucleotides using polymerase-endonuclease amplification reaction (PEAR). [PDF]
PLoS ONE, 2013 Antisense oligonucleotides (ASODNs) have been widely used as an important tool for regulating gene expression, and developed into therapeutics. Natural ODNs are susceptible to nuclease degradation, nucleic acid analogues, however, have less side effects,
Biao Li +7 more
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Effect of Terminal Groups of Dendrimers in the Complexation with Antisense Oligonucleotides and Cell Uptake [PDF]
, 2016 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
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Molecular Therapy: Nucleic Acids, 2019 Antisense oligonucleotide therapy has been reported to be associated with renal injury. Here, the mechanism of reversible proteinuria was investigated by combining clinical, pre-clinical, and in vitro data.
Manoe J. Janssen +10 more
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