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Published in 2023 at "Advanced materials"
DOI: 10.1002/adma.202300477
Abstract: Current vehicles used to deliver antisense oligonucleotides (ASOs) cannot distinguish between bacterial and mammalian cells, greatly hindering the preclinical or clinical treatment of bacterial infections, especially those caused by antibiotic-resistant bacteria. Herein, we leverage the…
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Keywords:
atp binding;
antisense oligonucleotides;
binding cassette;
antibiotic resistant ... See more keywords
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1
Published in 2023 at "Biotechnology progress"
DOI: 10.1002/btpr.3350
Abstract: Ultrafiltration/diafiltration (UF/DF) has been the hallmark for concentrating and buffer exchange of protein and peptide-based therapeutics for years. Here we examine the capabilities and limitations of UF/DF membranes to process oligonucleotides using antisense oligonucleotides (ASOs)…
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Keywords:
diafiltration;
use ultrafiltration;
antisense oligonucleotides;
processing antisense ... See more keywords
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1
Published in 2022 at "ChemBioChem"
DOI: 10.1002/cbic.202200168
Abstract: We analyzed the effect of modified nucleotides within gapmer antisense oligonucleotides on RNase H mediated gene silencing. Additionally, short hairpins were introduced into antisense oligonucleotides as structural motifs, and their influence on biological and physicochemical…
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Keywords:
knock;
antisense oligonucleotides;
gene;
gapmer antisense ... See more keywords
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1
Published in 2019 at "Human Mutation"
DOI: 10.1002/humu.23774
Abstract: One of most important factors for messenger RNA (mRNA) transcription is the spliceosomal component U1 small nuclear RNA (snRNA), which recognizes 5′ splicing donor sites at specific regions in pre‐mRNA. Mutations in these sites disrupt…
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Keywords:
modified snrna;
antisense oligonucleotides;
hearing loss;
snrna antisense ... See more keywords
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Published in 2020 at "Methods in molecular biology"
DOI: 10.1007/978-1-0716-0771-8_2
Abstract: DNA-like molecules called antisense oligonucleotides have opened new treatment possibilities for genetic diseases by offering a method of regulating gene expression. Antisense oligonucleotides are often used to suppress the expression of mutated genes which may…
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Keywords:
treatment;
chemistry;
antisense oligonucleotide;
antisense oligonucleotides ... See more keywords
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Published in 2021 at "Methods in molecular biology"
DOI: 10.1007/978-1-0716-1158-6_17
Abstract: With the rapid revolution in RNA/DNA sequencing technologies, it is evident that mammalian genomes express tens of thousands of long noncoding RNAs (lncRNAs). Since a large majority of lncRNAs have been functionally implicated in cancer…
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Keywords:
administration;
vivo administration;
therapeutic antisense;
chemistry ... See more keywords
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1
Published in 2018 at "Methods in molecular biology"
DOI: 10.1007/978-1-4939-7522-8_5
Abstract: Antisense oligonucleotides (AONs) are small molecules able to bind to the pre-mRNA and modulate splicing. The increasing amount of intronic mutations leading to pseudoexon insertion in genes underlying inherited retinal dystrophies (IRDs) has highlighted the…
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Keywords:
correct pre;
retinal dystrophies;
mrna splicing;
pre mrna ... See more keywords
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Published in 2017 at "Advances in experimental medicine and biology"
DOI: 10.1007/978-981-10-4310-9_9
Abstract: A number of diseases are caused by low levels of key proteins; therefore, increasing the amount of specific proteins in human bodies is of therapeutic interest. Protein expression is downregulated by some structural or sequence…
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Keywords:
protein;
increase;
translation;
protein levels ... See more keywords
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Published in 2021 at "Cell Reports"
DOI: 10.1016/j.celrep.2021.110108
Abstract: Summary Activation of the pro-degenerative protein SARM1 after diverse physical and disease-relevant injuries causes programmed axon degeneration. Original studies indicate that substantially decreased SARM1 levels are required for neuroprotection. However, we demonstrate, in Sarm1 haploinsufficient…
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Keywords:
programmed axon;
degeneration;
axon degeneration;
sarm1 levels ... See more keywords
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Published in 2017 at "Journal of Drug Delivery Science and Technology"
DOI: 10.1016/j.jddst.2016.12.001
Abstract: Abstract Treatment of cancer is a furtive problem in the current era in spite of significant advancement in drug delivery systems and identification of new therapeutic molecules. The ration of lab to market translation of…
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Keywords:
containing antisense;
cancer;
targeting nanocarriers;
cell ... See more keywords
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1
Published in 2022 at "Bioconjugate chemistry"
DOI: 10.1021/acs.bioconjchem.2c00379
Abstract: Antisense oligonucleotides (ASOs) are an important emerging therapeutic; however, they struggle to enter cells without a delivery vehicle, such as a cationic polymer. To understand the role of polymer architecture for ASO delivery, five linear…
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Keywords:
polymer;
polymer architecture;
antisense oligonucleotides;
delivery ... See more keywords