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Published in 2023 at "Analytical chemistry"
DOI: 10.1021/acs.analchem.2c04902
Abstract: siRNA therapeutics provide a selective and powerful approach to reduce the expression of disease-causing genes. For regulatory approval, these modalities require sequence confirmation which is typically achieved by intact tandem mass spectrometry sequencing. However, this…
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Keywords:
rna sequencing;
bottom;
sequence;
sirna therapeutics ... See more keywords
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1
Published in 2022 at "RNA Biology"
DOI: 10.1080/15476286.2022.2052641
Abstract: ABSTRACT Nucleic acid-based therapeutics that control gene expression have been steadily progressing towards achieving their full clinical potential throughout the last few decades. Rapid progress has been achieved in RNAi-based therapy by optimizing high specificity…
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Keywords:
effect;
dual ribose;
ribose modifications;
sirna ... See more keywords
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1
Published in 2022 at "Expert Review of Clinical Pharmacology"
DOI: 10.1080/17512433.2022.2136166
Abstract: ABSTRACT Introduction Small interfering RNA (siRNA) has emerged as a powerful tool for post-transcriptional downregulation of multiple genes for various therapies. Naked siRNA molecules are surrounded by several barriers that tackle their optimum delivery to…
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Keywords:
clinical pharmacology;
pharmacology sirna;
pharmacology;
sirna ... See more keywords
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1
Published in 2022 at "Nucleic Acids Research"
DOI: 10.1093/nar/gkac456
Abstract: Abstract At the time of writing, although siRNA therapeutics are approved for human use, no official regulatory guidance specific to this modality is available. In the absence of guidance, preclinical development for siRNA followed a…
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Keywords:
plasma protein;
binding drug;
protein binding;
drug drug ... See more keywords