Improved targeting of miRNA with antisense oligonucleotides

Improved targeting of miRNA with antisense oligonucleotides

MicroRNAs (miRNAs) are a class of 20-24 nt noncoding RNAs that regulate target mRNAs post-transcriptionally by binding with imperfect complementarity in the 3'-untranslated region (3'-UTR) and inhibiting translation or RNA stability. Current understanding of miRNA biology is limited, and antisense oligonucleotide (ASO) inhibition is a powerful technique for miRNA functionalization in vitro and ...

Antisense Oligonucleotides Targeting Protein

Targeting Kupffer cells with antisense oligonucleotides.

During proinflammatory reactions such as endotoxemia, ischemia-reperfusion and immune reactions, excessive amounts of cytokines and prostanoids are released resulting in liver injury. In the liver, Kupffer cells are the primary source of cytokines and prostanoids. Obliteration of Kupffer cells prevents experimentally-induced liver damage, suggesting a major role for Kupffer in the pathogenesis ...

MicroRNA fate upon targeting with anti-miRNA oligonucleotides as revealed by an improved Northern-blot-based method for miRNA detection.

MicroRNAs (miRNAs) are small non-coding RNAs involved in fine-tuning of gene regulation. Antisense oligonucleotides (ONs) are promising tools as anti-miRNA (anti-miR) agents toward therapeutic applications and to uncover miRNA function. Such anti-miR ONs include 2'-O-methyl (OMe), cationic peptide nucleic acids like K-PNA-K3, and locked nucleic acid (LNA)-based anti-miRs such as LNA/DNA or LNA/...

Evaluation of Antisense Oligonucleotides Targeting ATXN3 in SCA3 Mouse Models

The most common dominantly inherited ataxia, spinocerebellar ataxia type 3 (SCA3), is an incurable neurodegenerative disorder caused by a CAG repeat expansion in the ATXN3 gene that encodes an abnormally long polyglutamine tract in the disease protein, ATXN3. Mice lacking ATXN3 are phenotypically normal; hence, disease gene suppression offers a compelling approach to slow the neurodegenerative ...