ArticleBiological chemistry2011
Allele-selective inhibition of ataxin-3 (ATX3) expression by antisense oligomers and duplex RNAs.
Article in Biological chemistry, 2011. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 30 papers.
What it found
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Who cites it
30 citing papers in PubMed.
- Unlocking the potential of chemically modified peptide nucleic acids for RNA-based therapeutics.RNA (New York, N.Y.) · 2023Review
- Towards Personalized Allele-Specific Antisense Oligonucleotide Therapies for Toxic Gain-of-Function Neurodegenerative Diseases.Pharmaceutics · 2022Review
- Artificial miRNAs targeting CAG repeat expansion in ORFs cause rapid deadenylation and translation inhibition of mutant transcripts.Cellular and molecular life sciences : CMLS · 2021Article
- Toward allele-specific targeting therapy and pharmacodynamic marker for spinocerebellar ataxia type 3.Science translational medicine · 2020Article
- Pathogenic mechanisms underlying spinocerebellar ataxia type 1.Cellular and molecular life sciences : CMLS · 2020Review
- Limits of using oligonucleotides for allele-selective inhibition at trinucleotide repeat sequences - targeting the CAG repeat within ataxin-1.Nucleosides, nucleotides & nucleic acids · 2020Article
- Antisense oligonucleotides: A primer.Neurology. Genetics · 2019Review
- Genetics, Mechanisms, and Therapeutic Progress in Polyglutamine Spinocerebellar Ataxias.Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics · 2019Review
- Evaluation of Antisense Oligonucleotides Targeting ATXN3 in SCA3 Mouse Models.Molecular therapy. Nucleic acids · 2017Article
- Article
- miR-25 alleviates polyQ-mediated cytotoxicity by silencing ATXN3.FEBS letters · 2014Article
- Allele-specific silencing of mutant Ataxin-7 in SCA7 patient-derived fibroblasts.European journal of human genetics : EJHG · 2014Article
- Allele-selective inhibition of mutant atrophin-1 expression by duplex and single-stranded RNAs.Biochemistry · 2014Article
- Structural studies of CNG repeats.Nucleic acids research · 2014Review
- Ataxin-3 protein and RNA toxicity in spinocerebellar ataxia type 3: current insights and emerging therapeutic strategies.Molecular neurobiology · 2014Review
- The panorama of miRNA-mediated mechanisms in mammalian cells.Cellular and molecular life sciences : CMLS · 2014Review
- Oligonucleotide-based strategies to combat polyglutamine diseases.Nucleic acids research · 2014Review
- Consensus paper: pathological mechanisms underlying neurodegeneration in spinocerebellar ataxias.Cerebellum (London, England) · 2014Article
- Allele-selective inhibition of expression of huntingtin and ataxin-3 by RNA duplexes containing unlocked nucleic acid substitutions.Biochemistry · 2013Article
- Self-duplexing CUG repeats selectively inhibit mutant huntingtin expression.Nucleic acids research · 2013Article
Corrections and comments
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Authors and funding
10 authors.
Funding
Abstract
Spinocerebellar ataxia-3 (also known as Machado-Joseph disease) is an incurable neurodegenerative disorder caused by expression of a mutant variant of ataxin-3 (ATX3) protein. Inhibiting expression of ATX3 would provide a therapeutic strategy, but indiscriminant inhibition of both wild-type and mutant ATX3 might lead to undesirable side effects. An ideal silencing agent would block expression of mutant ATX3 while leaving expression of wild-type ATX3 intact. We have previously observed that peptide nucleic acid (PNA) conjugates targeting the expanded CAG repeat within ATX3 mRNA block expression of both alleles. We have now identified additional PNAs capable of inhibiting ATX3 expression that vary in length and in the nature of the conjugated cation chain. We can also achieve potent and selective inhibition using duplex RNAs containing one or more mismatches relative to the CAG repeat. Anti-CAG antisense bridged nucleic acid oligonucleotides that lack a cationic domain are potent inhibitors but are not allele-selective. Allele-selective inhibitors of ATX3 expression provide insights into the mechanism of selectivity and promising lead compounds for further development and in vivo investigation.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.