ArticleAnnals of the New York Academy of Sciences2009
Allele-selective inhibition of mutant huntingtin by peptide nucleic acid-peptide conjugates, locked nucleic acid, and small interfering RNA.
Article in Annals of the New York Academy of Sciences, 2009. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 33 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
33 citing papers in PubMed, 53 citations in OpenAlex.
- Targeting Oncogenic KRAS Using Peptide Nucleic Acid Oligomers Attached to Cell-Penetrating Peptides.International journal of molecular sciences · 2026Article
- Decanoic Acid Treatment Alleviates Non-cell Autonomous Transfer of HD Pathology by Secretome of Mutant Huntingtin Expressing Cells.Neurochemical research · 2026Article
- Unlocking the neuroprotective potential of peptide nucleic acids 5 (PNA5) in neurological diseases: molecular mechanisms to therapeutic approaches.Metabolic brain disease · 2025Review
- Enhancing RNA inhibitory activity using clamp-G-modified nucleobases.Cell reports. Physical science · 2024Article
- Sequencing-guided design of genetically encoded small RNAs targeting CAG repeats for selective inhibition of mutantMolecular therapy. Nucleic acids · 2024Article
- CRISPR/Cas9 Mediated Therapeutic Approach in Huntington's Disease.Molecular neurobiology · 2023Review
- Therapeutic Advances for Huntington's Disease.Brain sciences · 2020Review
- Combination of stem cell and gene therapy ameliorates symptoms in Huntington's disease mice.NPJ Regenerative medicine · 2019Article
- New directions in therapeutics for Huntington disease.Future neurology · 2018Review
- Gene suppression approaches to neurodegeneration.Alzheimer's research & therapy · 2017Review
- Influence of mismatched and bulged nucleotides on SNP-preferential RNase H cleavage of RNA-antisense gapmer heteroduplexes.Scientific reports · 2017Article
- Huntington's Disease-Update on Treatments.Current neurology and neuroscience reports · 2017Review
- Targeting CAG repeat RNAs reduces Huntington's disease phenotype independently of huntingtin levels.The Journal of clinical investigation · 2016Article
- Gene suppression strategies for dominantly inherited neurodegenerative diseases: lessons from Huntington's disease and spinocerebellar ataxia.Human molecular genetics · 2016Review
- The first crystal structures of RNA-PNA duplexes and a PNA-PNA duplex containing mismatches--toward anti-sense therapy against TREDs.Nucleic acids research · 2016Article
- Phosphorodiamidate morpholino oligomers suppress mutant huntingtin expression and attenuate neurotoxicity.Human molecular genetics · 2014Article
- Oligonucleotide-based strategies to combat polyglutamine diseases.Nucleic acids research · 2014Review
- Article
- Small non-coding RNAs add complexity to the RNA pathogenic mechanisms in trinucleotide repeat expansion diseases.Frontiers in molecular neuroscience · 2013Review
- Self-duplexing CUG repeats selectively inhibit mutant huntingtin expression.Nucleic acids research · 2013Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
3 authors at 1 institution in 1 country.
Funding
Abstract
The ability to inhibit expression of a mutant allele while retaining expression of a wild-type protein might provide a useful approach to treating Huntington's Disease (HD) and other inherited pathologies. The mutant form of huntingtin (HTT), the protein responsible for HD, is encoded by an mRNA containing an expanded CAG repeat. We demonstrate that peptide nucleic acid conjugates and locked nucleic acids complementary to the CAG repeat selectively block expression of mutant HTT. The selectivity of inhibition is at least as good as that shown by a small interfering RNA targeted to a deletion polymorphism. Our data suggest that antisense oligomers are promising subjects for further development as an anti-HD therapeutic strategy.
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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.