ArticleProceedings of the National Academy of Sciences of the United States of America2010
Tailor-made RNAi knockdown against triplet repeat disease-causing alleles.
Article in Proceedings of the National Academy of Sciences of the United States of America, 2010. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 18 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
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
18 citing papers in PubMed, 26 citations in OpenAlex.
- A chimeric RNA consisting of siRNA and aptamer for inhibiting dengue virus replication.NAR molecular medicine · 2024Article
- Development of versatile allele-specific siRNAs able to silence all the dominant dynamin 2 mutations.Molecular therapy. Nucleic acids · 2022Article
- Targeting N-Terminal Huntingtin with a Dual-sgRNA Strategy by CRISPR/Cas9.BioMed research international · 2019Article
- Supplemental Treatment for Huntington's Disease with miR-132 that Is Deficient in Huntington's Disease Brain.Molecular therapy. Nucleic acids · 2018Article
- Protein Misfolding and Aggregation as a Therapeutic Target for Polyglutamine Diseases.Brain sciences · 2017Review
- A novel measurement of allele discrimination for assessment of allele-specific silencing by RNA interference.Molecular biology reports · 2014Article
- Allele-specific Gene Silencing of Mutant mRNA Restores Cellular Function in Ullrich Congenital Muscular Dystrophy Fibroblasts.Molecular therapy. Nucleic acids · 2014Article
- Oligonucleotide-based strategies to combat polyglutamine diseases.Nucleic acids research · 2014Review
- Allele-specific RNA interference rescues the long-QT syndrome phenotype in human-induced pluripotency stem cell cardiomyocytes.European heart journal · 2014Article
- Research progress in allele-specific expression and its regulatory mechanisms.Journal of applied genetics · 2013Review
- 5' Unlocked Nucleic Acid Modification Improves siRNA Targeting.Molecular therapy. Nucleic acids · 2013Article
- Disease-causing allele-specific silencing by RNA interference.Pharmaceuticals (Basel, Switzerland) · 2013Article
- Specific inhibition of tumor cells by oncogenic EGFR specific silencing by RNA interference.PloS one · 2013Article
- Disease-causing allele-specific silencing against the ALK2 mutants, R206H and G356D, in fibrodysplasia ossificans progressiva.Gene therapy · 2012Article
- Allele-specific RNA interference in FOP silencing the FOP gene.Gene therapy · 2012Article
- An evaluation of oligonucleotide-based therapeutic strategies for polyQ diseases.BMC molecular biology · 2012Article
- Reduction of type IV collagen by upregulated miR-29 in normal elderly mouse and klotho-deficient, senescence-model mouse.PloS one · 2012Article
- Potent and selective antisense oligonucleotides targeting single-nucleotide polymorphisms in the Huntington disease gene / allele-specific silencing of mutant huntingtin.Molecular therapy : the journal of the American Society of Gene Therapy · 2011Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
7 authors at 3 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Nucleotide variations, including SNPs, in the coding regions of disease genes are important targets for RNAi treatment, which is a promising medical treatment for intractable diseases such as triplet repeat diseases. However, the identification of such nucleotide variations and the design of siRNAs conferring disease allele-specific RNAi are quite difficult. In this study we developed a pull-down method to rapidly identify coding SNP (cSNP) haplotypes of triple repeat, disease-causing alleles, and we demonstrated disease allele-specific RNAi that targeted cSNP sites in mutant Huntingtin alleles, each of which possessed a different cSNP haplotype. Therefore, the methods presented here allow for allele-specific RNAi knockdown against disease-causing alleles by using siRNAs specific to disease-linked cSNP haplotypes, and advanced progress toward tailor-made RNAi treatments for triplet repeat diseases.
Indexed as
Identifiers
What OpenQuestion holds
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.