ReviewGene therapy2006
RNAi: a potential therapy for the dominantly inherited nucleotide repeat diseases.
Review in Gene therapy, 2006. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 15 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.
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Who cites it
15 citing papers in PubMed, 40 citations in OpenAlex.
- Repeat-associated non-AUG (RAN) translation: insights from pathology.Laboratory investigation; a journal of technical methods and pathology · 2019Review
- New developments in RAN translation: insights from multiple diseases.Current opinion in genetics & development · 2017Review
- RNA interference-based therapeutics for inherited long QT syndrome.Experimental and therapeutic medicine · 2015Article
- Experimental models for identifying modifiers of polyglutamine-induced aggregation and neurodegeneration.Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics · 2013Article
- Triplet repeat RNA structure and its role as pathogenic agent and therapeutic target.Nucleic acids research · 2012Review
- Using non-coding small RNAs to develop therapies for Huntington's disease.Gene therapy · 2011Review
- RNAi: a potential new class of therapeutic for human genetic disease.Human genetics · 2011Review
- Allele-selective inhibition of ataxin-3 (ATX3) expression by antisense oligomers and duplex RNAs.Biological chemistry · 2011Article
- Allele-selective inhibition of mutant huntingtin by peptide nucleic acid-peptide conjugates, locked nucleic acid, and small interfering RNA.Annals of the New York Academy of Sciences · 2009Article
- Allele-specific silencing of mutant huntingtin and ataxin-3 genes by targeting expanded CAG repeats in mRNAs.Nature biotechnology · 2009Article
- Molecular Effects of the CTG Repeats in Mutant Dystrophia Myotonica Protein Kinase Gene.Current genomics · 2008Article
- Enhancement of allele discrimination by introduction of nucleotide mismatches into siRNA in allele-specific gene silencing by RNAi.PloS one · 2008Article
- Neuroprotection for Huntington's disease: ready, set, slow.Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics · 2008Review
- Modulating the expression of disease genes with RNA-based therapy.PLoS genetics · 2007Review
- Investigation of RNA interference to suppress expression of full-length and fragment human huntingtin.Neuromolecular medicine · 2007Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
2 authors at 2 institutions in 2 countries.
Funding
Abstract
Genetic diseases that are accompanied by central nervous system involvement are often fatal. Among these are the autosomal dominant neurogenetic diseases caused by nucleotide repeat expansion. For example, Huntington's disease (HD) and spinal cerebellar ataxia are caused by expansion of a tract of CAGs encoding glutamine. In HD and the other CAG-repeat expansion diseases, the expansion is in the coding region. Myotonic dystrophy is caused by repeat expansions of CUG or CCTG in noncoding regions, and the mutant RNA is disease causing. Treatments for these disorders are limited to symptomatic intervention. RNA interference (RNAi), which is a method for inhibiting target gene expression, provides a unique tool for therapy by attacking the fundamental problem directly. In this review, we describe briefly several representative disorders and their respective molecular targets, and methods to accomplish therapeutic RNAi. Finally, we summarize studies performed to date.
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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.