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Year Number of Results
2001 1
2003 1
2004 2
2005 1
2008 1
2009 1
2013 1
2014 1
2015 4
2016 1
2017 1
2018 2
2020 0
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Page 1
Precise small-molecule recognition of a toxic CUG RNA repeat expansion.
Rzuczek SG, et al. Nat Chem Biol 2017. PMID 27941760 Free PMC article.
We designed allele-selective compounds that target such an RNA, the toxic noncoding repeat expansion (r(CUG)(exp)) that causes myotonic dystrophy type 1 (DM1). ...
We designed allele-selective compounds that target such an RNA, the toxic noncoding repeat expansion (r(CUG)(exp)) that causes …
Activating frataxin expression by single-stranded siRNAs targeting the GAA repeat expansion.
Shen X, et al. Bioorg Med Chem Lett 2018. PMID 30076049 Free PMC article.
The genetic cause of the disease is an expanded GAA repeat within the FXN gene. Agents that increase expression of FXN protein are a potential approach to therapy. We previously described anti-trinucleotide GAA duplex RNAs (dsRNAs) and antisense oligonucleotides (ASOs) that activate FXN protein expression in multiple patient derived cell lines. ...
The genetic cause of the disease is an expanded GAA repeat within the FXN gene. Agents that increase expression of FXN protein are a …
Chemically induced increases and decreases in the rate of expansion of a CAG*CTG triplet repeat.
Gomes-Pereira M and Monckton DG. Nucleic Acids Res 2004. PMID 15155855 Free PMC article.
Somatic mosaicism of repeat length is prominent in repeat expansion disorders such as Huntington disease and myotonic dystrophy. ...These data establish that drug induced suppression of somatic expansion is possible. These data also suggest that highly unstable expanded simple sequence repeats may act as sensitive reporters of genotoxic assault in the soma....
Somatic mosaicism of repeat length is prominent in repeat expansion disorders such as Huntington disease and myotonic d …
Reducing Mutant Huntingtin Protein Expression in Living Cells by a Newly Identified RNA CAG Binder
Matthes F, et al. ACS Chem Neurosci 2018. PMID 29506378 Free article.
Expanded CAG trinucleotide repeats in Huntington's disease (HD) are causative for neurotoxicity. The mutant CAG repeat RNA encodes neurotoxic polyglutamine proteins and can lead to a toxic gain of function by aberrantly recruiting RNA-binding proteins. ...Here we have identified a set of CAG repeat binder candidates by in silico methods. One of those, furamidine, reduces the level of binding of HTT mRNA to MID1 and other target proteins in vitro. ...
Expanded CAG trinucleotide repeats in Huntington's disease (HD) are causative for neurotoxicity. The mutant CAG repeat RNA enc …
Chemotherapeutic deletion of CTG repeats in lymphoblast cells from DM1 patients
Hashem VI, et al. Nucleic Acids Res 2004. PMID 15576360 Free PMC article.
Treatment with EMS frequently prevented the repeat expansion observed during growth in culture. A significant reduction of CTG repeat length by 100-350 (CTG)....Potential mechanisms of drug-induced deletion are presented....
Treatment with EMS frequently prevented the repeat expansion observed during growth in culture. A significant reduction of CTG …
Suppression of Somatic Expansion Delays the Onset of Pathophysiology in a Mouse Model of Huntington's Disease.
Budworth H, et al. PLoS Genet 2015. PMID 26247199 Free PMC article.
The inherited disease allele expresses a toxic protein, and whether further somatic expansion adds to toxicity is unknown. We have created an HD mouse model that resolves the effects of the inherited and somatic expansions. We show here that suppressing somatic expansion substantially delays the onset of disease in littermates that inherit the same disease-length allele. ...
The inherited disease allele expresses a toxic protein, and whether further somatic expansion adds to toxicity is unknown. We have cr …
DNA instability in replicating Huntington's disease lymphoblasts
Cannella M, et al. BMC Med Genet 2009. PMID 19210789 Free PMC article.
To test this hypothesis we used, as a model, cultured HD patients' lymphoblasts with various CAG repeat lengths. RESULTS: Although most lymphoblastoid cell lines (88%) showed little or no repeat instability even after six or more months culture, in lymphoblasts with large expansion repeats beyond 60 CAG repeats the mutation size and triplet mosaicism always increased during replication, implying that the repeat mutability for highly expanded mutations may quantitatively depend on the triplet expansion size. ...The longest triplet expansions show wide somatic variations and may offer a mechanistic model to study triplet drug-controlled instability and genetic factors influencing it....
To test this hypothesis we used, as a model, cultured HD patients' lymphoblasts with various CAG repeat lengths. RESULTS: Although mo …
Inhibition of polyglutamine aggregation in R6/2 HD brain slices-complex dose-response profiles
Smith DL, et al. Neurobiol Dis 2001. PMID 11741397
Huntington's disease (HD) is a late onset neurodegenerative disorder caused by a CAG/polyglutamine (polyQ) repeat expansion. ...
Huntington's disease (HD) is a late onset neurodegenerative disorder caused by a CAG/polyglutamine (polyQ) repeat expansion. . …
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