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Triplet nucleotide repeat-based siRNAs are highly toxic to cancer cells
Lymphoblast cells from a patient with Freidriech's Ataxia were incubated with pyrrole-imidazole polyamides targeted to the GAA triplet repeat in the intron 1. The polyamides were shown in cell culture to increase levels of endogenous frataxin mRNA. A normal sibling derived lymphoblast cell line wa...
ORGANISM(S): Homo sapiens 
The inherited neurodegenerative disease Friedreichâ??s ataxia (FRDA) is caused by hyperexpansion of GAAâ?¢TTC trinucleotide repeats within the first intron of the FXN gene, encoding the mitochondrial protein frataxin. Long GAAâ?¢TTC repeats causes heterochromatin-mediated silencing and loss of frata...
ORGANISM(S): Homo sapiens 
Triplet repeat siRNAs as found amplified in diseases such as Huntingtons disease can be used to kill cancer cells
ORGANISM(S): Homo sapiens 
2018-01-12 | GSE104552 | GEO
Short tracts of trinucleotide repeats with less than 10 repeats are found frequently throughout the genome without any apparent negative impact on DNA replication fork progression or transcription elongation. CGG binding protein 1 (CGGBP1) binds to CGG triplet repeats and has been implicated in mult...
ORGANISM(S): Homo sapiens (Human) 
2025-10-20 | PXD045654 | Pride
The inherited neurodegenerative disease Friedreich’s ataxia (FRDA) is caused by hyperexpansion of GAA•TTC trinucleotide repeats within the first intron of the FXN gene, encoding the mitochondrial protein frataxin. Long GAA•TTC repeats causes heterochromatin-mediated silencing and loss of frataxin in...
ORGANISM(S): Homo sapiens 
2010-07-02 | GSE22651 | GEO
Effect of CWG-cPIP on CAG/CTG triplet repeat mouse brain
Whole genome sequence (WGS) data was generated on 58 samples with validated repeat lengths for the CAG repeat associated with Huntington disease, These samples were sequenced using 2x150bp reads on an Illumina HiSeqX sequencer and the repeat expansions were called using ExpansionHunter to demonstrat...
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