subject area of
- A dynamic structural model of expanded RNA CAG repeats: A refined X-ray structure and computational investigations using molecular dynamics and umbrella sampling simulations Academic Article
- Epigenetic characterization of the fmr1 gene and aberrant neurodevelopment in human induced pluripotent stem cell models of fragile x syndrome Academic Article
- Epigenetic therapy for Friedreich ataxia Academic Article
- Friedreich's ataxia induced pluripotent stem cells model intergenerational GAA.TTC triplet repeat instability Academic Article
- Genetic anticipation in Portuguese kindreds with familial amyloidotic polyneuropathy is unlikely to be caused by triplet repeat expansions Academic Article
- Ha novel approach to investigate tissue-specific trinucleotide repeat instability Academic Article
- In vivo cell-autonomous transcriptional abnormalities revealed in mice expressing mutant huntingtin in striatal but not cortical neurons Academic Article
- Inhibition of non-ATG translational events in cells via covalent small molecules targeting RNA Academic Article
- Length-dependent CTG·CAG triplet-repeat expansion in myotonic dystrophy patient-derived induced pluripotent stem cells Academic Article
- Polyglutamine-expanded spinocerebellar ataxia-7 protein disrupts normal SAGA and SLIK histone acetyltransferase activity Academic Article
- RNA toxicity and missplicing in the common eye disease fuchs endothelial corneal dystrophy Academic Article
- Rational design of ligands targeting triplet repeating transcripts that cause RNA dominant disease: Application to myotonic muscular dystrophy type 1 and spinocerebellar ataxia type 3 Academic Article
- Rationale for the development of 2-aminobenzamide histone deacetylase inhibitors as therapeutics for Friedreich ataxia Academic Article
- Role of mismatch repair enzymes in GAA.TTC triplet-repeat expansion in Friedreich ataxia induced pluripotent stem cells Academic Article
- Sequestration of DROSHA and DGCR8 by expanded CGG RNA repeats alters microRNA processing in Fragile X-associated tremor/ataxia syndrome Academic Article
- Structure and dynamics of RNA repeat expansions that cause Huntington's disease and myotonic dystrophy type 1 Academic Article
- Targeting the r(CGG) repeats that cause FXTAS with modularly assembled small molecules and oligonucleotides Academic Article