| http://purl.uniprot.org/citations/24981774 | http://www.w3.org/1999/02/22-rdf-syntax-ns#type | http://purl.uniprot.org/core/Journal_Citation |
| http://purl.uniprot.org/citations/24981774 | http://www.w3.org/2000/01/rdf-schema#comment | "Dentatorubral-pallidoluysian atrophy (DRPLA) is a progressive neurodegenerative disorder that currently has no curative treatments. DRPLA is caused by an expansion of a CAG trinucleotide repeat region within the protein-encoding sequence of the atrophin-1 (ATN-1) gene. Inhibition of mutant ATN-1 protein expression is one strategy for treating DRPLA, and allele-selective gene silencing agents that block mutant expression over wild-type expression would be lead compounds for therapeutic development. Here we develop an assay for distinguishing mutant from wild-type ATN-1 protein by gel electrophoresis. We use this assay to evaluate duplex RNAs and single-stranded silencing RNAs (ss-siRNAs) for allele-selective inhibition of ATN-1 protein expression. We observed potent and allele-selective inhibition by RNA duplexes that contain mismatched bases relative to the CAG target and have the potential to form miRNA-like complexes. ss-siRNAs that contained mismatches were as selective as mismatch-containing duplexes. We also report allele-selective inhibition by duplex RNAs containing unlocked nucleic acids or abasic substitutions, although selectivities are not as high. Five compounds that showed >8-fold allele selectivity for mutant ATN-1 were also selective for inhibiting the expression of two other trinucleotide repeat disease genes, ataxin-3 (ATXN-3) and huntingtin (HTT). These data demonstrate that the expanded trinucleotide repeat within ATN-1 mRNA is a potential target for compounds designed to achieve allele-selective inhibition of ATN-1 protein, and one agent may allow the targeting of multiple disease genes."xsd:string |
| http://purl.uniprot.org/citations/24981774 | http://purl.org/dc/terms/identifier | "doi:10.1021/bi500610r"xsd:string |
| http://purl.uniprot.org/citations/24981774 | http://purl.uniprot.org/core/author | "Liu J."xsd:string |
| http://purl.uniprot.org/citations/24981774 | http://purl.uniprot.org/core/author | "Hu J."xsd:string |
| http://purl.uniprot.org/citations/24981774 | http://purl.uniprot.org/core/author | "Martinez C."xsd:string |
| http://purl.uniprot.org/citations/24981774 | http://purl.uniprot.org/core/author | "Xiang Q."xsd:string |
| http://purl.uniprot.org/citations/24981774 | http://purl.uniprot.org/core/author | "Corey D.R."xsd:string |
| http://purl.uniprot.org/citations/24981774 | http://purl.uniprot.org/core/author | "Manoharan M."xsd:string |
| http://purl.uniprot.org/citations/24981774 | http://purl.uniprot.org/core/author | "Lima W.F."xsd:string |
| http://purl.uniprot.org/citations/24981774 | http://purl.uniprot.org/core/author | "Kuchimanchi S."xsd:string |
| http://purl.uniprot.org/citations/24981774 | http://purl.uniprot.org/core/author | "Prakash T.P."xsd:string |
| http://purl.uniprot.org/citations/24981774 | http://purl.uniprot.org/core/author | "Swayze E.E."xsd:string |
| http://purl.uniprot.org/citations/24981774 | http://purl.uniprot.org/core/author | "Rajeev K.G."xsd:string |
| http://purl.uniprot.org/citations/24981774 | http://purl.uniprot.org/core/author | "Lackey J.G."xsd:string |
| http://purl.uniprot.org/citations/24981774 | http://purl.uniprot.org/core/author | "Narayanannair K.J."xsd:string |
| http://purl.uniprot.org/citations/24981774 | http://purl.uniprot.org/core/date | "2014"xsd:gYear |
| http://purl.uniprot.org/citations/24981774 | http://purl.uniprot.org/core/name | "Biochemistry"xsd:string |
| http://purl.uniprot.org/citations/24981774 | http://purl.uniprot.org/core/pages | "4510-4518"xsd:string |
| http://purl.uniprot.org/citations/24981774 | http://purl.uniprot.org/core/title | "Allele-selective inhibition of mutant atrophin-1 expression by duplex and single-stranded RNAs."xsd:string |
| http://purl.uniprot.org/citations/24981774 | http://purl.uniprot.org/core/volume | "53"xsd:string |
| http://purl.uniprot.org/citations/24981774 | http://www.w3.org/2004/02/skos/core#exactMatch | http://purl.uniprot.org/pubmed/24981774 |
| http://purl.uniprot.org/citations/24981774 | http://xmlns.com/foaf/0.1/primaryTopicOf | https://pubmed.ncbi.nlm.nih.gov/24981774 |
| http://purl.uniprot.org/uniprot/#_P54259-mappedCitation-24981774 | http://www.w3.org/1999/02/22-rdf-syntax-ns#object | http://purl.uniprot.org/citations/24981774 |
| http://purl.uniprot.org/uniprot/#_Q86V38-mappedCitation-24981774 | http://www.w3.org/1999/02/22-rdf-syntax-ns#object | http://purl.uniprot.org/citations/24981774 |