Title : Oligonucleotides Targeting DNA Repeats Downregulate Huntingtin Gene Expression in Huntington's Patient-Derived Neural Model System.

Pub. Date : 2021 Dec

PMID : 34520257






4 Functional Relationships(s)
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Sentence
Compound Name
Protein Name
Organism
1 HD is caused by a CAG CTG trinucleotide-repeat expansion in exon 1 of the huntingtin (HTT) gene leading to the formation of mutant HTT (mtHTT) protein aggregates. trinucleotide huntingtin Homo sapiens
2 HD is caused by a CAG CTG trinucleotide-repeat expansion in exon 1 of the huntingtin (HTT) gene leading to the formation of mutant HTT (mtHTT) protein aggregates. trinucleotide huntingtin Homo sapiens
3 HD is caused by a CAG CTG trinucleotide-repeat expansion in exon 1 of the huntingtin (HTT) gene leading to the formation of mutant HTT (mtHTT) protein aggregates. trinucleotide huntingtin Homo sapiens
4 Previously, we designed a novel anti-gene oligonucleotide (AGO)-based strategy directly targeting the HTT trinucleotide-repeats in DNA and reported downregulation of mRNA and protein in HD patient fibroblasts. trinucleotide huntingtin Homo sapiens