共 526 条
[1]
Aronin N.(2014)Huntingtin-lowering strategies in Huntington’s disease: Antisense oligonucleotides, small RNAs, and gene editing Movement Disorders. 29 1455-1461
[2]
DiFiglia M.(2009)Allele-specific silencing of mutant huntingtin and ataxin-3 genes by targeting expanded CAG repeats in mRNAs Nat. Biotechnol. 27 478-1188
[3]
Hu J.(2010)Allele-selective inhibition of huntingtin expression by switching to an miRNA-like RNAi mechanism Chem. Biol. 17 1183-1074
[4]
Matsui M.(1995)Widespread expression of Huntington’s disease gene (IT15) protein product Neuron 14 1065-823
[5]
Gagnon K.T.(1995)Targeted disruption of the Huntington’s disease gene results in embryonic lethality and behavioral and morphological changes in heterozygotes Cell 81 811-1267
[6]
Schwartz J.C.(1987)Homozygotes for Huntington’s disease Nature 326 194-2370
[7]
Gabillet S.(1989)Homozygote for Huntington disease Am. J. Hum. Genet. 45 615-3070
[8]
Arar K.(2015)Making (anti-)sense out of huntingtin levels in Huntington disease Mol. Neurodegener. 10 21-108
[9]
Wu J.(2017)Novel allele-specific quantification methods reveal no effects of adult onset CAG repeats on HTT mRNA and protein levels Hum. Mol. Genet. 26 1258-3477
[10]
Bezprozvanny I.(2017)The pathogenic exon 1 HTT protein is produced by incomplete splicing in Huntington’s disease patients Sci. Rept. 7 1307-615