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Nuclear Localization of Huntingtin mRNA Is Specific to Cells of Neuronal Origin.
Rac1 Activity Is Modulated by Huntingtin and Dysregulated in Models of Huntington's Disease.
Loss of huntingtin function slows synaptic vesicle endocytosis in striatal neurons from the httQ140/Q140 mouse model of Huntington's disease.
Enriched chitosan nanoparticles loaded with siRNA are effective in lowering Huntington's disease gene expression following intranasal administration.
Structurally constrained phosphonate internucleotide linkage impacts oligonucleotide-enzyme interaction, and modulates siRNA activity and allele specificity.
Chemical engineering of therapeutic siRNAs for allele-specific gene silencing in Huntington's disease models.