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Five siRNAs targeting three SNPs may provide therapy for three-quarters of Huntington's disease patients.
Huntington's disease: silencing a brutal killer.
HTT-lowering reverses Huntington's disease immune dysfunction caused by NF?B pathway dysregulation.
Increased Steady-State Mutant Huntingtin mRNA in Huntington's Disease Brain.
Safety of Striatal Infusion of siRNA in a Transgenic Huntington's Disease Mouse Model.
Does the Mutant CAG Expansion in Huntingtin mRNA Interfere with Exonucleolytic Cleavage of its First Exon?
Cellular Analysis of Silencing the?Huntington's Disease Gene Using AAV9?Mediated Delivery of Artificial Micro?RNA into the Striatum of?Q140/Q140?Mice.
Allele-Selective Suppression of Mutant Huntingtin in Primary Human Blood Cells.
Alterations in mRNA 3' UTR Isoform Abundance Accompany Gene Expression Changes in Human Huntington's Disease Brains.
Artificial miRNAs Reduce Human Mutant Huntingtin Throughout the Striatum in a Transgenic Sheep Model of Huntington's Disease.
Huntington's Disease: Les Jeux Sont Faits?
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.