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Journal of Huntington'S Disease|October 16, 2018
Selective Neuronal Uptake and Distribution of AAVrh8, AAV9, and AAVrh10 in Sheep After Intra-Striatal AdministrationErica Mondo, Richard Moser, Guangping Gao, et al.
Nucleic Acids Research|December 1, 2021
Structurally constrained phosphonate internucleotide linkage impacts oligonucleotide-enzyme interaction, and modulates siRNA activity and allele specificityKen Yamada, Samuel Hildebrand, Sarah M Davis, et al.
Molecular Therapy. Nucleic Acids|December 2, 2015
Hydrophobically Modified siRNAs Silence Huntingtin mRNA in Primary Neurons and Mouse BrainJulia F Alterman, Lauren M Hall, Andrew H Coles, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|October 21, 2018
Exosomes Produced from 3D Cultures of MSCs by Tangential Flow Filtration Show Higher Yield and Improved ActivityReka Agnes Haraszti, Rachael Miller, Matteo Stoppato, et al.
Journal of Huntington'S Disease|December 31, 2018
Rac1 Activity Is Modulated by Huntingtin and Dysregulated in Models of Huntington's DiseaseAdelaide Tousley, Maria Iuliano, Elizabeth Weisman, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|August 26, 2018
Transvascular Delivery of Hydrophobically Modified siRNAs: Gene Silencing in the Rat Brain upon Disruption of the Blood-Brain BarrierBruno M D C Godinho, Nils Henninger, James Bouley, et al.
The Journal of Biological Chemistry|April 2, 2011
Cysteine oxidation within N-terminal mutant huntingtin promotes oligomerization and delays clearance of soluble proteinJonathan H Fox, Teal Connor, Megan Stiles, et al.
Journal of Huntington'S Disease|February 27, 2018
HttQ111/+ Huntington's Disease Knock-in Mice Exhibit Brain Region-Specific Morphological Changes and Synaptic DysfunctionMarina Kovalenko, Austen Milnerwood, James Giordano, et al.
Molecular Neurodegeneration|December 16, 2010
Huntingtin cleavage product A forms in neurons and is reduced by gamma-secretase inhibitorsKimberly B Kegel, Ellen Sapp, Jonathan Alexander, et al.
Human Molecular Genetics|September 5, 2025
ESC derived human cortical neurons harboring the NACC1 c.892C > T p.R298W missense mutation exhibit molecular differences from controls that influence neuronal maturationMark Deehan, Ellen Sapp, Andrew Iwanowicz, et al.
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