Showing results (61-70 of 83) with videos related to

Sort By:
Pageof 9
Human Gene Therapy|August 11, 2021
Allele-Specific Knockdown of Mutant Huntingtin Protein via Editing at Coding Region Single Nucleotide Polymorphism HeterozygositiesSarah R Oikemus, Edith L Pfister, Ellen Sapp, et al.
Cell Reports|September 6, 2018
Nuclear Localization of Huntingtin mRNA Is Specific to Cells of Neuronal OriginMarie-Cécile Didiot, Chantal M Ferguson, Socheata Ly, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|August 11, 2016
Exosome-mediated Delivery of Hydrophobically Modified siRNA for Huntingtin mRNA SilencingMarie-Cécile Didiot, Lauren M Hall, Andrew H Coles, et al.
The Journal of Neuroscience : the Official Journal of the Society for Neuroscience|February 22, 2024
Nacc1 Mutation in Mice Models Rare Neurodevelopmental Disorder with Underlying Synaptic DysfunctionMark A Deehan, Josine M Kothuis, Ellen Sapp, et al.
Molecular and Cellular Biology|September 16, 2009
Mutant huntingtin impairs vesicle formation from recycling endosomes by interfering with Rab11 activityXueyi Li, Clive Standley, Ellen Sapp, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|May 13, 2023
Di-valent siRNA-mediated silencing of MSH3 blocks somatic repeat expansion in mouse models of Huntington's diseaseDaniel O'Reilly, Jillian Belgrad, Chantal Ferguson, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|December 29, 2015
Widespread Central Nervous System Gene Transfer and Silencing After Systemic Delivery of Novel AAV-AS VectorSourav R Choudhury, Anne F Harris, Damien J Cabral, et al.
Nature Communications|October 3, 2022
Chemical engineering of therapeutic siRNAs for allele-specific gene silencing in Huntington's disease modelsFaith Conroy, Rachael Miller, Julia F Alterman, et al.
Acta Neuropathologica Communications|September 10, 2014
Behavioral deficits, early gliosis, dysmyelination and synaptic dysfunction in a mouse model of mucolipidosis IVYulia Grishchuk, Sarmi Sri, Nikita Rudinskiy, et al.
Research Square|July 3, 2023
Extended Nucleic Acid (exNA): A Novel, Biologically Compatible Backbone that Significantly Enhances Oligonucleotide Efficacy in vivoVignesh Narayan Hariharan, Jillian Caiazzi, Rachael Miller, et al.
Pageof 9