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Nature Reviews. Disease Primers|October 20, 2018
Author Correction: Lysosomal storage diseasesFrances M Platt, Alessandra d'Azzo, Beverly L Davidson, et al.
Molecular Therapy. Nucleic Acids|September 24, 2024
Cas9 editing of ATXN1 in a spinocerebellar ataxia type 1 mice and human iPSC-derived neuronsKelly J Fagan, Guillem Chillon, Ellie M Carrell, et al.
Nature Reviews. Disease Primers|October 3, 2018
Lysosomal storage diseasesFrances M Platt, Alessandra d'Azzo, Beverly L Davidson, et al.
Molecular Therapy. Nucleic Acids|July 16, 2014
Platelet Activating Factor Receptor Activation Improves siRNA Uptake and RNAi Responses in Well-differentiated Airway EpitheliaSateesh Krishnamurthy, Mark A Behlke, Michael A Apicella, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|April 4, 2025
Current trends in gene therapy to treat inherited disorders of the brainZaneta Matuszek, Brandon L Brown, Carolyn M Yrigollen, et al.
Neurobiology of Disease|April 17, 2018
Modulating membrane fluidity corrects Batten disease phenotypes in vitro and in vivoMark L Schultz, Luis Tecedor, Elena Lysenko, et al.
Genome Biology|April 28, 2022
DeepRepeat: direct quantification of short tandem repeats on signal data from nanopore sequencingLi Fang, Qian Liu, Alex Mas Monteys, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|June 18, 2009
Allele-specific RNAi mitigates phenotypic progression in a transgenic model of Alzheimer's diseaseEdgardo Rodríguez-Lebrón, Cynthia M Gouvion, Steven A Moore, et al.
Neurobiology of Disease|April 16, 2013
RNAi or overexpression: alternative therapies for Spinocerebellar Ataxia Type 1Megan S Keiser, James C Geoghegan, Ryan L Boudreau, et al.
Molecular Therapy. Nucleic Acids|April 8, 2015
Artificial miRNAs Targeting Mutant Huntingtin Show Preferential Silencing In Vitro and In VivoAlex Mas Monteys, Matthew J Wilson, Ryan L Boudreau, et al.
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