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Plos One|May 6, 2014
CLN3 deficient cells display defects in the ARF1-Cdc42 pathway and actin-dependent eventsMark L Schultz, Luis Tecedor, Colleen S Stein, et al.Nature Reviews. Disease Primers|May 19, 2019
Publisher Correction: Lysosomal storage diseasesFrances M Platt, Alessandra d'Azzo, Beverly L Davidson, et al.Molecular Neurodegeneration|May 29, 2026
Temporal single-cell atlas of full-length Huntington's disease mouse model defines stage-specific signatures of corticostriatal dysfunctionAshley B Robbins, Paul T Ranum, Icnelia Huerta-Ocampo, et al.Molecular Therapy. Nucleic Acids|January 10, 2013
In vivo SELEX for Identification of Brain-penetrating AptamersCongsheng Cheng, Yong Hong Chen, Kim A Lennox, et al.Annals of Neurology|October 1, 2016
RNAi prevents and reverses phenotypes induced by mutant human ataxin-1Megan S Keiser, Alejandro Mas Monteys, Romuald Corbau, et al.Biorxiv : the Preprint Server for Biology|January 16, 2026
Temporal single-cell atlas of full-length Huntington's disease mouse model defines stage-specific signatures of corticostriatal dysfunctionAshley B Robbins, Paul T Ranum, Icnelia Huerta-Ocampo, et al.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.Pageof 23