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Molecular Therapy : the Journal of the American Society of Gene Therapy|January 12, 2002
Viral-mediated gene transfer to mouse primary neural progenitor cellsStephanie M Hughes, Farid Moussavi-Harami, Sybille L Sauter, et al.
Nucleic Acids Research|February 3, 2004
Targeting Alzheimer's disease genes with RNA interference: an efficient strategy for silencing mutant allelesVictor M Miller, Cynthia M Gouvion, Beverly L Davidson, et al.
Annals of Neurology|June 5, 2003
Toward therapy for DYT1 dystonia: allele-specific silencing of mutant TorsinAPedro Gonzalez-Alegre, Victor M Miller, Beverly L Davidson, et al.
Molecular Therapy. Methods & Clinical Development|May 16, 2022
Combined overexpression of ATXN1L and mutant ATXN1 knockdown by AAV rescue motor phenotypes and gene signatures in SCA1 miceEllie M Carrell, Megan S Keiser, Ashley B Robbins, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|January 28, 2017
CRISPR/Cas9 Editing of the Mutant Huntingtin Allele In Vitro and In VivoAlex Mas Monteys, Shauna A Ebanks, Megan S Keiser, et al.
Veterinary Sciences|September 23, 2020
Foodborne Disease and the Need for Greater Foodborne Disease Surveillance in the CaribbeanBrendan Lee
Molecular Therapy. Methods & Clinical Development|February 21, 2025
CRISPR screen reveals modifiers of rAAV production including known rAAV infection genes playing an unexpected role in vector productionEmily E O'Driscoll, Sakshi Arora, Jonathan F Lang, et al.
The Journal of Neuroscience : the Official Journal of the Society for Neuroscience|October 14, 2005
Functional correction of CNS phenotypes in a lysosomal storage disease model using adeno-associated virus type 4 vectorsGumei Liu, Inês Martins, John A Wemmie, et al.
Human Molecular Genetics|May 13, 2010
Evolution of alternative splicing in primate brain transcriptomesLan Lin, Shihao Shen, Peng Jiang, et al.
Human Molecular Genetics|December 26, 2013
Human-specific microRNA regulation of FOXO1: implications for microRNA recognition element evolutionHayley S McLoughlin, Ji Wan, Ryan M Spengler, et al.
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