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Pfister, Edith L; Chase, Kathryn O; Sun, Huaming et al. (2017) Safe and Efficient Silencing with a Pol II, but Not a Pol lII, Promoter Expressing an Artificial miRNA Targeting Human Huntingtin. Mol Ther Nucleic Acids 7:324-334
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Haraszti, Reka A; Coles, Andrew; Aronin, Neil et al. (2017) Loading of Extracellular Vesicles with Chemically Stabilized Hydrophobic siRNAs for the Treatment of Disease in the Central Nervous System. Bio Protoc 7:
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Nikan, Mehran; Osborn, Maire F; Coles, Andrew H et al. (2017) Synthesis and Evaluation of Parenchymal Retention and Efficacy of a Metabolically Stable O-Phosphocholine-N-docosahexaenoyl-l-serine siRNA Conjugate in Mouse Brain. Bioconjug Chem 28:1758-1766
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Alterman, Julia F; Coles, Andrew H; Hall, Lauren M et al. (2017) A High-throughput Assay for mRNA Silencing in Primary Cortical Neurons in vitro with Oligonucleotide Therapeutics. Bio Protoc 7:
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Romo, Lindsay; Ashar-Patel, Ami; Pfister, Edith et al. (2017) Alterations in mRNA 3' UTR Isoform Abundance Accompany Gene Expression Changes in Human Huntington's Disease Brains. Cell Rep 20:3057-3070
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Didiot, Marie-Cécile; Hall, Lauren M; Coles, Andrew H et al. (2016) Exosome-mediated Delivery of Hydrophobically Modified siRNA for Huntingtin mRNA Silencing. Mol Ther 24:1836-1847
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Coles, Andrew H; Osborn, Maire F; Alterman, Julia F et al. (2016) A High-Throughput Method for Direct Detection of Therapeutic Oligonucleotide-Induced Gene Silencing In Vivo. Nucleic Acid Ther 26:86-92
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Liu, Wanzhao; Pfister, Edith L; Kennington, Lori A et al. (2016) Does the Mutant CAG Expansion in Huntingtin mRNA Interfere with Exonucleolytic Cleavage of its First Exon? J Huntingtons Dis 5:33-8
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Choudhury, Sourav R; Harris, Anne F; Cabral, Damien J et al. (2016) Widespread Central Nervous System Gene Transfer and Silencing After Systemic Delivery of Novel AAV-AS Vector. Mol Ther 24:726-35
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Vodicka, Petr; Mo, Shunyan; Tousley, Adelaide et al. (2015) Mass Spectrometry Analysis of Wild-Type and Knock-in Q140/Q140 Huntington's Disease Mouse Brains Reveals Changes in Glycerophospholipids Including Alterations in Phosphatidic Acid and Lyso-Phosphatidic Acid. J Huntingtons Dis 4:187-201
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Showing the most recent 10 out of 43 publications
