Publications by authors named "Nicholas Caron"

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Molecular and imaging biomarker responses to brain mutant HTT lowering in a mouse model of Huntington disease.
Nicholas S Caron, Jianjia Fan, Da Ma, Andrew Yung, Seunghyun Ko

Mol Ther Nucleic Acids· December 2025

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CAG-targeted brain-permeable therapy tested in biallelic humanized polyQ mouse models.
Magdalena Surdyka, Żaneta Kalinowska-Pośka, Anna Niewiadomska-Cimicka, Ewelina Jesion, Agnieszka Fiszer, Nicholas S Caron

Mol Ther Nucleic Acids· June 2025

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Challenges and advances for huntingtin detection in cerebrospinal fluid: in support of relative quantification.
Rachel J Harding, Yuanyun Xie, Nicholas S Caron, Hailey Findlay-Black, Caroline Lyu

Biomark Res· April 2025

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Exon 1-targeting miRNA reduces the pathogenic exon 1 HTT protein in Huntington's disease models.
Marina Sogorb-Gonzalez, Christian Landles, Nicholas S Caron, Anouk Stam, Georgina Osborne

Brain· December 2024

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Elevated plasma and CSF neurofilament light chain concentrations are stabilized in response to mutant huntingtin lowering in the brains of Huntington's disease mice.
Nicholas S Caron, Lauren M Byrne, Fanny L Lemarié, Jeffrey N Bone, Amirah E-E Aly

Transl Neurodegener· October 2024

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Challenges and advances for huntingtin detection in cerebrospinal fluid: in support of relative quantification.
Rachel J Harding, Yuanyun Xie, Nicholas S Caron, Hailey Findlay-Black, Caroline Lyu

bioRxiv· September 2024

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Systemic delivery of mutant huntingtin lowering antisense oligonucleotides to the brain using apolipoprotein A-I nanodisks for Huntington disease.
Nicholas S Caron, Amirah E-E Aly, Hailey Findlay Black, Dale D O Martin, Mandi E Schmidt

J Control Release· March 2024

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Limitations of Dual-Single Guide RNA CRISPR Strategies for the Treatment of Central Nervous System Genetic Disorders.
Fábio Duarte, Gabriel Vachey, Nicholas S Caron, Melanie Sipion, Maria Rey

Hum Gene Ther· September 2023

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Delivery of mutant huntingtin-lowering antisense oligonucleotides to the brain by intranasally administered apolipoprotein A-I nanodisks.
Amirah E-E Aly, Nicholas S Caron, Hailey Findlay Black, Mandi E Schmidt, Christine Anderson

J Control Release· August 2023

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Allele-specific quantitation of ATXN3 and HTT transcripts in polyQ disease models.
Paweł Joachimiak, Adam Ciesiołka, Emilia Kozłowska, Paweł M Świtoński, Grzegorz Figura, Nicholas S Caron

BMC Biol· February 2023

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Cerebrospinal fluid biomarkers for assessing Huntington disease onset and severity.
Nicholas S Caron, Arsalan S Haqqani, Akshdeep Sandhu, Amirah E Aly, Hailey Findlay Black

Brain Commun· November 2022

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Cerebrospinal fluid mutant huntingtin is a biomarker for huntingtin lowering in the striatum of Huntington disease mice.
Nicholas S Caron, Raul Banos, Amirah E Aly, Yuanyun Xie, Seunghyun Ko

Neurobiol Dis· May 2022

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Functional characterization of variants of unknown significance in a spinocerebellar ataxia patient using an unsupervised machine learning pipeline.
Siddharth Nath, Nicholas S Caron, Linda May, Oxana B Gluscencova, Jill Kolesar

Hum Genome Var· April 2022

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Reliable Resolution of Full-Length Huntingtin Alleles by Quantitative Immunoblotting.
Nicholas S Caron, Christine Anderson, Hailey Findlay Black, Shaun S Sanders, Fanny L Lemarié

J Huntingtons Dis· January 2022

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Rescue of aberrant huntingtin palmitoylation ameliorates mutant huntingtin-induced toxicity.
Fanny L Lemarié, Nicholas S Caron, Shaun S Sanders, Mandi E Schmidt, Yen T N Nguyen

Neurobiol Dis· October 2021

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Mutant Huntingtin Is Cleared from the Brain via Active Mechanisms in Huntington Disease.
Nicholas S Caron, Raul Banos, Christopher Yanick, Amirah E Aly, Lauren M Byrne

J Neurosci· January 2021

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Frequency of the loss of CAA interruption in the HTT CAG tract and implications for Huntington disease in the reduced penetrance range.
Hailey Findlay Black, Galen E B Wright, Jennifer A Collins, Nicholas Caron, Chris Kay

Genet Med· December 2020

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Interrupting sequence variants and age of onset in Huntington's disease: clinical implications and emerging therapies.
Galen E B Wright, Hailey Findlay Black, Jennifer A Collins, Terence Gall-Duncan, Nicholas S Caron

Lancet Neurol· November 2020

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DAPK1 Promotes Extrasynaptic GluN2B Phosphorylation and Striatal Spine Instability in the YAC128 Mouse Model of Huntington Disease.
Mandi E Schmidt, Nicholas S Caron, Amirah E Aly, Fanny L Lemarié, Louisa Dal Cengio

Front Cell Neurosci· November 2020

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Gene expression profiles complement the analysis of genomic modifiers of the clinical onset of Huntington disease.
Galen E B Wright, Nicholas S Caron, Bernard Ng, Lorenzo Casal, William Casazza

Hum Mol Genet· September 2020

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The Interaction of Aging and Cellular Stress Contributes to Pathogenesis in Mouse and Human Huntington Disease Neurons.
Emily Machiela, Ritika Jeloka, Nicholas S Caron, Shagun Mehta, Mandi E Schmidt

Front Aging Neurosci· September 2020

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Potent and sustained huntingtin lowering via AAV5 encoding miRNA preserves striatal volume and cognitive function in a humanized mouse model of Huntington disease.
Nicholas S Caron, Amber L Southwell, Cynthia C Brouwers, Louisa Dal Cengio, Yuanyun Xie

Nucleic Acids Res· January 2020

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A Comprehensive Haplotype-Targeting Strategy for Allele-Specific HTT Suppression in Huntington Disease.
Chris Kay, Jennifer A Collins, Nicholas S Caron, Luciana de Andrade Agostinho, Hailey Findlay-Black

Am J Hum Genet· December 2019

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Activation of Caspase-6 Is Promoted by a Mutant Huntingtin Fragment and Blocked by an Allosteric Inhibitor Compound.
Dagmar E Ehrnhoefer, Niels H Skotte, Jeanette Reinshagen, Xiaofan Qiu, Björn Windshügel, Nicholas S Caron

Cell Chem Biol· September 2019

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