Search Results - "CARON, Nicholas"

Therapeutic approaches to Huntington disease: from the bench to the clinic by Caron, Nicholas S., Dorsey, E. Ray, Hayden, Michael R.

“…New therapies, including RNA-based and gene therapies, are poised to change the therapeutic landscape for Huntington disease. In this article, Hayden and…”
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Interrupting sequence variants and age of onset in Huntington's disease: clinical implications and emerging therapies by Wright, Galen E B, Black, Hailey Findlay, Collins, Jennifer A, Gall-Duncan, Terence, Caron, Nicholas S, Pearson, Christopher E, Hayden, Michael R

“…Huntington's disease is a fatal neurodegenerative disorder that is caused by CAG-CAA repeat expansion, encoding polyglutamine, in the huntingtin (HTT) gene…”
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Using FLIM-FRET to measure conformational changes of transglutaminase type 2 in live cells by Caron, Nicholas S, Munsie, Lise N, Keillor, Jeffrey W, Truant, Ray

“…Transglutaminase type 2 (TG2) is a ubiquitously expressed member of the transglutaminase family, capable of mediating a transamidation reaction between a…”
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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 by Caron, Nicholas S, Southwell, Amber L, Brouwers, Cynthia C, Cengio, Louisa Dal, Xie, Yuanyun, Black, Hailey Findlay, Anderson, Lisa M, Ko, Seunghyun, Zhu, Xiang, van Deventer, Sander J, Evers, Melvin M, Konstantinova, Pavlina, Hayden, Michael R

“…Abstract Huntington disease (HD) is a fatal neurodegenerative disease caused by a pathogenic expansion of a CAG repeat in the huntingtin (HTT) gene. There are…”
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Exon 1-targeting miRNA reduces the pathogenic exon 1 HTT protein in Huntington's disease models by Sogorb-Gonzalez, Marina, Landles, Christian, Caron, Nicholas S, Stam, Anouk, Osborne, Georgina, Hayden, Michael R, Howland, David, van Deventer, Sander, Bates, Gillian P, Vallès, Astrid, Evers, Melvin

“…Abstract Huntington’s disease (HD) is a fatal neurodegenerative disease caused by a trinucleotide repeat expansion in exon 1 of the huntingtin gene (HTT) that…”
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Intrinsic mutant HTT-mediated defects in oligodendroglia cause myelination deficits and behavioral abnormalities in Huntington disease by Bardile, Costanza Ferrari, Garcia-Miralles, Marta, Caron, Nicholas S., Rayan, Nirmala Arul, Langley, Sarah R., Harmston, Nathan, Rondelli, Ana Maria, Teo, Roy Tang Yi, Waltl, Sabine, Anderson, Lisa M., Bae, Han-Gyu, Jung, Sangyong, Williams, Anna, Prabhakar, Shyam, Petretto, Enrico, Hayden, Michael R., Pouladi, Mahmoud A.

“…Whitematter abnormalities are a nearly universal pathological feature of neurodegenerative disorders including Huntington disease (HD). A long-held assumption…”
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Cerebrospinal fluid mutant huntingtin is a biomarker for huntingtin lowering in the striatum of Huntington disease mice by Caron, Nicholas S., Banos, Raul, Aly, Amirah E., Xie, Yuanyun, Ko, Seunghyun, Potluri, Nalini, Anderson, Christine, Black, Hailey Findlay, Anderson, Lisa M., Gordon, Benjamin, Southwell, Amber L., Hayden, Michael R.

“…Huntington disease (HD) is a neurodegenerative disease caused by a trinucleotide repeat expansion in the HTT gene encoding an elongated polyglutamine tract in…”
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Mutant Huntingtin Is Cleared from the Brain via Active Mechanisms in Huntington Disease by Caron, Nicholas S, Banos, Raul, Yanick, Christopher, Aly, Amirah E, Byrne, Lauren M, Smith, Ethan D, Xie, Yuanyun, Smith, Stephen E P, Potluri, Nalini, Findlay Black, Hailey, Casal, Lorenzo, Ko, Seunghyun, Cheung, Daphne, Kim, Hyeongju, Seong, Ihn Sik, Wild, Edward J, Song, Ji-Joon, Hayden, Michael R, Southwell, Amber L

“…Huntington disease (HD) is a neurodegenerative disease caused by a CAG trinucleotide repeat expansion in the huntingtin ( ) gene. Therapeutics that lower HTT…”
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Allele-specific quantitation of ATXN3 and HTT transcripts in polyQ disease models by Joachimiak, Paweł, Ciesiołka, Adam, Kozłowska, Emilia, Świtoński, Paweł M, Figura, Grzegorz, Ciołak, Agata, Adamek, Grażyna, Surdyka, Magdalena, Kalinowska-Pośka, Żaneta, Figiel, Maciej, Caron, Nicholas S, Hayden, Michael R, Fiszer, Agnieszka

“…The majority of genes in the human genome is present in two copies but the expression levels of both alleles is not equal. Allelic imbalance is an aspect of…”
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Rescue of aberrant huntingtin palmitoylation ameliorates mutant huntingtin-induced toxicity by Lemarié, Fanny L., Caron, Nicholas S., Sanders, Shaun S., Schmidt, Mandi E., Nguyen, Yen T.N., Ko, Seunghyun, Xu, Xiaohong, Pouladi, Mahmoud A., Martin, Dale D.O., Hayden, Michael R.

“…Huntington disease (HD) is a neurodegenerative disorder caused by a CAG expansion in the HTT gene that codes for an elongated polyglutamine tract in the…”
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A Comprehensive Haplotype-Targeting Strategy for Allele-Specific HTT Suppression in Huntington Disease by Kay, Chris, Collins, Jennifer A., Caron, Nicholas S., Agostinho, Luciana de Andrade, Findlay-Black, Hailey, Casal, Lorenzo, Sumathipala, Dulika, Dissanayake, Vajira H.W., Cornejo-Olivas, Mario, Baine, Fiona, Krause, Amanda, Greenberg, Jacquie L., Paiva, Carmen Lúcia Antão, Squitieri, Ferdinando, Hayden, Michael R.

“…Huntington disease (HD) is a fatal neurodegenerative disorder caused by a gain-of-function mutation in HTT. Suppression of mutant HTT has emerged as a leading…”
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Huntingtin Haplotypes Provide Prioritized Target Panels for Allele-specific Silencing in Huntington Disease Patients of European Ancestry by Kay, Chris, Collins, Jennifer A, Skotte, Niels H, Southwell, Amber L, Warby, Simon C, Caron, Nicholas S, Doty, Crystal N, Nguyen, Betty, Griguoli, Annamaria, Ross, Colin J, Squitieri, Ferdinando, Hayden, Michael R

“…Huntington disease (HD) is a dominant neurodegenerative disorder caused by a CAG repeat expansion in the Huntingtin gene (HTT). Heterozygous polymorphisms in…”
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Gene expression profiles complement the analysis of genomic modifiers of the clinical onset of Huntington disease by Wright, Galen E B, Caron, Nicholas S, Ng, Bernard, Casal, Lorenzo, Casazza, William, Xu, Xiaohong, Ooi, Jolene, Pouladi, Mahmoud A, Mostafavi, Sara, Ross, Colin J D, Hayden, Michael R

“…Abstract Huntington disease (HD) is a neurodegenerative disorder that is caused by a CAG repeat expansion in HTT. The length of this repeat, however, only…”
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Systemic delivery of mutant huntingtin lowering antisense oligonucleotides to the brain using apolipoprotein A-I nanodisks for Huntington disease by Caron, Nicholas S., Aly, Amirah E.-E., Findlay Black, Hailey, Martin, Dale D.O., Schmidt, Mandi E., Ko, Seunghyun, Anderson, Christine, Harvey, Emily M., Casal, Lorenzo L., Anderson, Lisa M., Rahavi, Seyed M.R., Reid, Gregor S.D., Oda, Michael N., Stanimirovic, Danica, Abulrob, Abedelnasser, McBride, Jodi L., Leavitt, Blair R., Hayden, Michael R.

“…Efficient delivery of therapeutics to the central nervous system (CNS) remains a major challenge for the treatment of neurological diseases. Huntington disease…”
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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 by Findlay Black, Hailey, Wright, Galen E. B., Collins, Jennifer A., Caron, Nicholas, Kay, Chris, Xia, Qingwen, Arning, Larissa, Bijlsma, Emilia K., Squitieri, Ferdinando, Nguyen, Huu Phuc, Hayden, Michael R.

“…Purpose In some Huntington disease (HD) patients, the “loss of interruption” (LOI) variant eliminates an interrupting codon in the HTT CAG-repeat tract, which…”
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Laquinimod rescues striatal, cortical and white matter pathology and results in modest behavioural improvements in the YAC128 model of Huntington disease by Garcia-Miralles, Marta, Hong, Xin, Tan, Liang Juin, Caron, Nicholas S., Huang, Yihui, To, Xuan Vinh, Lin, Rachel Yanping, Franciosi, Sonia, Papapetropoulos, Spyros, Hayardeny, Liat, Hayden, Michael R., Chuang, Kai-Hsiang, Pouladi, Mahmoud A.

“…Increasing evidence supports a role for abnormal immune activation and inflammatory responses in Huntington disease (HD). In this study, we evaluated the…”
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Ultrasensitive measurement of huntingtin protein in cerebrospinal fluid demonstrates increase with Huntington disease stage and decrease following brain huntingtin suppression by Southwell, Amber L., Smith, Stephen E.P., Davis, Tessa R., Caron, Nicholas S., Villanueva, Erika B., Xie, Yuanyun, Collins, Jennifer A., Li Ye, Min, Sturrock, Aaron, Leavitt, Blair R., Schrum, Adam G., Hayden, Michael R.

“…Quantitation of huntingtin protein in the brain is needed, both as a marker of Huntington disease (HD) progression and for use in clinical gene silencing…”
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Delivery of mutant huntingtin-lowering antisense oligonucleotides to the brain by intranasally administered apolipoprotein A-I nanodisks by Aly, Amirah E.-E., Caron, Nicholas S., Black, Hailey Findlay, Schmidt, Mandi E., Anderson, Christine, Ko, Seunghyun, Baddeley, Helen J.E., Anderson, Lisa, Casal, Lorenzo L., Rahavi, Reza S.M., Martin, Dale D.O., Hayden, Michael R.

“…Lowering mutant huntingtin (mHTT) in the central nervous system (CNS) using antisense oligonucleotides (ASOs) is a promising approach currently being evaluated…”
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Live cell imaging and biophotonic methods reveal two types of mutant huntingtin inclusions by Caron, Nicholas S, Hung, Claudia L, Atwal, Randy S, Truant, Ray

“…Huntington's disease (HD) is an autosomal dominant, neurodegenerative disorder that can be characterized by the presence of protein inclusions containing…”
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DAPK1 Promotes Extrasynaptic GluN2B Phosphorylation and Striatal Spine Instability in the YAC128 Mouse Model of Huntington Disease by Schmidt, Mandi E, Caron, Nicholas S, Aly, Amirah E, Lemarié, Fanny L, Dal Cengio, Louisa, Ko, Yun, Lazic, Nikola, Anderson, Lisa, Nguyen, Betty, Raymond, Lynn A, Hayden, Michael R

“…Huntington disease (HD) is a devastating neurodegenerative disorder caused by a CAG repeat expansion in the huntingtin gene. Disrupted cortico-striatal…”
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