Publications by authors named "Nicholas Wightman"

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Dynamic changes in chromosome and nuclear architecture during maturation of normal and ALS C9orf72 motor neurons.
Özgün Uyan, Snehal Sambare, Marlies E Oomen, Nicholas Wightman, Allana Schooley

bioRxiv· September 2025

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RNAi-mediated silencing of SOD1 profoundly extends survival and functional outcomes in ALS mice.
Alexandra Weiss, James W Gilbert, Iris Valeria Rivera Flores, Jillian Belgrad, Chantal Ferguson, Nicholas Wightman

Mol Ther· August 2025

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RNAi-mediated silencing of SOD1 profoundly extends survival and functional outcomes in ALS mice.
Alexandra Weiss, James W Gilbert, Iris Valeria Rivera Flores, Jillian Belgrad, Chantal Ferguson, Nicholas Wightman

bioRxiv· June 2024

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Artificial microRNA suppresses C9ORF72 variants and decreases toxic dipeptide repeat proteins in vivo.
Gabriela Toro Cabrera, Katharina E Meijboom, Abbas Abdallah, Helene Tran, Zachariah Foster, Nicholas Wightman

Gene Ther· March 2024

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Up-regulation of cholesterol synthesis pathways and limited neurodegeneration in a knock-in mutant mouse model of ALS.
Janice A Dominov, Laura A Madigan, Joshua P Whitt, Katerina L Rademacher, Kristin M Webster, Nicholas Wightman

bioRxiv· May 2023

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Suppression of mutant C9orf72 expression by a potent mixed backbone antisense oligonucleotide.
Hélène Tran, Michael P Moazami, Huiya Yang, Diane McKenna-Yasek, Catherine L Douthwright, Nicholas Wightman

Nat Med· January 2022

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BET bromodomain inhibitors PFI-1 and JQ1 are identified in an epigenetic compound screen to enhance C9ORF72 gene expression and shown to ameliorate C9ORF72-associated pathological and behavioral abnormalities in a C9ALS/FTD model.
Esteban Quezada, Claudio Cappelli, Iván Diaz, Nur Jury, Nicholas Wightman

Clin Epigenetics· March 2021

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Suppression with Adeno-Associated Virus and MicroRNA in Familial ALS.
Christian Mueller, James D Berry, Diane M McKenna-Yasek, Gwladys Gernoux, Margaret A Owegi, Nicholas S Wightman

N Engl J Med· July 2020

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Prospective natural history study of ALS clinical characteristics and biomarkers.
Alexander J Cammack, Nazem Atassi, Theodore Hyman, Leonard H van den Berg, Matthew Harms, Nicholas Wightman

Neurology· October 2019

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Trade in Zambian Edible Orchids-DNA Barcoding Reveals the Use of Unexpected Orchid Taxa for .
Sarina Veldman, Seol-Jong Kim, Tinde R van Andel, Maria Bello Font, Ruth E Bone, Nicholas Wightman

Genes (Basel)· November 2018

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Human C9ORF72 Hexanucleotide Expansion Reproduces RNA Foci and Dipeptide Repeat Proteins but Not Neurodegeneration in BAC Transgenic Mice.
Owen M Peters, Gabriela Toro Cabrera, Helene Tran, Tania F Gendron, Jeanne E McKeon, Nicholas Wightman

Neuron· December 2015

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A high-throughput screen to identify inhibitors of SOD1 transcription.
Paul D Wright, Nicholas Wightman, Mickey Huang, Alexandra Weiss, Peter C Sapp

Front Biosci (Elite Ed)· June 2012

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Screening for inhibitors of the SOD1 gene promoter: pyrimethamine does not reduce SOD1 levels in cell and animal models.
Paul D Wright, Mickey Huang, Alexandra Weiss, Jonathan Matthews, Nicholas Wightman

Neurosci Lett· October 2010

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