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Page 1
Prospective natural history study of C9orf72 ALS clinical characteristics and biomarkers.
Cammack AJ, Atassi N, Hyman T, van den Berg LH, Harms M, Baloh RH, Brown RH, van Es MA, Veldink JH, de Vries BS, Rothstein JD, Drain C, Jockel-Balsarotti J, Malcolm A, Boodram S, Salter A, Wightman N, Yu H, Sherman AV, Esparza TJ, McKenna-Yasek D, Owegi MA, Douthwright C; Alzheimer's Disease Neuroimaging Initiative; McCampbell A, Ferguson T, Cruchaga C, Cudkowicz M, Miller TM. Cammack AJ, et al. Among authors: wightman n. Neurology. 2019 Oct 22;93(17):e1605-e1617. doi: 10.1212/WNL.0000000000008359. Epub 2019 Oct 2. Neurology. 2019. PMID: 31578300 Free PMC article.
A high-throughput screen to identify inhibitors of SOD1 transcription.
Wright PD, Wightman N, Huang M, Weiss A, Sapp PC, Cuny GD, Ivinson AJ, Glicksman MA, Ferrante RJ, Matson W, Matson S, Brown RH Jr. Wright PD, et al. Among authors: wightman n. Front Biosci (Elite Ed). 2012 Jun 1;4(8):2701-8. doi: 10.2741/e584. Front Biosci (Elite Ed). 2012. PMID: 22652679 Free PMC article.
Human C9ORF72 Hexanucleotide Expansion Reproduces RNA Foci and Dipeptide Repeat Proteins but Not Neurodegeneration in BAC Transgenic Mice.
Peters OM, Cabrera GT, Tran H, Gendron TF, McKeon JE, Metterville J, Weiss A, Wightman N, Salameh J, Kim J, Sun H, Boylan KB, Dickson D, Kennedy Z, Lin Z, Zhang YJ, Daughrity L, Jung C, Gao FB, Sapp PC, Horvitz HR, Bosco DA, Brown SP, de Jong P, Petrucelli L, Mueller C, Brown RH Jr. Peters OM, et al. Among authors: wightman n. Neuron. 2015 Dec 2;88(5):902-909. doi: 10.1016/j.neuron.2015.11.018. Neuron. 2015. PMID: 26637797 Free PMC article.
SOD1 Suppression with Adeno-Associated Virus and MicroRNA in Familial ALS.
Mueller C, Berry JD, McKenna-Yasek DM, Gernoux G, Owegi MA, Pothier LM, Douthwright CL, Gelevski D, Luppino SD, Blackwood M, Wightman NS, Oakley DH, Frosch MP, Flotte TR, Cudkowicz ME, Brown RH Jr. Mueller C, et al. Among authors: wightman ns. N Engl J Med. 2020 Jul 9;383(2):151-158. doi: 10.1056/NEJMoa2005056. N Engl J Med. 2020. PMID: 32640133
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.
Quezada E, Cappelli C, Diaz I, Jury N, Wightman N, Brown RH Jr, Montecino M, van Zundert B. Quezada E, et al. Among authors: wightman n. Clin Epigenetics. 2021 Mar 16;13(1):56. doi: 10.1186/s13148-021-01039-z. Clin Epigenetics. 2021. PMID: 33726839 Free PMC article.
Suppression of mutant C9orf72 expression by a potent mixed backbone antisense oligonucleotide.
Tran H, Moazami MP, Yang H, McKenna-Yasek D, Douthwright CL, Pinto C, Metterville J, Shin M, Sanil N, Dooley C, Puri A, Weiss A, Wightman N, Gray-Edwards H, Marosfoi M, King RM, Kenderdine T, Fabris D, Bowser R, Watts JK, Brown RH Jr. Tran H, et al. Among authors: wightman n. Nat Med. 2022 Jan;28(1):117-124. doi: 10.1038/s41591-021-01557-6. Epub 2021 Dec 23. Nat Med. 2022. PMID: 34949835 Free PMC article.
Up-regulation of cholesterol synthesis pathways and limited neurodegeneration in a knock-in Sod1 mutant mouse model of ALS.
Dominov JA, Madigan LA, Whitt JP, Rademacher KL, Webster KM, Zhang H, Banno H, Tang S, Zhang Y, Wightman N, Shychuck EM, Page J, Weiss A, Kelly K, Kucukural A, Brodsky MH, Jaworski A, Fallon JR, Lipscombe D, Brown RH. Dominov JA, et al. Among authors: wightman n. bioRxiv [Preprint]. 2023 May 5:2023.05.05.539444. doi: 10.1101/2023.05.05.539444. bioRxiv. 2023. PMID: 37205335 Free PMC article. Preprint.