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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: douthwright cl. 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.
Poly(GR) and poly(GA) in cerebrospinal fluid as potential biomarkers for C9ORF72-ALS/FTD.
Krishnan G, Raitcheva D, Bartlett D, Prudencio M, McKenna-Yasek DM, Douthwright C, Oskarsson BE, Ladha S, King OD, Barmada SJ, Miller TM, Bowser R, Watts JK, Petrucelli L, Brown RH, Kankel MW, Gao FB. Krishnan G, et al. Nat Commun. 2022 May 19;13(1):2799. doi: 10.1038/s41467-022-30387-4. Nat Commun. 2022. PMID: 35589711 Free PMC article.
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. Neurology. 2019 Oct 22;93(17):e1605-e1617. doi: 10.1212/WNL.0000000000008359. Epub 2019 Oct 2. Neurology. 2019. PMID: 31578300 Free PMC article.
AAV gene therapy for Tay-Sachs disease.
Flotte TR, Cataltepe O, Puri A, Batista AR, Moser R, McKenna-Yasek D, Douthwright C, Gernoux G, Blackwood M, Mueller C, Tai PWL, Jiang X, Bateman S, Spanakis SG, Parzych J, Keeler AM, Abayazeed A, Rohatgi S, Gibson L, Finberg R, Barton BA, Vardar Z, Shazeeb MS, Gounis M, Tifft CJ, Eichler FS, Brown RH Jr, Martin DR, Gray-Edwards HL, Sena-Esteves M. Flotte TR, et al. Nat Med. 2022 Feb;28(2):251-259. doi: 10.1038/s41591-021-01664-4. Epub 2022 Feb 10. Nat Med. 2022. PMID: 35145305 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: douthwright cl. N Engl J Med. 2020 Jul 9;383(2):151-158. doi: 10.1056/NEJMoa2005056. N Engl J Med. 2020. PMID: 32640133