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Roadmap for C9ORF72 in Frontotemporal Dementia and Amyotrophic Lateral Sclerosis: Report on the C9ORF72 FTD/ALS Summit.
Sattler R, Traynor BJ, Robertson J, Van Den Bosch L, Barmada SJ, Svendsen CN, Disney MD, Gendron TF, Wong PC, Turner MR, Boxer A, Babu S, Benatar M, Kurnellas M, Rohrer JD, Donnelly CJ, Bustos LM, Van Keuren-Jensen K, Dacks PA, Sabbagh MN; Attendees of the inaugural C9ORF72 FTD/ALS Summit. Sattler R, et al. Neurol Ther. 2023 Dec;12(6):1821-1843. doi: 10.1007/s40120-023-00548-8. Epub 2023 Oct 17. Neurol Ther. 2023. PMID: 37847372 Free PMC article.
Antisense oligonucleotides targeting the miR-29b binding site in the GRN mRNA increase progranulin translation.
Aggarwal G, Banerjee S, Jones SA, Benchaar Y, Bélanger J, Sévigny M, Smith DM, Niehoff ML, Pavlack M, de Vera IMS, Petkau TL, Leavitt BR, Ling K, Jafar-Nejad P, Rigo F, Morley JE, Farr SA, Dutchak PA, Sephton CF, Nguyen AD. Aggarwal G, et al. J Biol Chem. 2023 Dec;299(12):105475. doi: 10.1016/j.jbc.2023.105475. Epub 2023 Nov 18. J Biol Chem. 2023. PMID: 37981208 Free PMC article.
Safety, tolerability, and pharmacokinetics of antisense oligonucleotide BIIB078 in adults with C9orf72-associated amyotrophic lateral sclerosis: a phase 1, randomised, double blinded, placebo-controlled, multiple ascending dose study.
van den Berg LH, Rothstein JD, Shaw PJ, Babu S, Benatar M, Bucelli RC, Genge A, Glass JD, Hardiman O, Libri V, Mobach T, Oskarsson B, Pattee GL, Ravits J, Shaw CE, Weber M, Zinman L, Jafar-Nejad P, Rigo F, Lin L, Ferguson TA, Gotter AL, Graham D, Monine M, Inra J, Sinks S, Eraly S, Garafalo S, Fradette S. van den Berg LH, et al. Among authors: jafar nejad p. Lancet Neurol. 2024 Sep;23(9):901-912. doi: 10.1016/S1474-4422(24)00216-3. Epub 2024 Jul 23. Lancet Neurol. 2024. PMID: 39059407 Clinical Trial.
PFTK1 kinase regulates axogenesis during development via RhoA activation.
González YR, Kamkar F, Jafar-Nejad P, Wang S, Qu D, Alvarez LS, Hawari D, Sonnenfeld M, Slack RS, Albert PR, Park DS, Joselin A. González YR, et al. BMC Biol. 2023 Oct 31;21(1):240. doi: 10.1186/s12915-023-01732-w. BMC Biol. 2023. PMID: 37907898 Free PMC article.
59 results