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Year Number of Results
2017 1
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2019 1
2020 2
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Page 1
Conjugation to a transferrin receptor 1-binding Bicycle peptide enhances ASO and siRNA potency in skeletal and cardiac muscles.
Østergaard ME, Carrer M, Anderson BA, Afetian M, Bakooshli MA, Santos JA, Klein SK, Capitanio J, Freestone GC, Tanowitz M, Galindo-Murillo R, Gaus HJ, Dwyer CA, Jackson M, Jafar-Nejad P, Rigo F, Seth PP, Gaynor KU, Stanway SJ, Urbonas L, St Denis MA, Pellegrino S, Bezerra GA, Rigby M, Gowans E, Van Rietschoten K, Beswick P, Chen L, Skynner MJ, Swayze EE. Østergaard ME, et al. Nucleic Acids Res. 2025 Apr 10;53(7):gkaf270. doi: 10.1093/nar/gkaf270. Nucleic Acids Res. 2025. PMID: 40207629 Free PMC article.
Stathmin-2 loss leads to neurofilament-dependent axonal collapse driving motor and sensory denervation.
López-Erauskin J, Bravo-Hernandez M, Presa M, Baughn MW, Melamed Z, Beccari MS, Agra de Almeida Quadros AR, Arnold-Garcia O, Zuberi A, Ling K, Platoshyn O, Niño-Jara E, Ndayambaje IS, McAlonis-Downes M, Cabrera L, Artates JW, Ryan J, Hermann A, Ravits J, Bennett CF, Jafar-Nejad P, Rigo F, Marsala M, Lutz CM, Cleveland DW, Lagier-Tourenne C. López-Erauskin J, et al. Nat Neurosci. 2024 Jan;27(1):34-47. doi: 10.1038/s41593-023-01496-0. Epub 2023 Nov 23. Nat Neurosci. 2024. PMID: 37996528 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. 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 Free article. Clinical Trial.
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.
Therapeutic antisense oligonucleotide mitigates retinal dysfunction in a pig model of CLN3 Batten disease.
Stratton MP, Centa JL, Swier VJ, Pfeifer WL, Booth CD, Albert K, Hunyara JL, Rechtzigel MJ, Duelli FJ, Leppert HG, Rigo F, Smit T, Jafar-Nejad P, Weimer JM, Drack AV, Hastings ML. Stratton MP, et al. bioRxiv [Preprint]. 2025 May 31:2025.05.30.656864. doi: 10.1101/2025.05.30.656864. bioRxiv. 2025. Update in: Nucleic Acids Res. 2025 Oct 28;53(20):gkaf1141. doi: 10.1093/nar/gkaf1141. PMID: 40501755 Free PMC article. Updated. Preprint.
20 results