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Development of a sensitive trial-ready poly(GP) CSF biomarker assay for C9orf72-associated frontotemporal dementia and amyotrophic lateral sclerosis.
Wilson KM, Katona E, Glaria I, Carcolé M, Swift IJ, Sogorb-Esteve A, Heller C, Bouzigues A, Heslegrave AJ, Keshavan A, Knowles K, Patil S, Mohapatra S, Liu Y, Goyal J, Sanchez-Valle R, Laforce RJ, Synofzik M, Rowe JB, Finger E, Vandenberghe R, Butler CR, Gerhard A, Van Swieten JC, Seelaar H, Borroni B, Galimberti D, de Mendonça A, Masellis M, Tartaglia MC, Otto M, Graff C, Ducharme S, Schott JM, Malaspina A, Zetterberg H, Boyanapalli R, Rohrer JD, Isaacs AM; Genetic FTD Initiative (GENFI). Wilson KM, et al. Among authors: liu y. J Neurol Neurosurg Psychiatry. 2022 Jul;93(7):761-771. doi: 10.1136/jnnp-2021-328710. Epub 2022 Apr 4. J Neurol Neurosurg Psychiatry. 2022. PMID: 35379698 Free PMC article.
Variant-selective stereopure oligonucleotides protect against pathologies associated with C9orf72-repeat expansion in preclinical models.
Liu Y, Dodart JC, Tran H, Berkovitch S, Braun M, Byrne M, Durbin AF, Hu XS, Iwamoto N, Jang HG, Kandasamy P, Liu F, Longo K, Ruschel J, Shelke J, Yang H, Yin Y, Donner A, Zhong Z, Vargeese C, Brown RH Jr. Liu Y, et al. Among authors: liu f. Nat Commun. 2021 Feb 8;12(1):847. doi: 10.1038/s41467-021-21112-8. Nat Commun. 2021. PMID: 33558503 Free PMC article.
Preclinical evaluation of WVE-004, aninvestigational stereopure oligonucleotide forthe treatment of C9orf72-associated ALS or FTD.
Liu Y, Andreucci A, Iwamoto N, Yin Y, Yang H, Liu F, Bulychev A, Hu XS, Lin X, Lamore S, Patil S, Mohapatra S, Purcell-Estabrook E, Taborn K, Dale E, Vargeese C. Liu Y, et al. Among authors: liu f. Mol Ther Nucleic Acids. 2022 Apr 20;28:558-570. doi: 10.1016/j.omtn.2022.04.007. eCollection 2022 Jun 14. Mol Ther Nucleic Acids. 2022. PMID: 35592494 Free PMC article.
Impact of guanidine-containing backbone linkages on stereopure antisense oligonucleotides in the CNS.
Kandasamy P, Liu Y, Aduda V, Akare S, Alam R, Andreucci A, Boulay D, Bowman K, Byrne M, Cannon M, Chivatakarn O, Shelke JD, Iwamoto N, Kawamoto T, Kumarasamy J, Lamore S, Lemaitre M, Lin X, Longo K, Looby R, Marappan S, Metterville J, Mohapatra S, Newman B, Paik IH, Patil S, Purcell-Estabrook E, Shimizu M, Shum P, Standley S, Taborn K, Tripathi S, Yang H, Yin Y, Zhao X, Dale E, Vargeese C. Kandasamy P, et al. Among authors: liu y. Nucleic Acids Res. 2022 Jun 10;50(10):5401-5423. doi: 10.1093/nar/gkac037. Nucleic Acids Res. 2022. PMID: 35106589 Free PMC article.
Preclinical evaluation of stereopure antisense oligonucleotides for allele-selective lowering of mutant HTT.
Iwamoto N, Liu Y, Frank-Kamenetsky M, Maguire A, Tseng WC, Taborn K, Kothari N, Akhtar A, Bowman K, Shelke JD, Lamattina A, Hu XS, Jang HG, Kandasamy P, Liu F, Longo K, Looby R, Meena, Metterville J, Pan Q, Purcell-Estabrook E, Shimizu M, Prakasha PS, Standley S, Upadhyay H, Yang H, Yin Y, Zhao A, Francis C, Byrne M, Dale E, Verdine GL, Vargeese C. Iwamoto N, et al. Among authors: liu y, liu f. Mol Ther Nucleic Acids. 2024 Jun 11;35(3):102246. doi: 10.1016/j.omtn.2024.102246. eCollection 2024 Sep 10. Mol Ther Nucleic Acids. 2024. PMID: 39027419 Free PMC article.
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