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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: yin y. 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.
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: yin 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 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: yin y. 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 stereopure chimeric backbone chemistries on the potency and durability of gene silencing by RNA interference.
Liu W, Iwamoto N, Marappan S, Luu K, Tripathi S, Purcell-Estabrook E, Shelke JD, Shah H, Lamattina A, Pan Q, Schrand B, Favaloro F, Bedekar M, Chatterjee A, Desai J, Kawamoto T, Lu G, Metterville J, Samaraweera M, Prakasha PS, Yang H, Yin Y, Yu H, Giangrande PH, Byrne M, Kandasamy P, Vargeese C. Liu W, et al. Among authors: yin y. Nucleic Acids Res. 2023 May 22;51(9):4126-4147. doi: 10.1093/nar/gkad268. Nucleic Acids Res. 2023. PMID: 37070173 Free PMC article.
Stereochemistry Enhances Potency, Efficacy, and Durability of Malat1 Antisense Oligonucleotides In Vitro and In Vivo in Multiple Species.
Byrne M, Vathipadiekal V, Apponi L, Iwamoto N, Kandasamy P, Longo K, Liu F, Looby R, Norwood L, Shah A, Shelke JD, Shivalila C, Yang H, Yin Y, Guo L, Bowman K, Vargeese C. Byrne M, et al. Among authors: yin y. Transl Vis Sci Technol. 2021 Jan 12;10(1):23. doi: 10.1167/tvst.10.1.23. eCollection 2021 Jan. Transl Vis Sci Technol. 2021. PMID: 33510962 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: yin y. Nat Commun. 2021 Feb 8;12(1):847. doi: 10.1038/s41467-021-21112-8. Nat Commun. 2021. PMID: 33558503 Free PMC article.
Control of backbone chemistry and chirality boost oligonucleotide splice switching activity.
Kandasamy P, McClorey G, Shimizu M, Kothari N, Alam R, Iwamoto N, Kumarasamy J, Bommineni GR, Bezigian A, Chivatakarn O, Butler DCD, Byrne M, Chwalenia K, Davies KE, Desai J, Shelke JD, Durbin AF, Ellerington R, Edwards B, Godfrey J, Hoss A, Liu F, Longo K, Lu G, Marappan S, Oieni J, Paik IH, Estabrook EP, Shivalila C, Tischbein M, Kawamoto T, Rinaldi C, Rajão-Saraiva J, Tripathi S, Yang H, Yin Y, Zhao X, Zhou C, Zhang J, Apponi L, Wood MJA, Vargeese C. Kandasamy P, et al. Among authors: yin y. Nucleic Acids Res. 2022 Jun 10;50(10):5443-5466. doi: 10.1093/nar/gkac018. Nucleic Acids Res. 2022. PMID: 35061895 Free PMC article.
Endogenous ADAR-mediated RNA editing in non-human primates using stereopure chemically modified oligonucleotides.
Monian P, Shivalila C, Lu G, Shimizu M, Boulay D, Bussow K, Byrne M, Bezigian A, Chatterjee A, Chew D, Desai J, Favaloro F, Godfrey J, Hoss A, Iwamoto N, Kawamoto T, Kumarasamy J, Lamattina A, Lindsey A, Liu F, Looby R, Marappan S, Metterville J, Murphy R, Rossi J, Pu T, Bhattarai B, Standley S, Tripathi S, Yang H, Yin Y, Yu H, Zhou C, Apponi LH, Kandasamy P, Vargeese C. Monian P, et al. Among authors: yin y. Nat Biotechnol. 2022 Jul;40(7):1093-1102. doi: 10.1038/s41587-022-01225-1. Epub 2022 Mar 7. Nat Biotechnol. 2022. PMID: 35256816
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