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Developing antisense oligonucleotides for a TECPR2 mutation-induced, ultra-rare neurological disorder using patient-derived cellular models.
Williams LA, Gerber DJ, Elder A, Tseng WC, Baru V, Delaney-Busch N, Ambrosi C, Mahimkar G, Joshi V, Shah H, Harikrishnan K, Upadhyay H, Rajendran SH, Dhandapani A, Meier J, Ryan SJ, Lewarch C, Black L, Douville J, Cinquino S, Legakis H, Nalbach K, Behrends C, Sato A, Galluzzi L, Yu TW, Brown D, Agrawal S, Margulies D, Kopin A, Dempsey GT. Williams LA, et al. Among authors: upadhyay h. Mol Ther Nucleic Acids. 2022 Jun 22;29:189-203. doi: 10.1016/j.omtn.2022.06.015. eCollection 2022 Sep 13. Mol Ther Nucleic Acids. 2022. PMID: 35860385 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: upadhyay h. 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.
Deep functional measurements of Fragile X syndrome human neurons reveal multiparametric electrophysiological disease phenotype.
Fink JJ, Delaney-Busch N, Dawes R, Nanou E, Folts C, Harikrishnan K, Hempel C, Upadhyay H, Nguyen T, Shroff H, Stoppel D, Ryan SJ, Jacques J, Grooms J, Berry-Kravis E, Bear MF, Williams LA, Gerber D, Bunnage M, Furey B, Dempsey GT. Fink JJ, et al. Among authors: upadhyay h. Commun Biol. 2024 Nov 6;7(1):1447. doi: 10.1038/s42003-024-07120-6. Commun Biol. 2024. PMID: 39506078 Free PMC article.
Video-based pooled screening yields improved far-red genetically encoded voltage indicators.
Tian H, Davis HC, Wong-Campos JD, Park P, Fan LZ, Gmeiner B, Begum S, Werley CA, Borja GB, Upadhyay H, Shah H, Jacques J, Qi Y, Parot V, Deisseroth K, Cohen AE. Tian H, et al. Among authors: upadhyay h. Nat Methods. 2023 Jul;20(7):1082-1094. doi: 10.1038/s41592-022-01743-5. Epub 2023 Jan 9. Nat Methods. 2023. PMID: 36624211 Free PMC article.
78 results