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2021 1
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Page 1
Enhancing siRNA efficacy in vivo with extended nucleic acid backbones.
Yamada K, Hariharan VN, Caiazzi J, Miller R, Ferguson CM, Sapp E, Fakih HH, Tang Q, Yamada N, Furgal RC, Paquette JD, Biscans A, Bramato BM, McHugh N, Summers A, Lochmann C, Godinho BMDC, Hildebrand S, Jackson SO, Echeverria D, Hassler MR, Alterman JF, DiFiglia M, Aronin N, Khvorova A. Yamada K, et al. Among authors: hariharan vn. Nat Biotechnol. 2025 Jun;43(6):904-913. doi: 10.1038/s41587-024-02336-7. Epub 2024 Aug 1. Nat Biotechnol. 2025. PMID: 39090305
Systematic analysis of siRNA and mRNA features impacting fully chemically modified siRNA efficacy.
Davis SM, Hildebrand S, MacMillan HJ, Monopoli KR, Buchwald J, Sousa J, Cooper D, Ly S, Echeverria D, McHugh N, Ferguson C, Coles A, Hariharan VN, O'Reilly D, Tang Q, Furgal R, Yamada K, Alterman JF, Gilbert JW, Knox E, Pineda Y, Weston CN, Baer CE, Pai AA, Khvorova A. Davis SM, et al. Among authors: hariharan vn. Nucleic Acids Res. 2025 Jun 20;53(12):gkaf479. doi: 10.1093/nar/gkaf479. Nucleic Acids Res. 2025. PMID: 40548938 Free PMC article.
Divalent siRNAs are bioavailable in the lung and efficiently block SARS-CoV-2 infection.
Hariharan VN, Shin M, Chang CW, O'Reilly D, Biscans A, Yamada K, Guo Z, Somasundaran M, Tang Q, Monopoli K, Krishnamurthy PM, Devi G, McHugh N, Cooper DA, Echeverria D, Cruz J, Chan IL, Liu P, Lim SY, McConnell J, Singh SP, Hildebrand S, Sousa J, Davis SM, Kennedy Z, Ferguson C, Godinho BMDC, Thillier Y, Caiazzi J, Ly S, Muhuri M, Kelly K, Humphries F, Cousineau A, Parsi KM, Li Q, Wang Y, Maehr R, Gao G, Korkin D, McDougall WM, Finberg RW, Fitzgerald KA, Wang JP, Watts JK, Khvorova A. Hariharan VN, et al. Proc Natl Acad Sci U S A. 2023 Mar 14;120(11):e2219523120. doi: 10.1073/pnas.2219523120. Epub 2023 Mar 9. Proc Natl Acad Sci U S A. 2023. PMID: 36893269 Free PMC article.
Dendritic amphiphilic siRNA: Selective albumin binding, in vivo efficacy, and low toxicity.
Fakih HH, Tang Q, Summers A, Shin M, Buchwald JE, Gagnon R, Hariharan VN, Echeverria D, Cooper DA, Watts JK, Khvorova A, Sleiman HF. Fakih HH, et al. Among authors: hariharan vn. Mol Ther Nucleic Acids. 2023 Nov 17;34:102080. doi: 10.1016/j.omtn.2023.102080. eCollection 2023 Dec 12. Mol Ther Nucleic Acids. 2023. PMID: 38089931 Free PMC article.
Chemical optimization of siRNA for safe and efficient silencing of placental sFLT1.
Davis SM, Hariharan VN, Lo A, Turanov AA, Echeverria D, Sousa J, McHugh N, Biscans A, Alterman JF, Karumanchi SA, Moore MJ, Khvorova A. Davis SM, et al. Among authors: hariharan vn. Mol Ther Nucleic Acids. 2022 Jun 22;29:135-149. doi: 10.1016/j.omtn.2022.06.009. eCollection 2022 Sep 13. Mol Ther Nucleic Acids. 2022. PMID: 35847173 Free PMC article.
Single-dose administration of therapeutic divalent siRNA targeting MECP2 prevents lethality for one year in an MECP2 duplication mouse model.
Hariharan VN, Summers A, Clipperton-Allen AE, Caiazzi J, Hildebrand SR, O'Reilly D, Tang Q, Kennedy Z, Echeverria D, McHugh N, Cooper D, Souza J, Ferguson C, Bogdanik L, Coenraads M, Khvorova A. Hariharan VN, et al. bioRxiv [Preprint]. 2025 Mar 29:2025.03.26.645328. doi: 10.1101/2025.03.26.645328. bioRxiv. 2025. PMID: 40196492 Free PMC article. Preprint.
Single-dose administration of therapeutic divalent siRNA targeting MECP2 prevents lethality for one year in an MECP2 duplication mouse model.
Hariharan VN, Summers A, Clipperton-Allen AE, Caiazzi J, Hildebrand SR, O' Reilly D, Tang Q, Kennedy Z, Echeverria D, McHugh N, Cooper D, Souza J, Ferguson C, Bogdanik L, Coenraads M, Khvorova A. Hariharan VN, et al. Res Sq [Preprint]. 2025 Apr 25:rs.3.rs-6465542. doi: 10.21203/rs.3.rs-6465542/v1. Res Sq. 2025. PMID: 40313754 Free PMC article. Preprint.
Extended Nucleic Acid (exNA): A Novel, Biologically Compatible Backbone that Significantly Enhances Oligonucleotide Efficacy in vivo.
Hariharan VN, Caiazzi J, Miller R, Ferguson C, Sapp E, Fakih H, Tang Q, Yamada N, Furgal R, Paquette J, Bramato B, McHugh N, Summers A, Lochmann C, Godinho B, Hildebrand S, Echeverria D, Hassler M, Alterman J, DiFiglia M, Aronin N, Khvorova A, Yamada K. Hariharan VN, et al. Res Sq [Preprint]. 2023 Jun 1:rs.3.rs-2987323. doi: 10.21203/rs.3.rs-2987323/v1. Res Sq. 2023. Update in: Nat Biotechnol. 2025 Jun;43(6):904-913. doi: 10.1038/s41587-024-02336-7. PMID: 37398145 Free PMC article. Updated. Preprint.
11 results