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Page 1
Multispecies-targeting siRNAs for the modulation of JAK1 in the skin.
Tang Q, Gross KY, Fakih HH, Jackson SO, Zain U I Abideen M, Monopoli KR, Blanchard C, Bouix-Peter C, Portal T, Harris JE, Khvorova A, Alterman JF. Tang Q, et al. Among authors: alterman jf. Mol Ther Nucleic Acids. 2024 Jan 9;35(1):102117. doi: 10.1016/j.omtn.2024.102117. eCollection 2024 Mar 12. Mol Ther Nucleic Acids. 2024. PMID: 38304729 Free PMC article.
Single intravitreal administration of a tetravalent siRNA exhibits robust and efficient gene silencing in mouse and pig photoreceptors.
Cheng SY, Caiazzi J, Biscans A, Alterman JF, Echeverria D, McHugh N, Hassler M, Jolly S, Giguere D, Cipi J, Khvorova A, Punzo C. Cheng SY, et al. Among authors: alterman jf. Mol Ther Nucleic Acids. 2023 Dec 5;35(1):102088. doi: 10.1016/j.omtn.2023.102088. eCollection 2024 Mar 12. Mol Ther Nucleic Acids. 2023. PMID: 38192611 Free PMC article.
Rational design of a JAK1-selective siRNA inhibitor for the modulation of autoimmunity in the skin.
Tang Q, Fakih HH, Zain Ui Abideen M, Hildebrand SR, Afshari K, Gross KY, Sousa J, Maebius AS, Bartholdy C, Søgaard PP, Jackerott M, Hariharan V, Summers A, Fan X, Okamura K, Monopoli KR, Cooper DA, Echeverria D, Bramato B, McHugh N, Furgal RC, Dresser K, Winter SJ, Biscans A, Chuprin J, Haddadi NS, Sherman S, Yıldız-Altay Ü, Rashighi M, Richmond JM, Bouix-Peter C, Blanchard C, Clauss A, Alterman JF, Khvorova A, Harris JE. Tang Q, et al. Among authors: alterman jf. Nat Commun. 2023 Nov 4;14(1):7099. doi: 10.1038/s41467-023-42714-4. Nat Commun. 2023. PMID: 37925520 Free PMC article.
Single intravitreal administration of a tetravalent siRNA exhibits robust and efficient gene silencing in rodent and swine photoreceptors.
Cheng SY, Caiazzi J, Biscans A, Alterman JF, Echeverria D, McHugh N, Hassler M, Jolly S, Giguere D, Cipi J, Khvorova A, Punzo C. Cheng SY, et al. Among authors: alterman jf. bioRxiv [Preprint]. 2023 Sep 22:2023.09.20.558641. doi: 10.1101/2023.09.20.558641. bioRxiv. 2023. PMID: 37790464 Free PMC article. Updated. Preprint.
Extended Nucleic Acid (exNA): A Novel, Biologically Compatible Backbone that Significantly Enhances Oligonucleotide Efficacy in vivo.
Yamada K, Hariharan VN, Caiazzi J, Miller R, Furguson C, Sapp E, Fakih H, Tan Q, Yamada N, Furgal RC, Paquette J, Bramato B, McHugh N, Summers A, Lochmann C, Godinho BMDC, Hildebrand S, Echeverria D, Hassler MR, Alterman JF, DiFiglia M, Aronin N, Khvorova A. Yamada K, et al. Among authors: alterman jf. bioRxiv [Preprint]. 2023 May 26:2023.05.26.542506. doi: 10.1101/2023.05.26.542506. bioRxiv. 2023. PMID: 37292886 Free PMC article. Preprint.
Chemical engineering of therapeutic siRNAs for allele-specific gene silencing in Huntington's disease models.
Conroy F, Miller R, Alterman JF, Hassler MR, Echeverria D, Godinho BMDC, Knox EG, Sapp E, Sousa J, Yamada K, Mahmood F, Boudi A, Kegel-Gleason K, DiFiglia M, Aronin N, Khvorova A, Pfister EL. Conroy F, et al. Among authors: alterman jf. Nat Commun. 2022 Oct 3;13(1):5802. doi: 10.1038/s41467-022-33061-x. Nat Commun. 2022. PMID: 36192390 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: alterman jf. 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.
PK-modifying anchors significantly alter clearance kinetics, tissue distribution, and efficacy of therapeutics siRNAs.
Godinho BMDC, Knox EG, Hildebrand S, Gilbert JW, Echeverria D, Kennedy Z, Haraszti RA, Ferguson CM, Coles AH, Biscans A, Caiazzi J, Alterman JF, Hassler MR, Khvorova A. Godinho BMDC, et al. Among authors: alterman jf. Mol Ther Nucleic Acids. 2022 Jun 13;29:116-132. doi: 10.1016/j.omtn.2022.06.005. eCollection 2022 Sep 13. Mol Ther Nucleic Acids. 2022. PMID: 35795486 Free PMC article.
Comparative route of administration studies using therapeutic siRNAs show widespread gene modulation in Dorset sheep.
Ferguson CM, Godinho BM, Alterman JF, Coles AH, Hassler M, Echeverria D, Gilbert JW, Knox EG, Caiazzi J, Haraszti RA, King RM, Taghian T, Puri A, Moser RP, Gounis MJ, Aronin N, Gray-Edwards H, Khvorova A. Ferguson CM, et al. Among authors: alterman jf. JCI Insight. 2021 Dec 22;6(24):e152203. doi: 10.1172/jci.insight.152203. JCI Insight. 2021. PMID: 34935646 Free PMC article.
Structurally constrained phosphonate internucleotide linkage impacts oligonucleotide-enzyme interaction, and modulates siRNA activity and allele specificity.
Yamada K, Hildebrand S, Davis SM, Miller R, Conroy F, Sapp E, Caiazzi J, Alterman JF, Roux L, Echeverria D, Hassler MR, Pfister EL, DiFiglia M, Aronin N, Khvorova A. Yamada K, et al. Among authors: alterman jf. Nucleic Acids Res. 2021 Dec 2;49(21):12069-12088. doi: 10.1093/nar/gkab1126. Nucleic Acids Res. 2021. PMID: 34850120 Free PMC article.
28 results