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A divalent siRNA chemical scaffold for potent and sustained modulation of gene expression throughout the central nervous system.
Alterman JF, Godinho BMDC, Hassler MR, Ferguson CM, Echeverria D, Sapp E, Haraszti RA, Coles AH, Conroy F, Miller R, Roux L, Yan P, Knox EG, Turanov AA, King RM, Gernoux G, Mueller C, Gray-Edwards HL, Moser RP, Bishop NC, Jaber SM, Gounis MJ, Sena-Esteves M, Pai AA, DiFiglia M, Aronin N, Khvorova A. Alterman JF, et al. Nat Biotechnol. 2019 Aug;37(8):884-894. doi: 10.1038/s41587-019-0205-0. Epub 2019 Aug 2. Nat Biotechnol. 2019. PMID: 31375812 Free PMC article.
Exosome-mediated Delivery of Hydrophobically Modified siRNA for Huntingtin mRNA Silencing.
Didiot MC, Hall LM, Coles AH, Haraszti RA, Godinho BM, Chase K, Sapp E, Ly S, Alterman JF, Hassler MR, Echeverria D, Raj L, Morrissey DV, DiFiglia M, Aronin N, Khvorova A. Didiot MC, et al. Among authors: alterman jf. Mol Ther. 2016 Oct;24(10):1836-1847. doi: 10.1038/mt.2016.126. Epub 2016 Jun 27. Mol Ther. 2016. PMID: 27506293 Free PMC article.
Comparison of partially and fully chemically-modified siRNA in conjugate-mediated delivery in vivo.
Hassler MR, Turanov AA, Alterman JF, Haraszti RA, Coles AH, Osborn MF, Echeverria D, Nikan M, Salomon WE, Roux L, Godinho BMDC, Davis SM, Morrissey DV, Zamore PD, Karumanchi SA, Moore MJ, Aronin N, Khvorova A. Hassler MR, et al. Among authors: alterman jf. Nucleic Acids Res. 2018 Mar 16;46(5):2185-2196. doi: 10.1093/nar/gky037. Nucleic Acids Res. 2018. PMID: 29432571 Free PMC article.
Transvascular Delivery of Hydrophobically Modified siRNAs: Gene Silencing in the Rat Brain upon Disruption of the Blood-Brain Barrier.
Godinho BMDC, Henninger N, Bouley J, Alterman JF, Haraszti RA, Gilbert JW, Sapp E, Coles AH, Biscans A, Nikan M, Echeverria D, DiFiglia M, Aronin N, Khvorova A. Godinho BMDC, et al. Among authors: alterman jf. Mol Ther. 2018 Nov 7;26(11):2580-2591. doi: 10.1016/j.ymthe.2018.08.005. Epub 2018 Aug 8. Mol Ther. 2018. PMID: 30143435 Free PMC article.
RNAi modulation of placental sFLT1 for the treatment of preeclampsia.
Turanov AA, Lo A, Hassler MR, Makris A, Ashar-Patel A, Alterman JF, Coles AH, Haraszti RA, Roux L, Godinho BMDC, Echeverria D, Pears S, Iliopoulos J, Shanmugalingam R, Ogle R, Zsengeller ZK, Hennessy A, Karumanchi SA, Moore MJ, Khvorova A. Turanov AA, et al. Among authors: alterman jf. Nat Biotechnol. 2018 Nov 19:10.1038/nbt.4297. doi: 10.1038/nbt.4297. Online ahead of print. Nat Biotechnol. 2018. PMID: 30451990 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.
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.
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.
28 results