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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: roux l. Nucleic Acids Res. 2018 Mar 16;46(5):2185-2196. doi: 10.1093/nar/gky037. Nucleic Acids Res. 2018. PMID: 29432571 Free PMC article.
Pharmacokinetic Profiling of Conjugated Therapeutic Oligonucleotides: A High-Throughput Method Based Upon Serial Blood Microsampling Coupled to Peptide Nucleic Acid Hybridization Assay.
Godinho BMDC, Gilbert JW, Haraszti RA, Coles AH, Biscans A, Roux L, Nikan M, Echeverria D, Hassler M, Khvorova A. Godinho BMDC, et al. Among authors: roux l. Nucleic Acid Ther. 2017 Dec;27(6):323-334. doi: 10.1089/nat.2017.0690. Epub 2017 Oct 12. Nucleic Acid Ther. 2017. PMID: 29022758 Free PMC article.
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. Among authors: roux l. 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.
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: roux l. Nucleic Acids Res. 2021 Dec 2;49(21):12069-12088. doi: 10.1093/nar/gkab1126. Nucleic Acids Res. 2021. PMID: 34850120 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: roux l. 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.
Gene therapy with AR isoform 2 rescues spinal and bulbar muscular atrophy phenotype by modulating AR transcriptional activity.
Lim WF, Forouhan M, Roberts TC, Dabney J, Ellerington R, Speciale AA, Manzano R, Lieto M, Sangha G, Banerjee S, Conceição M, Cravo L, Biscans A, Roux L, Pourshafie N, Grunseich C, Duguez S, Khvorova A, Pennuto M, Cortes CJ, La Spada AR, Fischbeck KH, Wood MJA, Rinaldi C. Lim WF, et al. Among authors: roux l. Sci Adv. 2021 Aug 20;7(34):eabi6896. doi: 10.1126/sciadv.abi6896. Print 2021 Aug. Sci Adv. 2021. PMID: 34417184 Free PMC article.
267 results