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Page 1
RNAi-mediated silencing of SOD1 profoundly extends survival and functional outcomes in ALS mice.
Weiss A, Gilbert JW, Flores IVR, Belgrad J, Ferguson C, Dogan EO, Wightman N, Mocarski K, Echeverria D, Summers A, Bramato B, McHugh N, Furgal R, Yamada N, Cooper D, Monopoli K, Godinho BMDC, Hassler MR, Yamada K, Greer P, Henninger N, Brown RH Jr, Khvorova A. Weiss A, et al. Among authors: gilbert jw. bioRxiv [Preprint]. 2024 Jun 25:2024.06.20.599943. doi: 10.1101/2024.06.20.599943. bioRxiv. 2024. PMID: 38979291 Free PMC article. Preprint.
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: gilbert jw. 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.
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: gilbert jw. 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.
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: gilbert jw. JCI Insight. 2021 Dec 22;6(24):e152203. doi: 10.1172/jci.insight.152203. JCI Insight. 2021. PMID: 34935646 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: gilbert jw. 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.
Identification of selective and non-selective C9ORF72 targeting in vivo active siRNAs.
Gilbert JW, Kennedy Z, Godinho BMDC, Summers A, Weiss A, Echeverria D, Bramato B, McHugh N, Cooper D, Yamada K, Hassler M, Tran H, Gao FB, Brown RH Jr, Khvorova A. Gilbert JW, et al. Mol Ther Nucleic Acids. 2024 Jul 31;35(3):102291. doi: 10.1016/j.omtn.2024.102291. eCollection 2024 Sep 10. Mol Ther Nucleic Acids. 2024. PMID: 39233852 Free PMC article.
94 results