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Page 1
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. Among authors: hassler m. 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.
Cell Type Impacts Accessibility of mRNA to Silencing by RNA Interference.
Ferguson CM, Echeverria D, Hassler M, Ly S, Khvorova A. Ferguson CM, et al. Among authors: hassler m. Mol Ther Nucleic Acids. 2020 Sep 4;21:384-393. doi: 10.1016/j.omtn.2020.06.006. Epub 2020 Jun 12. Mol Ther Nucleic Acids. 2020. PMID: 32650236 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: hassler mr. 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.
A combinatorial approach for achieving CNS-selective RNAi.
Ferguson CM, Godinho BMDC, Echeverria D, Hassler M, Vangjeli L, Sousa J, McHugh N, Alterman J, Hariharan V, Krishnamurthy PM, Watts J, Rogaev E, Khvorova A. Ferguson CM, et al. Among authors: hassler m. Nucleic Acids Res. 2024 May 22;52(9):5273-5284. doi: 10.1093/nar/gkae100. Nucleic Acids Res. 2024. PMID: 38348876 Free PMC article.
Hydrophobically Modified siRNAs Silence Huntingtin mRNA in Primary Neurons and Mouse Brain.
Alterman JF, Hall LM, Coles AH, Hassler MR, Didiot MC, Chase K, Abraham J, Sottosanti E, Johnson E, Sapp E, Osborn MF, Difiglia M, Aronin N, Khvorova A. Alterman JF, et al. Among authors: hassler mr. Mol Ther Nucleic Acids. 2015 Dec 1;4(12):e266. doi: 10.1038/mtna.2015.38. Mol Ther Nucleic Acids. 2015. PMID: 26623938 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: hassler mr. 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.
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: hassler m. 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.
198 results