Anastasia Khvorova
Affiliations: | RNA Therapeutics Institute | University Of Massachusetts Med Sch Worcester |
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Sign in to add traineeReka Agnes Haraszti | grad student | 2014-2018 | University Of Massachusetts Med Sch Worcester |
Daniel O'Reilly | post-doc | 2020- | University of Massachusetts Medical School, USA (Chemistry Tree) |
Bruno M.D.C. Godinho | post-doc | 2015-2019 | University Of Massachusetts Med Sch Worcester |
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Publications
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Yamada K, Hariharan VN, Caiazzi J, et al. (2024) Enhancing siRNA efficacy in vivo with extended nucleic acid backbones. Nature Biotechnology |
Allen S, O'Reilly D, Miller R, et al. (2024) mRNA nuclear clustering leads to a difference in mutant huntingtin mRNA and protein silencing by siRNAs . Biorxiv : the Preprint Server For Biology |
Belgrad J, Tang Q, Hildebrand S, et al. (2024) A programmable dual-targeting siRNA scaffold supports potent two-gene modulation in the central nervous system. Nucleic Acids Research |
Sarli SL, Fakih HH, Kelly K, et al. (2024) Quantifying the activity profile of ASO and siRNA conjugates in glioblastoma xenograft tumors in vivo. Nucleic Acids Research |
Ferguson CM, Godinho BMDC, Echeverria D, et al. (2024) A combinatorial approach for achieving CNS-selective RNAi. Nucleic Acids Research |
Fakih HH, Tang Q, Summers A, et al. (2023) Dendritic amphiphilic siRNA: Selective albumin binding, efficacy, and low toxicity. Molecular Therapy. Nucleic Acids. 34: 102080 |
Zhang H, Kelly K, Lee J, et al. (2023) Self-delivering, chemically modified CRISPR RNAs for AAV co-delivery and genome editing in vivo. Nucleic Acids Research |
Engelbeen S, O'Reilly D, Van De Vijver D, et al. (2023) Challenges of Assessing Exon 53 Skipping of the Human Transcript with Locked Nucleic Acid-Modified Antisense Oligonucleotides in a Mouse Model for Duchenne Muscular Dystrophy. Nucleic Acid Therapeutics. 33: 348-360 |
O'Reilly D, Belgrad J, Ferguson C, et al. (2023) Di-valent siRNA-mediated silencing of MSH3 blocks somatic repeat expansion in mouse models of Huntington's disease. Molecular Therapy : the Journal of the American Society of Gene Therapy |
Hariharan VN, Caiazzi J, Miller R, et al. (2023) Extended Nucleic Acid (exNA): A Novel, Biologically Compatible Backbone that Significantly Enhances Oligonucleotide Efficacy in vivo. Research Square |