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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Hariharan VN, Summers A, Clipperton-Allen AE, et al. (2025) Single-dose administration of therapeutic divalent siRNA targeting MECP2 prevents lethality for one year in an MECP2 duplication mouse model. Research Square |
Hariharan VN, Summers A, Clipperton-Allen AE, et al. (2025) Single-dose administration of therapeutic divalent siRNA targeting MECP2 prevents lethality for one year in an MECP2 duplication mouse model. Biorxiv : the Preprint Server For Biology |
Sapp E, Boudi A, Iwanowicz A, et al. (2025) Detection of HTTex1p by western blot and immunostaining of HD human and mouse brain using neo-epitope antibody P90 highlights impact of CAG repeat expansion on its size, solubility, and response to MSH3 silencing. Biorxiv : the Preprint Server For Biology |
Mathews EW, Coffey SR, Gärtner A, et al. (2024) Suppression of Huntington's Disease Somatic Instability by Transcriptional Repression and Direct CAG Repeat Binding. Biorxiv : the Preprint Server For Biology |
Gilbert JW, Kennedy Z, Godinho BMDC, et al. (2024) Identification of selective and non-selective targeting active siRNAs. Molecular Therapy. Nucleic Acids. 35: 102291 |
Rivera Flores IV, Monopoli K, Jackson S, et al. (2024) Near Sequence Homology Does Not Guarantee siRNA Cross-Species Efficacy. Nucleic Acid Therapeutics |
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 . Nucleic Acid Therapeutics |
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 |