Anastasia Khvorova

Affiliations: 
RNA Therapeutics Institute University Of Massachusetts Med Sch Worcester 
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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
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