Anastasia Khvorova

Affiliations: 
RNA Therapeutics Institute University Of Massachusetts Med Sch Worcester 
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"Anastasia Khvorova"
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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
Tang Q, Khvorova A. (2024) RNAi-based drug design: considerations and future directions. Nature Reviews. Drug Discovery
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
Yamada K, Hariharan VN, Caiazzi J, et al. (2023) Extended Nucleic Acid (exNA): A Novel, Biologically Compatible Backbone that Significantly Enhances Oligonucleotide Efficacy . Biorxiv : the Preprint Server For Biology
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