Author

A. Khvorova

Listed by name: these papers carry no ORCID for this author, so different people with the same name may be mixed together.

Biology Disciplines

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Biochemistry· 3 Pharmacology & Toxicology· 1

Signature Assays, Organisms & Methods

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Research Focus:drug response / drug discovery· 4 Approach:experimental assay· 4 Research Focus:gene regulatory network inference· 2 Research Focus:cell type annotation· 1 Assay:CRISPR screen· 1 Research Focus:differential expression· 1 Organism:human· 1 Research Focus:variant calling· 1

Frequent Co-Authors

D. Echeverria· 4 shared A. Biscans· 2 shared M. Hassler· 2 shared M. R. Hassler· 2 shared A. Coles· 1 shared A. H. Coles· 1 shared A. J. Debacker· 1 shared A. Mir· 1 shared

Activity by Year

2018-2023 · peak 3/yr

Co-Author Collaboration Network

10 top collaborators · 21 mutual co-author links

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4 shared papersBiochemistry

D. Echeverria

Mutual co-authors in cluster

Author profile →

4 papers

Recent·Most cited

Extended Nucleic Acid (exNA): A Novel, Biologically Compatible Backbone that Significantly Enhances Oligonucleotide Efficacy in vivo

K. Yamada, V. N. Hariharan, J. Caiazzi, R. Miller, C. Ferguson, E. Sapp, H. Fakih, Q. Tan, N. Yamada, R. Furgal, J. Paquette, B. Bramato, N. McHugh, A. Summer, C. Lochmann, , A. Khvorova

This paper introduces exNA, a novel nucleic acid backbone that enhances oligonucleotide stability and efficacy in vivo, showing improved resistance to exonucleases and increased tissue exposure and potency.

bioRxiv · May 26, 2023Biochemistry

Heavily and Fully Modified RNAs Guide Efficient SpyCas9-Mediated Genome Editing

A. Mir, J. F. Alterman, M. R. Hassler, A. J. Debacker, E. Hudgens, D. Echeverria, M. H. Brodsky, A. Khvorova, J. K. Watts, E. J. Sontheimer

This study identifies heavily and fully modified crRNA and tracrRNA that enhance Cas9-mediated genome editing efficiency and stability in human cells.

bioRxiv · Mar 28, 2018Biochemistry

Diverse lipid conjugates for functional extra-hepatic siRNA delivery in vivo

A. Biscans, A. Coles, R. Haraszti, D. Echeverria, M. Hassler, M. Osborn, A. Khvorova

This study develops lipophilic conjugates to enable siRNA delivery to extra-hepatic tissues, showing effective gene silencing in multiple organs.

bioRxiv · Mar 26, 2018Biochemistry

Hydrophobicity drives the systemic distribution of lipid-conjugated siRNAs via lipid transport pathways

M. F. Osborn, A. H. Coles, A. Biscans, R. A. Haraszti, L. Roux, S. Davis, S. Ly, D. Echeverria, M. R. Hassler, B. M. D. C. Godinho, M. Nikan, A. Khvorova

This study shows that lipid conjugation affects siRNA hydrophobicity, influencing its distribution via lipoprotein pathways and enabling tissue-specific mRNA silencing without toxicity.

bioRxiv · Mar 23, 2018Pharmacology & Toxicology