Khvorova, Anastasia

Minimal information for studies of extracellular vesicles 2018 (MISEV2018): a position statement of the International Society for Extracellular Vesicles and update of the MISEV2014 guidelines

Théry, C., Witwer, K. W., Aikawa, E., Alcaraz, M. J., Anderson, J. D., Andriantsitohaina, R., Antoniou, A., Arab, T., Archer, F., Atkin‐Smith, G. K., et al. (2018) · Journal of Extracellular Vesicles

Extracellular vesicles in diseaseCellular transport and secretion

Cited by 11,099

Functional siRNAs and miRNAs exhibit strand bias

Khvorova, A., Reynolds, A., & Jayasena, S. D. (2003) · Cell

RNA Interference and Gene DeliveryRNA and protein synthesis mechanisms

Cited by 2,645

Rational siRNA design for RNA interference

Reynolds, A., Leake, D., Boese, Q., Scaringe, S., Marshall, W. S., & Khvorova, A. (2004) · Nature Biotechnology

RNA Interference and Gene DeliveryCRISPR and Genetic Engineering

Cited by 2,094

The chemical evolution of oligonucleotide therapies of clinical utility

Khvorova, A., & Watts, J. K. (2017) · Nature Biotechnology

RNA Interference and Gene DeliveryDNA and Nucleic Acid Chemistry

Cited by 1,216

3′ UTR seed matches, but not overall identity, are associated with RNAi off-targets

Birmingham, A., Anderson, E. M., Reynolds, A., Ilsley-Tyree, D., Leake, D., Fedorov, Y., Baskerville, S., Maksimova, E., Robinson, K., Karpilow, J., et al. (2006) · Nature Methods

RNA Interference and Gene DeliveryCRISPR and Genetic Engineering

Cited by 920

High-resolution proteomic and lipidomic analysis of exosomes and microvesicles from different cell sources

Haraszti, R. A., Didiot, M., Sapp, E., Leszyk, J., Shaffer, S. A., Rockwell, H. E., Gao, F., Narain, N. R., DiFiglia, M., Kiebish, M. A., et al. (2016) · Journal of Extracellular Vesicles

Extracellular vesicles in diseaseNanoparticle-Based Drug Delivery

Cited by 746

Exosomes Produced from 3D Cultures of MSCs by Tangential Flow Filtration Show Higher Yield and Improved Activity

Haraszti, R. A., Miller, R., Stoppato, M., Sere, Y. Y., Coles, A., Didiot, M.-C., Wollacott, R., Sapp, E., Dubuke, M. L., Li, X., et al. (2018) · Molecular Therapy

Extracellular vesicles in diseaseMesenchymal stem cell research

Cited by 567

Exosome-mediated delivery of hydrophobically modified siRNA for huntingtin mRNA silencing

Didiot, M.-C., Hall, L. M., Coles, A. H., Haraszti, R. A., Godinho, B. M., Chase, K., Sapp, E., Ly, S., Alterman, J. F., Hassler, M. R., et al. (2016) · Molecular Therapy

RNA Interference and Gene DeliveryExtracellular vesicles in disease

Cited by 483

A divalent siRNA chemical scaffold for potent and sustained modulation of gene expression throughout the central nervous system

Alterman, J. F., Godinho, B. M. D. C., Hassler, M. R., Ferguson, C. M., Echeverria, D., Sapp, E., Haraszti, R. A., Coles, A. H., Conroy, F., Miller, R., et al. (2019) · Nature Biotechnology

RNA Interference and Gene DeliveryDNA and Nucleic Acid Chemistry

Cited by 227

Comparison of partially and fully chemically-modified siRNA in conjugate-mediated delivery in vivo

Hassler, M. R., Turanov, A. A., Alterman, J. F., Haraszti, R. A., Coles, A. H., Osborn, M. F., Echeverria, D., Nikan, M., Salomon, W. E., Roux, L., et al. (2018) · Nucleic Acids Research

RNA Interference and Gene DeliveryNanoparticle-Based Drug Delivery

Cited by 181

Hydrophobically Modified siRNAs Silence Huntingtin mRNA in Primary Neurons and Mouse Brain

Alterman, J. F., Hall, L. M., Coles, A. H., Hassler, M. R., Didiot, M.-C., Chase, K., Abraham, J., Sottosanti, E., Johnson, E., Sapp, E., et al. (2015) · Molecular Therapy - Nucleic Acids

RNA Interference and Gene DeliveryGenetic Neurodegenerative Diseases

Cited by 158

Improving siRNA Delivery In Vivo Through Lipid Conjugation

Osborn, M. F., & Khvorova, A. (2018) · Nucleic Acid Therapeutics

RNA Interference and Gene DeliveryNanoparticle-Based Drug Delivery

Cited by 137

Visualization of self-delivering hydrophobically modified siRNA cellular internalization

Ly, S., Navaroli, D. M., Didiot, M.-C., Cardia, J., Pandarinathan, L., Alterman, J. F., Fogarty, K., Standley, C., Lifshitz, L. M., Bellve, K. D., et al. (2017) · Nucleic Acids Research

RNA Interference and Gene DeliveryCellular transport and secretion

Cited by 132

Binding and disruption of phospholipid bilayers by supramolecular RNA complexes

Vlassov, A., Khvorova, A., & Yarus, M. (2001) · Proceedings of the National Academy of Sciences

RNA and protein synthesis mechanismsLipid Membrane Structure and Behavior

Cited by 105

Docosahexaenoic Acid Conjugation Enhances Distribution and Safety of siRNA upon Local Administration in Mouse Brain

Nikan, M., Osborn, M. F., Coles, A. H., Godinho, B. M., Hall, L. M., Haraszti, R. A., Hassler, M. R., Echeverria, D., Aronin, N., & Khvorova, A. (2016) · Molecular Therapy - Nucleic Acids

RNA Interference and Gene DeliveryNanoparticle-Based Drug Delivery

Cited by 85

Nuclear Localization of Huntingtin mRNA Is Specific to Cells of Neuronal Origin

Didiot, M.-C., Ferguson, C. M., Ly, S., Coles, A. H., Smith, A. O., Bicknell, A. A., Hall, L. M., Sapp, E., Echeverria, D., Pai, A. A., et al. (2018) · Cell Reports

Genetic Neurodegenerative DiseasesRNA Interference and Gene Delivery

Cited by 64

Pharmacokinetic Profiling of Conjugated Therapeutic Oligonucleotides: A High-Throughput Method Based Upon Serial Blood Microsampling Coupled to Peptide Nucleic Acid Hybridization Assay

Godinho, B. M. D. C., Gilbert, J. W., Haraszti, R. A., Coles, A. H., Biscans, A., Roux, L., Nikan, M., Echeverria, D., Hassler, M., & Khvorova, A. (2017) · Nucleic Acid Therapeutics

RNA Interference and Gene DeliveryBiosimilars and Bioanalytical Methods

Cited by 52

An RNAi therapeutic targeting hepatic DGAT2 in a genetically obese mouse model of nonalcoholic steatohepatitis

Yenilmez, B., Wetoska, N., Kelly, M., Echeverria, D., Min, K., Lifshitz, L., Alterman, J. F., Hassler, M. R., Hildebrand, S., DiMarzio, C., et al. (2022) · Molecular Therapy

Liver Disease Diagnosis and TreatmentRNA Interference and Gene Delivery

Cited by 46

Synthesis and Evaluation of Parenchymal Retention and Efficacy of a Metabolically Stable O-Phosphocholine-N-docosahexaenoyl-l-serine siRNA Conjugate in Mouse Brain

Nikan, M., Osborn, M. F., Coles, A. H., Biscans, A., Godinho, B. M. D. C., Haraszti, R. A., Sapp, E., Echeverria, D., DiFiglia, M., Aronin, N., et al. (2017) · Bioconjugate Chemistry

RNA Interference and Gene DeliveryNanoparticle-Based Drug Delivery

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Advancements in APOE and dementia research: Highlights from the 2023 AAIC Advancements: APOE conference

Kloske, C. M., Belloy, M. E., Blue, E. E., Bowman, G. R., Carrillo, M. C., Chen, X., Chiba‐Falek, O., Davis, A. A., Paolo, G. D., Garretti, F., et al. (2024) · Alzheimer's & Dementia

Alzheimer's disease research and treatmentsDementia and Cognitive Impairment Research

Cited by 33

Comparative route of administration studies using therapeutic siRNAs show widespread gene modulation in Dorset sheep

Ferguson, C. M., Godinho, B. M. D. C., Alterman, J. F., Coles, A. H., Hassler, M., Echeverria, D., Gilbert, J. W., Knox, E. G., Caiazzi, J., Haraszti, R. A., et al. (2021) · JCI Insight

RNA Interference and Gene DeliveryBarrier Structure and Function Studies

Cited by 27

Quantifying the activity profile of ASO and siRNA conjugates in glioblastoma xenograft tumors in vivo

Sarli, S. L., Fakih, H. H., Kelly, K., Devi, G., Rembetsy-Brown, J. M., McEachern, H. R., Ferguson, C. M., Echeverria, D., Lee, J., Sousa, J., et al. (2024) · Nucleic Acids Research

RNA Interference and Gene DeliveryDNA and Nucleic Acid Chemistry

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