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A. Khvorova · SciRah

Researcher profile · Semantic Scholar

A. Khvorova

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A. Khvorova — researcher profile on SciRah

h-index: 59
Works: 249
Citations: 31K

Most-cited papers

RNAi-based drug design: considerations and future directions
Qi Tang, A. Khvorova
2024Nature reviews. Drug discovery262 citations Open access
Semantic Scholar
Correction to “Minimal information for studies of extracellular vesicles (MISEV2023): From basic to advanced approaches”
Delaram Khamari, E. Khomyakova, A. Khvorova, Peter Kierulf +159 more
2024Journal of Extracellular Vesicles256 citations Open access
Semantic Scholar
Nucleic Acid Therapeutics: Successes, Milestones, and Upcoming Innovation
Jillian Belgrad, H. Fakih, A. Khvorova
2024Nucleic Acid Therapeutics109 citations Open access
Semantic Scholar
The NIH Somatic Cell Genome Editing program
Krishanu Saha, E. Sontheimer, P. Brooks, M. Dwinell +71 more
2021Nature106 citations Open access
Semantic Scholar
AIM2 regulates anti-tumor immunity and is a viable therapeutic target for melanoma
K. Fukuda, Ken Okamura, R. Riding, Xueli Fan +11 more
2021Journal of Experimental Medicine66 citations Open access
Semantic Scholar
Enhancing siRNA efficacy in vivo with extended nucleic acid backbones
Ken Yamada, V. Hariharan, Jillian Caiazzi, Rachael Miller +21 more
2024Nature Biotechnology50 citations Open access
Semantic Scholar
Chemical optimization of siRNA for safe and efficient silencing of placental sFLT1
Sarah M Davis, V. Hariharan, Agnes Lo, A. A. Turanov +8 more
2022Molecular Therapy: Nucleic Acids50 citations Open access
Semantic Scholar
Rational design of a JAK1-selective siRNA inhibitor for the modulation of autoimmunity in the skin
Qi Tang, H. Fakih, Mohammad Zain Ui Abideen, Samuel Hildebrand +32 more
2023Nature Communications47 citations Open access
Semantic Scholar
Engineered ionizable lipid siRNA conjugates enhance endosomal escape but induce toxicity in vivo
Annabelle Biscans, Socheata Ly, Nicholas McHugh, David Cooper +1 more
2022Journal of Controlled Release45 citations Open access
Semantic Scholar
Divalent siRNAs are bioavailable in the lung and efficiently block SARS-CoV-2 infection
V. Hariharan, Min-Kyu Shin, Ching-Wen Chang, D. O'reilly +42 more
2023Proceedings of the National Academy of Sciences of the United States of America44 citations Open access
Semantic Scholar
Di-valent siRNA-mediated silencing of MSH3 blocks somatic repeat expansion in mouse models of Huntington’s disease
D. O'reilly, Jillian Belgrad, Chantal M. Ferguson, Ashley Summers +17 more
2023Molecular Therapy39 citations Open access
Semantic Scholar
Chemical engineering of therapeutic siRNAs for allele-specific gene silencing in Huntington’s disease models
Faith Conroy, Rachael Miller, J. Alterman, M. Hassler +13 more
2022Nature Communications38 citations Open access
Semantic Scholar
An RNAi therapeutic targeting hepatic DGAT2 in a genetically obese mouse model of nonalcoholic steatohepatitis
Batuhan Yenilmez, Nicole Wetoska, Mark Kelly, Dimas Echeverria +14 more
2021Molecular Therapy38 citations Open access
Semantic Scholar
Silencing Apoe with divalent‐siRNAs improves amyloid burden and activates immune response pathways in Alzheimer's disease
Chantal M. Ferguson, Samuel Hildebrand, B. Godinho, J. Buchwald +11 more
2024Alzheimer's & Dementia34 citations Open access
Semantic Scholar
Gene therapy with AR isoform 2 rescues spinal and bulbar muscular atrophy phenotype by modulating AR transcriptional activity
W. F. Lim, M. Forouhan, T. Roberts, J. Dabney +20 more
2021Science Advances34 citations Open access
Semantic Scholar
Intratracheally administered LNA gapmer antisense oligonucleotides induce robust gene silencing in mouse lung fibroblasts
Min-Kyu Shin, Io Long Chan, Yu-Ming Cao, A. Gruntman +18 more
2022Nucleic Acids Research32 citations Open access
Semantic Scholar
A programmable dual-targeting siRNA scaffold supports potent two-gene modulation in the central nervous system
Jillian Belgrad, Qi Tang, Sam Hildebrand, Ashley Summers +12 more
2024Nucleic Acids Research26 citations Open access
Semantic Scholar
RNAi-based modulation of IFN-γ signaling in skin
Q. Tang, Jacquelyn Sousa, Dimas Echeverria, Xueli Fan +11 more
2022Molecular Therapy26 citations Open access
Semantic Scholar
Advancements in APOE and dementia research: Highlights from the 2023 AAIC Advancements: APOE conference
Courtney M. Kloske, M. Belloy, Elizabeth E. Blue, Greg R Bowman +56 more
2024Alzheimer's & Dementia25 citations Open access
Semantic Scholar
PK-modifying anchors significantly alter clearance kinetics, tissue distribution, and efficacy of therapeutics siRNAs
B. Godinho, Emily G. Knox, Samuel Hildebrand, James W. Gilbert +10 more
2022Molecular Therapy: Nucleic Acids24 citations Open access
Semantic Scholar
Asymmetric trichotomous partitioning overcomes dataset limitations in building machine learning models for predicting siRNA efficacy
Kathryn R. Monopoli, Dmitry Korkin, A. Khvorova
2023Molecular Therapy: Nucleic Acids23 citations Open access
Semantic Scholar
Systematic analysis of siRNA and mRNA features impacting fully chemically modified siRNA efficacy
Sarah M Davis, Sam Hildebrand, H. MacMillan, Kathryn R. Monopoli +21 more
2025Nucleic Acids Research21 citations Open access
Semantic Scholar
Preventing acute neurotoxicity of CNS therapeutic oligonucleotides with the addition of Ca2+ and Mg2+ in the formulation
Rachael Miller, Joseph D. Paquette, Alexandra Barker, E. Sapp +11 more
2024bioRxiv20 citations Open access
Semantic Scholar
Multispecies-targeting siRNAs for the modulation of JAK1 in the skin
Qi Tang, Katherine Y. Gross, H. Fakih, Samuel O. Jackson +8 more
2024Molecular Therapy: Nucleic Acids20 citations Open access
Semantic Scholar
Mutant huntingtin messenger RNA forms neuronal nuclear clusters in rodent and human brains
Socheata Ly, Marie-Cécile Didiot, Chantal M. Ferguson, Andrew H. Coles +7 more
2022Brain Communications20 citations Open access
Semantic Scholar
Delivering siRNA Compounds During HOPE to Modulate Organ Function: A Proof-of-concept Study in a Rat Liver Transplant Model
E. Bonaccorsi-Riani, Andrew R. Gillooly, S. Iesari, Isabel M. A. Brüggenwirth +12 more
2022Transplantation19 citations Open access
Semantic Scholar
Dendritic amphiphilic siRNA: Selective albumin binding, in vivo efficacy, and low toxicity
H. Fakih, Qi Tang, Ashley Summers, Min-Kyu Shin +8 more
2023Molecular Therapy: Nucleic Acids18 citations Open access
Semantic Scholar
Self-delivering, chemically modified CRISPR RNAs for AAV co-delivery and genome editing in vivo
Han Zhang, Karen Kelly, Jonathan Lee, Dimas Echeverria +14 more
2023Nucleic Acids Research18 citations Open access
Semantic Scholar
Divalent siRNA for prion disease
Juliana E. Gentile, Taylor L. Corridon, Fiona E. Serack, Dimas Echeverria +24 more
2026bioRxiv17 citations Open access
Semantic Scholar
Lactate transporter MCT1 in hepatic stellate cells promotes fibrotic collagen expression in nonalcoholic steatohepatitis
Kyounghee Min, Batuhan Yenilmez, Mark Kelly, Dimas Echeverria +13 more
2024eLife16 citations Open access
Semantic Scholar
Quantifying the activity profile of ASO and siRNA conjugates in glioblastoma xenograft tumors in vivo
Samantha L. Sarli, H. Fakih, Karen Kelly, G. Devi +9 more
2024Nucleic Acids Research16 citations Open access
Semantic Scholar
Di-valent siRNA-mediated silencing of MSH3 blocks somatic repeat expansion in mouse models of Huntington’s disease
Daniel O’Reilly, Jillian Belgrad, Chantal M. Ferguson, Ashley Summers +17 more
2022bioRxiv16 citations Open access
Semantic Scholar
Kinetics of HTT lowering in brain of YAC 128 mice following single and repetitive intranasal dosing of siRNA packaged in chitosan-based nanoparticle
V. Sava, O. Fihurka, A. Khvorova, J. Sanchez-Ramos
2021Journal of Drug Delivery Science and Technology15 citations Open access
Semantic Scholar
Single intravitreal administration of a tetravalent siRNA exhibits robust and efficient gene silencing in mouse and pig photoreceptors
Shun-Yun Cheng, Jillian Caiazzi, Annabelle Biscans, J. Alterman +8 more
2023Molecular Therapy: Nucleic Acids14 citations Open access
Semantic Scholar
Challenges of Assessing Exon 53 Skipping of the Human DMD Transcript with Locked Nucleic Acid-Modified Antisense Oligonucleotides in a Mouse Model for Duchenne Muscular Dystrophy
S. Engelbeen, Daniel O'Reilly, Davy Van De Vijver, I. Verhaart +6 more
2023Nucleic Acid Therapeutics13 citations Open access
Semantic Scholar
Divalent siRNA for prion disease
Juliana E. Gentile, Taylor L. Corridon, Fiona E. Serack, Dimas Echeverria +24 more
2026Nucleic Acids Research12 citations Open access
Semantic Scholar
Preventing acute neurotoxicity of CNS therapeutic oligonucleotides with the addition of Ca2+ and Mg2+ in the formulation
Rachael Miller, Joseph D. Paquette, Alexandra Barker, E. Sapp +11 more
2024Molecular Therapy: Nucleic Acids12 citations Open access
Semantic Scholar
siRNAs-A New Class of Medicines.
A. Khvorova
2023Journal of the American Medical Association (JAMA)12 citations
Semantic Scholar
A combinatorial approach for achieving CNS-selective RNAi
Chantal M. Ferguson, B. Godinho, Dimas Echeverria, M. Hassler +9 more
2024Nucleic Acids Research11 citations Open access
Semantic Scholar
Lowering the HTT1a transcript as an effective therapy for Huntington’s disease
A. Papadopoulou, J. Alterman, Christian Landles, Edward J Smith +17 more
2025bioRxiv10 citations Open access
Semantic Scholar
Suppression of Huntington’s Disease Somatic Instability by Transcriptional Repression and Direct CAG Repeat Binding
Ella W. Mathews, S. Coffey, Annette Gärtner, Jillian Belgrad +22 more
2024bioRxiv10 citations Open access
Semantic Scholar
mRNA-specific readthrough of nonsense codons by antisense oligonucleotides (R-ASOs)
Denis Susorov, Dimas Echeverria, A. Khvorova, A. Korostelev
2024Nucleic Acids Research9 citations Open access
Semantic Scholar
Extended Nucleic Acid (exNA): A Novel, Biologically Compatible Backbone that Significantly Enhances Oligonucleotide Efficacy in vivo.
V. Hariharan, Jillian Caiazzi, Rachael Miller, Chantal M. Ferguson +19 more
2023bioRxiv9 citations Open access
Semantic Scholar
RNAi-mediated silencing of SOD1 profoundly extends survival and functional outcomes in ALS mice
Alexandra Weiss, James W. Gilbert, Iris Valeria Rivera Flores, Jillian Belgrad +20 more
2025Molecular Therapy8 citations Open access
Semantic Scholar
More than 185 CAG repeats: a point of no return in Huntington's disease biology.
Jillian Belgrad, A. Khvorova
2024Brain : a journal of neurology8 citations Open access
Semantic Scholar
RNAi-mediated silencing of SOD1 profoundly extends survival and functional outcomes in ALS mice
Alexandra Weiss, James W. Gilbert, Iris Valeria Rivera Flores, Jillian Belgrad +19 more
2024bioRxiv8 citations Open access
Semantic Scholar
Phosphatidylcholine head group chemistry alters the extrahepatic accumulation of lipid-conjugated siRNA
V. Hariharan, Takahiro I Nakamura, Minwook Shin, Qi Tang +5 more
2024Molecular Therapy: Nucleic Acids8 citations Open access
Semantic Scholar
Modulation of DNA transcription: The future of ASO therapeutics?
A. Khvorova
2022Cell8 citations Open access
Semantic Scholar
Identification of selective and non-selective C9ORF72 targeting in vivo active siRNAs
James W. Gilbert, Zachary C. Kennedy, B. Godinho, Ashley Summers +11 more
2024Molecular Therapy: Nucleic Acids7 citations Open access
Semantic Scholar
Albumin-binding dendritic siRNA improves delivery and efficacy to solid tumors in a melanoma model
H. Fakih, Qi Tang, Ashley Summers, Katherine Y. Gross +6 more
2025Molecular Therapy: Nucleic Acids6 citations Open access
Semantic Scholar

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