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Anastasia Khvorova

university of massachusetts chan medical school

58H-index
209Paper Count
3.1WCitation Count
Published Papers 115
Publication Date
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Single-dose administration of therapeutic divalent siRNA targeting MECP2 prevents lethality in an MECP2 duplication mouse model
errNature Communications
IF15.7
err2026-07-21
err0
errOAAI
errVignesh N. Hariharan; Ashley Summers; Amy E. Clipperton-Allen; Jillian Caiazzi; Samuel R. Hildebrand; Daniel O’ Reilly; Qi Tang; Zachary Kennedy; Dimas Echeverria; Nicholas McHugh; David Cooper; Jacquelyn Sousa; Chantal Ferguson; Hassan H. Fakih; Laurent Bogdanik; Monica Coenraads; Anastasia Khvorova
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errSave
Intradermal delivery of lipophilic siRNAs enables prolonged skin retention and sustained gene silencing in a porcine model
errNature Communications
IF15.7
err2026-02-03
err0
errOAAI
errHassan H. Fakih; Mohammad Zain UI Abideen; Mohamad Omar Rachid; Katherine Y. Gross; Thomas J. R. Ormsby; Vella M. Ross; Rosemary Gagnon; Christopher Dahlke; Raymond C. Furgal; Clemens Lochmann; Ashley Summers; Hanadi F. Sleiman; Sylvia M. Fürst; Shanté N. Jackson; Juliana O. Gordilho; Claire Bouix-Peter; Thibaud Portal; Qi Tang; John E. Harris; Anastasia Khvorova; Carine Blanchard; Julia F. Alterman
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errSave
Therapeutic delivery of albumin-binding siRNA targeting IRS2 to diverse cell types reduces mammary tumor growth
errMolecular Therapy
IF12
err2026-01-29
err0
errOAAI
errClaire E. Tocheny; Julianna E. Buchwald; Christopher D. Dahlke; Hassan H. Fakih; Jennifer S. Morgan; Ashley Summers; Christi A. Silva; Samuel O. Jackson; Ji-Sun Lee; Michael-Anthony Card; Dimas Echeverria; Cornelia Peterson; Arthur M. Mercurio; Anastasia Khvorova; Leslie M. Shaw
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errSave
Suppression of Huntington’s Disease Somatic Instability by Transcriptional Repression and Direct CAG Repeat Binding
errNature Communications
IF15.7
err2025-11-14
err0
errOAAI
errElla W. Mathews; Sydney R. Coffey; Annette Gärtner; Jillian Belgrad; Robert M. Bragg; Daniel O’Reilly; Jeffrey P. Cantle; Cassandra McHugh; Ashley Summers; Joachim Fentz; Tom Schwagarus; Antje Cornelius; Ioannis Lingos; Zoe Burch; Marina Kovalenko; Marissa A. Andrew; C. Frank Bennett; Holly B. Kordasiewicz; Deanna M. Marchionini; Hilary Wilkinson; Thomas F. Vogt; Paolo Beuzer; Ricardo M. Pinto; Anastasia Khvorova; David Howland; Vanessa C. Wheeler; Jeffrey B. Carroll
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errSave
Mutant huntingtin exon 1 protein detected in mouse brain with neoepitope antibody: effects of CAG repeat expansion, MutS Homolog 3 silencing and aggregation
errBrain Communications
IF4.5
err2025-09-01
err0
errOAAI
errEllen Sapp; Adel Boudi; Andrew Iwanowicz; Jillian Belgrad; Rachael Miller; Riannon Robertson; Daniel O’Reilly; Ken Yamada; Yunping Deng; Marion Joni; Xueyi Li; Kimberly Kegel-Gleason; Anastasia Khvorova; Anton Reiner; Neil Aronin; Marian DiFiglia
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errSave
Systematic analysis of siRNA and mRNA features impacting fully chemically modified siRNA efficacy
errNUCLEIC ACIDS RESEARCH
IF13.1
err2025-07-08
err4
errOAAI
errDavis, Sarah M.; Hildebrand, Samuel; MacMillan, Hannah J.; Monopoli, Kathryn R.; Buchwald, Julianna; Sousa, Jacquelyn; Cooper, David; Ly, Socheata; Echeverria, Dimas; McHugh, Nicholas; Ferguson, Chantal; Coles, Andrew; Hariharan, Vignesh N.; O'Reilly, Daniel; Tang, Qi; Furgal, Raymond; Yamada, Ken; Alterman, Julia F.; Gilbert, James W.; Knox, Emily; Pineda, Yamilett; Weston, Caitlyn N.; Baer, Christina E.; Pai, Athma A.; Khvorova, Anastasia
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errSave
A systemically deliverable lipid-conjugated siRNA targeting DUX4 as an facioscapulohumeral muscular dystrophy therapeutic
errMolecular Therapy Methods & Clinical Development
IF4.7
err2025-06-16
err0
errOAAI
errKatelyn Daman; Jing Yan; Annabelle Biscans; Dimas Echeverria; Taisia Shmushkovich; Alexey Wolfson; Julia F. Alterman; Anastasia Khvorova; Charles P. Emerson
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errSave
Albumin-binding dendritic siRNA improves delivery and efficacy to solid tumors in a melanoma model
errMolecular Therapy Nucleic Acids
IF6.1
err2025-05-24
err0
errOAAI
errHassan H. Fakih; Qi Tang; Ashley Summers; Katherine Y. Gross; Mohamad Omar Rachid; Ken Okamura; Nuria Martinez; Hanadi F. Sleiman; John E. Harris; Anastasia Khvorova
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errSave
RNAi-mediated silencing of SOD1 profoundly extends survival and functional outcomes in ALS mice
errMolecular Therapy
IF12
err2025-05-09
err0
errOAAI
errAlexandra Weiss; James W. Gilbert; Iris Valeria Rivera Flores; Jillian Belgrad; Chantal Ferguson; Elif O. Dogan; Nicholas Wightman; Kit Mocarski; Dimas Echeverria; Ashley L. Harkins; Ashley Summers; Brianna Bramato; Nicholas McHugh; Raymond Furgal; Nozomi Yamada; David Cooper; Kathryn Monopoli; Bruno M.D.C. Godinho; Matthew R. Hassler; Ken Yamada; Paul Greer; Nils Henninger; Robert H. Brown; Anastasia Khvorova
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errSave
Preventing acute neurotoxicity of CNS therapeutic oligonucleotides with the addition of Ca2+and Mg2+in the formulation
errMOLECULAR THERAPY NUCLEIC ACIDS
IF6.1
err2024-12-01
err1
errOAAI
errMiller, Rachael; Paquette, Joseph; Barker, Alexandra; Sapp, Ellen; McHugh, Nicholas; Bramato, Brianna; Yamada, Nozomi; Alterman, Julia; Echeveria, Dimas; Yamada, Ken; Watts, Jonathan; Anaclet, Christelle; DiFiglia, Marian; Khvorova, Anastasia; Aronin, Neil
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errSave
Near Sequence Homology Does Not Guarantee siRNA Cross-Species Efficacy
errNUCLEIC ACID THERAPEUTICS
IF4.7
err2024-10-01
err1
PREAI
errFlores, Iris Valeria Rivera; Monopoli, Kathryn; Jackson, Samuel; Echeverria, Dimas; OReilly, Daniel; Brown, Robert H.; Khvorova, Anastasia
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errSave
Identification of selective and non-selective C9ORF72 targeting in vivo active siRNAs
errMOLECULAR THERAPY NUCLEIC ACIDS
IF6.1
err2024-09-01
err1
errOAAI
errGilbert, James W.; Kennedy, Zachary; Godinho, Bruno M. D. C.; Summers, Ashley; Weiss, Alexandra; Echeverria, Dimas; Bramato, Brianna; McHugh, Nicholas; Cooper, David; Yamada, Ken; Hassler, Matthew; Tran, Helene; Gao, Fen Biao; Brown Jr, Robert H.; Khvorova, Anastasia
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errSave
mRNA Nuclear Clustering Leads to a Difference in Mutant Huntingtin mRNA and Protein Silencing by siRNAs In Vivo
errNUCLEIC ACID THERAPEUTICS
IF4.7
err2024-08-01
err1
PREAI
errAllen, Sarah; O'Reilly, Daniel; Miller, Rachael; Sapp, Ellen; Summers, Ashley; Paquette, Joseph; Moreno, Dimas Echeverria; Bramato, Brianna; McHugh, Nicholas; Yamada, Ken; Aronin, Neil; DiFiglia, Marian; Khvorova, Anastasia
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errSave
Enhancing siRNA efficacy in vivo with extended nucleic acid backbones
errNATURE BIOTECHNOLOGY
IF41.7
err2024-08-01
err1
PREAI
errYamada, Ken; Hariharan, Vignesh N.; Caiazzi, Jillian; Miller, Rachael; Ferguson, Chantal M.; Sapp, Ellen; Fakih, Hassan H.; Tang, Qi; Yamada, Nozomi; Furgal, Raymond C.; Paquette, Joseph D.; Biscans, Annabelle; Bramato, Brianna M.; Mchugh, Nicholas; Summers, Ashley; Lochmann, Clemens; Godinho, Bruno M. D. C.; Hildebrand, Samuel; Jackson, Samuel O.; Echeverria, Dimas; Hassler, Matthew R.; Alterman, Julia F.; Difiglia, Marian; Aronin, Neil; Khvorova, Anastasia
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errSave
mRNA-specific readthrough of nonsense codons by antisense oligonucleotides (R-ASOs)
errNUCLEIC ACIDS RESEARCH
IF13.1
err2024-07-16
err1
errOAAI
errSusorov, Denis; Echeverria, Dimas; Khvorova, Anastasia; Korostelev, Andrei A.
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errSave
A programmable dual-targeting siRNA scaffold supports potent two-gene modulation in the central nervous system
errNUCLEIC ACIDS RESEARCH
IF13.1
err2024-05-10
err1
errOAAI
errBelgrad, Jillian; Tang, Qi; Hildebrand, Sam; Summers, Ashley; Sapp, Ellen; Echeverria, Dimas; O'Reilly, Dan; Luu, Eric; Bramato, Brianna; Allen, Sarah; Cooper, David; Alterman, Julia; Yamada, Ken; Aronin, Neil; Difiglia, Marian; Khvorova, Anastasia
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errSave
Quantifying the activity profile of ASO and siRNA conjugates in glioblastoma xenograft tumors in vivo
errNUCLEIC ACIDS RESEARCH
IF13.1
err2024-04-13
err2
errOAAI
errSarli, Samantha L.; Fakih, Hassan H.; Kelly, Karen; Devi, Gitali; Rembetsy-Brown, Julia M.; McEachern, Holly R.; Ferguson, Chantal M.; Echeverria, Dimas; Lee, Jonathan; Sousa, Jacquelyn; Sleiman, Hanadi F.; Khvorova, Anastasia; Watts, Jonathan K.
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errSave
More than 185 CAG repeats: a point of no return in Huntington's disease biology
errBRAIN
IF11.7
err2024-04-08
err2
errOAAI
errBelgrad, Jillian; Khvorova, Anastasia
errShare
errSave
RNAi-based drug design: considerations and future directions
errNATURE REVIEWS DRUG DISCOVERY
IF101.8
err2024-04-03
err24
errOAAI
errTang, Qi; Khvorova, Anastasia
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errSave
Lactate transporter MCT1 in hepatic stellate cells promotes fibrotic collagen expression in nonalcoholic steatohepatitis
errELIFE
IF0
err2024-04-02
err2
errOAAI
errMin, Kyounghee; Yenilmez, Batuhan; Kelly, Mark; Echeverria, Dimas; Elleby, Michael; Lifshitz, Lawrence M.; Raymond, Naideline; Tsagkaraki, Emmanouela; Harney, Shauna M.; DiMarzio, Chloe; Wang, Hui; McHugh, Nicholas; Bramato, Brianna; Morrison, Brett; Rothstein, Jeffery D.; Khvorova, Anastasia; Czech, Michael P.
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Research Directions

No research directions
Co-authors
Cooperation Journals
J
Jennifer A. Doudna
H-index: 149 · Papers: 672
R
Raghu Kalluri
H-index: 142 · Papers: 573
C
Carolyn R. Bertozzi
H-index: 140 · Papers: 942
K
Katherine A. Fitzgerald
H-index: 136 · Papers: 607
J
Joan S. Brugge
H-index: 126 · Papers: 597
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