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Anastasia Khvorova PhD

Endowed TitleRemondi Family Chair in Biomedical Research
InstitutionUMass Chan Medical School
DepartmentRNA Therapeutics Institute
AddressUMass Chan Medical School
368 Plantation Street AS4.1049
Worcester MA 01605
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    Other Positions
    InstitutionT.H. Chan School of Medicine
    DepartmentNeuroNexus Institute

    InstitutionT.H. Chan School of Medicine
    DepartmentProgram in Molecular Medicine

    InstitutionT.H. Chan School of Medicine
    DepartmentRNA Therapeutics Institute

    InstitutionMorningside Graduate School of Biomedical Sciences
    DepartmentBiochemistry and Molecular Biotechnology

    InstitutionMorningside Graduate School of Biomedical Sciences
    DepartmentInterdisciplinary Graduate Program

    InstitutionMorningside Graduate School of Biomedical Sciences
    DepartmentMD/PhD Program

    InstitutionMorningside Graduate School of Biomedical Sciences

    InstitutionMorningside Graduate School of Biomedical Sciences
    DepartmentPostbaccalaureate Research Education Program

    InstitutionMorningside Graduate School of Biomedical Sciences
    DepartmentTranslational Science

    Collapse Biography 
    Collapse education and training
    Moscow State University, Moscow, , Russian FederationBSBiology
    Moscow State University, Moscow, , Russian FederationMSMolecular Biology
    Russian Academy of Sciences, Moscow, , Russian FederationPHDBiochemistry

    Collapse Overview 
    Collapse Rotation Projects
    Our laboratory uses a combination of nucleic acid and oligonucleotide chemistry, biochemistry, cell biology, and pharmacology and develops and evaluate novel technologies for development of oligonucleotide therapeutics. A rotation project is available to develop and identify chemically modified self-delivering RNAi compounds for treatment of Huntington and other diseases.
    Collapse Post Docs

    Excellent training opportunity for a person considering industrial carrier in drug development
    Oligonucleotides represent a new class of drugs, which have the promise to become a major class of future therapeutics. There are several classes of oligonucleotides which can efficiently silence genes in various tissues with acceptable level of toxicity. There are also new chemistries being developed to further improve oligonucleotide tissue distribution and cellular uptake.
    The successful candidate will be a key part of an intra-disciplinary team working with biologists and chemists to develop novel types of therapeutics for treatment of neurological disease, liver disease and orphan indications. The work will include compounds identification, screening, characterization, in vivo efficacy and PK/PD studies, novel assays development etc. In addition exposure and training in nucleic acid pre-clinical development, project management etc will be provided.

    QUALIFICATIONS: Applicants must have (or expect to obtain shortly) a Ph.D. in Chemistry, Organic Chemistry Biochemistry, Cell Biology, Molecular Biology or a related field , be bright, ambitious,  independent and collaborative and ideally,  have research experience in nucleic acid molecular cell biology or pharmacology.

    Develop and characterize novel RNA chemistries to promote efficient oligonucleotide internalization and tissue distribution.

    Oligonucleotides represent a new class of drugs, which have the promise to become a major class of future therapeutics. There are several chemistries and formulations developed by us and others that effectively support oligo internalization and favorable PK/PD. In most cases in vivo efficacy is limited by (1) poor PK (2) inefficient tissue distribution (3) compound entrapment in biologically inactive intracellular compartments.
    The objective of future studies will be development of novel chemistries, which promote receptor mediated cellular internalization and/ or endosomal escape. The successful candidate will be responsible for the design and synthesis of novel RNA precursors, optimization of RNA synthesis and conjugation protocols and interface with the biology part of the lab. In addition, exposure to pre-clinical development programs and oligonucleotide manufacturing will be available.

     QUALIFICATIONS: Applicants must have (or expect to obtain shortly) a Ph.D. in Chemistry, Organic Chemistry Biochemistry, Cell Biology, Molecular Biology or a related field , be bright, ambitious, independent and collaborative and have previous experience in nucleic acid / oligonucleotide chemistry.

    Discover, characterize and adapt mechanisms of small RNA extracellular and intracellular trafficking for development of novel classes of oligonucleotide drugs.

     A serious limitation in realizing the potential of oligonucleotide based therapies has been the exceedingly inefficient transit of oligonucleotides from outside cells to the intracellular compartments where the biologically relevant activity happens.  On the other hand, there are multiple lines of evidence supporting the notion that nature has implemented evolutionarily conserved mechanisms and pathways for efficiently trafficking small RNAs across cellular boundaries. The objective of future studies will  be the systematic evaluation of mechanisms of cellular uptake of different classes of oligonucleotides and their comparison to mechanisms of  native RNA trafficking using a combination of experimental approaches, including advanced RNA chemistry and high temporal and spatial resolution microscopy in living cells, Mass spectrometry, cell biology and others.  The successful candidate will work closely with Victor Ambros and Silvia Corvera labs and the UMASS imaging group and receive training in RNA chemistry, biochemistry, cell biology and pre-clinical development.

    QUALIFICATIONS:Applicants must have (or expect to obtain shortly) a Ph.D. in Chemistry, Organic Chemistry Biochemistry, Cell Biology, Molecular Biology or a related field , be bright, ambitious,  independent and collaborative and ideally,  have research experience in nucleic acid molecular and cell biology

    Collapse Bibliographic 
    Collapse selected publications
    Publications listed below are automatically derived from MEDLINE/PubMed and other sources, which might result in incorrect or missing publications. Faculty can login to make corrections and additions.
    Newest   |   Oldest   |   Most Cited   |   Most Discussed   |   Timeline   |   Field Summary   |   Plain Text
    PMC Citations indicate the number of times the publication was cited by articles in PubMed Central, and the Altmetric score represents citations in news articles and social media. (Note that publications are often cited in additional ways that are not shown here.) Fields are based on how the National Library of Medicine (NLM) classifies the publication's journal and might not represent the specific topic of the publication. Translation tags are based on the publication type and the MeSH terms NLM assigns to the publication. Some publications (especially newer ones and publications not in PubMed) might not yet be assigned Field or Translation tags.) Click a Field or Translation tag to filter the publications.
    1. Sarli SL, Fakih HH, Kelly K, Devi G, Rembetsy-Brown JM, McEachern HR, Ferguson CM, Echeverria D, Lee J, Sousa J, Sleiman HF, Khvorova A, Watts JK. Quantifying the activity profile of ASO and siRNA conjugates in glioblastoma xenograft tumors in vivo. Nucleic Acids Res. 2024 Apr 13. PMID: 38613388.
    2. Belgrad J, Khvorova A. More than 185 CAG repeats: a point of no return in Huntington's disease biology. Brain. 2024 Apr 08. PMID: 38588052.
    3. Tang Q, Khvorova A. RNAi-based drug design: considerations and future directions. Nat Rev Drug Discov. 2024 Apr 03. PMID: 38570694.
    4. Min K, Yenilmez B, Kelly M, Echeverria D, Elleby M, Lifshitz LM, Raymond N, Tsagkaraki E, Harney SM, DiMarzio C, Wang H, McHugh N, Bramato B, Morrison B, Rothstein JD, Khvorova A, Czech MP. Lactate transporter MCT1 in hepatic stellate cells promotes fibrotic collagen expression in nonalcoholic steatohepatitis. Elife. 2024 Apr 02; 12. PMID: 38564479.
    5. Belgrad J, Fakih HH, Khvorova A. Nucleic Acid Therapeutics: Successes, Milestones, and Upcoming Innovation. Nucleic Acid Ther. 2024 Apr; 34(2):52-72. PMID: 38507678.
    6. Ferguson CM, Hildebrand S, Godinho BMDC, Buchwald J, Echeverria D, Coles A, Grigorenko A, Vangjeli L, Sousa J, McHugh N, Hassler M, Santarelli F, Heneka MT, Rogaev E, Khvorova A. Silencing Apoe with divalent-siRNAs improves amyloid burden and activates immune response pathways in Alzheimer's disease. Alzheimers Dement. 2024 Feb 20. PMID: 38375983.
    7. Ferguson CM, Godinho BMDC, Echeverria D, Hassler M, Vangjeli L, Sousa J, McHugh N, Alterman J, Hariharan V, Krishnamurthy PM, Watts J, Rogaev E, Khvorova A. A combinatorial approach for achieving CNS-selective RNAi. Nucleic Acids Res. 2024 Feb 13. PMID: 38348876.
    8. Zhang H, Kelly K, Lee J, Echeverria D, Cooper D, Panwala R, Amrani N, Chen Z, Gaston N, Wagh A, Newby GA, Xie J, Liu DR, Gao G, Wolfe SA, Khvorova A, Watts JK, Sontheimer EJ. Self-delivering, chemically modified CRISPR RNAs for AAV co-delivery and genome editing in vivo. Nucleic Acids Res. 2024 Jan 25; 52(2):977-997. PMID: 38033325.
      Citations:    Fields:    Translation:Animals
    9. Tang Q, Gross KY, Fakih HH, Jackson SO, Zain U I Abideen M, Monopoli KR, Blanchard C, Bouix-Peter C, Portal T, Harris JE, Khvorova A, Alterman JF. Multispecies-targeting siRNAs for the modulation of JAK1 in the skin. Mol Ther Nucleic Acids. 2024 Mar 12; 35(1):102117. PMID: 38304729.
    10. Belgrad J, Tang Q, Hildebrand S, Summers A, Sapp E, Echeverria D, O'Reilly D, Luu E, Bramato B, Allen S, Cooper D, Alterman J, Yamada K, Aronin N, DiFiglia M, Khvorova A. A programmable dual-targeting di-valent siRNA scaffold supports potent multi-gene modulation in the central nervous system. bioRxiv. 2023 Dec 19. PMID: 38187561.
    11. Cheng SY, Caiazzi J, Biscans A, Alterman JF, Echeverria D, McHugh N, Hassler M, Jolly S, Giguere D, Cipi J, Khvorova A, Punzo C. Single intravitreal administration of a tetravalent siRNA exhibits robust and efficient gene silencing in mouse and pig photoreceptors. Mol Ther Nucleic Acids. 2024 Mar 12; 35(1):102088. PMID: 38192611.
    12. Engelbeen S, O'Reilly D, Van De Vijver D, Verhaart I, van Putten M, Hariharan V, Hassler M, Khvorova A, Damha MJ, Aartsma-Rus A. 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. Nucleic Acid Ther. 2023 Dec; 33(6):348-360. PMID: 38010230.
      Citations:    Fields:    Translation:HumansAnimals
    13. Fakih HH, Tang Q, Summers A, Shin M, Buchwald JE, Gagnon R, Hariharan VN, Echeverria D, Cooper DA, Watts JK, Khvorova A, Sleiman HF. Dendritic amphiphilic siRNA: Selective albumin binding, in?vivo efficacy, and low toxicity. Mol Ther Nucleic Acids. 2023 Dec 12; 34:102080. PMID: 38089931.
    14. Tang Q, Fakih HH, Zain Ui Abideen M, Hildebrand SR, Afshari K, Gross KY, Sousa J, Maebius AS, Bartholdy C, S?gaard PP, Jackerott M, Hariharan V, Summers A, Fan X, Okamura K, Monopoli KR, Cooper DA, Echeverria D, Bramato B, McHugh N, Furgal RC, Dresser K, Winter SJ, Biscans A, Chuprin J, Haddadi NS, Sherman S, Yildiz-Altay ?, Rashighi M, Richmond JM, Bouix-Peter C, Blanchard C, Clauss A, Alterman JF, Khvorova A, Harris JE. Rational design of a JAK1-selective siRNA inhibitor for the modulation of autoimmunity in the skin. Nat Commun. 2023 11 04; 14(1):7099. PMID: 37925520.
      Citations: 2     Fields:    Translation:HumansAnimalsCells
    15. Min K, Yenilmez B, Kelly M, Echeverria D, Elleby M, Lifshitz LM, Raymond N, Tsagkaraki E, Harney SM, DiMarzio C, Wang H, McHugh N, Bramato B, Morrision B, Rothstein JD, Khvorova A, Czech MP. Lactate transporter MCT1 in hepatic stellate cells promotes fibrotic collagen expression in nonalcoholic steatohepatitis. bioRxiv. 2023 Oct 19. PMID: 37205462.
    16. Benatti HR, Prestigiacomo RD, Taghian T, Miller R, King R, Gounis MJ, Celik U, Bertrand S, Tuominen S, Bierfeldt L, Parsley E, Gallagher J, Hall EF, McElroy AW, Sena-Esteves M, Khvorova A, Aronin N, Gray-Edwards HL. Awake intracerebroventricular delivery and safety assessment of oligonucleotides in a large animal model. Mol Ther Methods Clin Dev. 2023 Dec 14; 31:101122. PMID: 37920238.
    17. O'Reilly D, Belgrad J, Ferguson C, Summers A, Sapp E, McHugh C, Mathews E, Boudi A, Buchwald J, Ly S, Moreno D, Furgal R, Luu E, Kennedy Z, Hariharan V, Monopoli K, Yang XW, Carroll J, DiFiglia M, Aronin N, Khvorova A. Di-valent siRNA-mediated silencing of MSH3 blocks somatic repeat expansion in mouse models of Huntington's disease. Mol Ther. 2023 Nov 01; 31(11):3355-3356. PMID: 37751745.
      Citations: 1     Fields:    
    18. Cheng SY, Caiazzi J, Biscans A, Alterman JF, Echeverria D, McHugh N, Hassler M, Jolly S, Giguere D, Cipi J, Khvorova A, Punzo C. Single intravitreal administration of a tetravalent siRNA exhibits robust and efficient gene silencing in rodent and swine photoreceptors. bioRxiv. 2023 Sep 22. PMID: 37790464.
    19. Yenilmez B, Harney S, DiMarzio C, Kelly M, Min K, Echeverria D, Bramato BM, Jackson SO, Reddig K, Kim JK, Khvorova A, Czech MP. Dual targeting of hepatocyte DGAT2 and stellate cell FASN alleviates nonalcoholic steatohepatitis in mice. bioRxiv. 2023 Jul 05. PMID: 37461560.
    20. Khvorova A. siRNAs-A New Class of Medicines. JAMA. 2023 06 27; 329(24):2185-2186. PMID: 37306980.
      Citations: 3     Fields:    
    21. Monopoli KR, Korkin D, Khvorova A. Asymmetric trichotomous partitioning overcomes dataset limitations in building machine learning models for predicting siRNA efficacy. Mol Ther Nucleic Acids. 2023 Sep 12; 33:93-109. PMID: 37456778.
    22. Hariharan VN, Caiazzi J, Miller R, Ferguson C, Sapp E, Fakih H, Tang Q, Yamada N, Furgal R, Paquette J, Bramato B, McHugh N, Summers A, Lochmann C, Godinho B, Hildebrand S, Echeverria D, Hassler M, Alterman J, DiFiglia M, Aronin N, Khvorova A, Yamada K. Extended Nucleic Acid (exNA): A Novel, Biologically Compatible Backbone that Significantly Enhances Oligonucleotide Efficacy in vivo. Res Sq. 2023 Jun 01. PMID: 37398145.
    23. Yamada K, Hariharan VN, Caiazzi J, Miller R, Furguson C, Sapp E, Fakih H, Tan Q, Yamada N, Furgal RC, Paquette J, Bramato B, McHugh N, Summers A, Lochmann C, Godinho BMDC, Hildebrand S, Echeverria D, Hassler MR, Alterman JF, DiFiglia M, Aronin N, Khvorova A. Extended Nucleic Acid (exNA): A Novel, Biologically Compatible Backbone that Significantly Enhances Oligonucleotide Efficacy in vivo. bioRxiv. 2023 May 26. PMID: 37292886.
    24. O'Reilly D, Belgrad J, Ferguson C, Summers A, Sapp E, McHugh C, Mathews E, Boudi A, Buchwald J, Ly S, Moreno D, Furgal R, Luu E, Kennedy Z, Hariharan V, Monopoli K, Yang XW, Carroll J, DiFiglia M, Aronin N, Khvorova A. Di-valent siRNA-mediated silencing of MSH3 blocks somatic repeat expansion in mouse models of Huntington's disease. Mol Ther. 2023 06 07; 31(6):1661-1674. PMID: 37177784.
      Citations: 4     Fields:    Translation:AnimalsCells
    25. Hildebrand SR, Hedger AK, Yamada K, Watts JK, Khvorova A. Formamide significantly enhances the efficiency of chemical adenylation of RNA sequencing ligation adaptors. RNA. 2023 07; 29(7):1077-1083. PMID: 37059467.
      Citations:    Fields:    
    26. Zhang H, Kelly K, Lee J, Echeverria D, Cooper D, Panwala R, Chen Z, Gaston N, Newby GA, Xie J, Liu DR, Gao G, Wolfe SA, Khvorova A, Watts JK, Sontheimer EJ. Self-delivering CRISPR RNAs for AAV Co-delivery and Genome Editing in vivo. bioRxiv. 2023 Mar 23. PMID: 36993169.
    27. Hariharan VN, Shin M, Chang CW, O'Reilly D, Biscans A, Yamada K, Guo Z, Somasundaran M, Tang Q, Monopoli K, Krishnamurthy PM, Devi G, McHugh N, Cooper DA, Echeverria D, Cruz J, Chan IL, Liu P, Lim SY, McConnell J, Singh SP, Hildebrand S, Sousa J, Davis SM, Kennedy Z, Ferguson C, Godinho BMDC, Thillier Y, Caiazzi J, Ly S, Muhuri M, Kelly K, Humphries F, Cousineau A, Parsi KM, Li Q, Wang Y, Maehr R, Gao G, Korkin D, McDougall WM, Finberg RW, Fitzgerald KA, Wang JP, Watts JK, Khvorova A. Divalent siRNAs are bioavailable in the lung and efficiently block SARS-CoV-2 infection. Proc Natl Acad Sci U S A. 2023 03 14; 120(11):e2219523120. PMID: 36893269.
      Citations: 4     Fields:    Translation:HumansAnimalsCells
    28. Ly S, Didiot MC, Ferguson CM, Coles AH, Miller R, Chase K, Echeverria D, Wang F, Sadri-Vakili G, Aronin N, Khvorova A. Mutant huntingtin messenger RNA forms neuronal nuclear clusters in rodent and human brains. Brain Commun. 2022; 4(6):fcac248. PMID: 36458209.
    29. Conroy F, Miller R, Alterman JF, Hassler MR, Echeverria D, Godinho BMDC, Knox EG, Sapp E, Sousa J, Yamada K, Mahmood F, Boudi A, Kegel-Gleason K, DiFiglia M, Aronin N, Khvorova A, Pfister EL. Chemical engineering of therapeutic siRNAs for allele-specific gene silencing in Huntington's disease models. Nat Commun. 2022 10 03; 13(1):5802. PMID: 36192390.
      Citations: 3     Fields:    Translation:Animals
    30. Shin M, Chan IL, Cao Y, Gruntman AM, Lee J, Sousa J, Rodr?guez TC, Echeverria D, Devi G, Debacker AJ, Moazami MP, Krishnamurthy PM, Rembetsy-Brown JM, Kelly K, Yukselen O, Donnard E, Parsons TJ, Khvorova A, Sontheimer EJ, Maehr R, Garber M, Watts JK. Intratracheally administered LNA gapmer antisense oligonucleotides induce robust gene silencing in mouse lung fibroblasts. Nucleic Acids Res. 2022 08 26; 50(15):8418-8430. PMID: 35920332.
      Citations: 6     Fields:    Translation:AnimalsCells
    31. Biscans A, Ly S, McHugh N, Cooper DA, Khvorova A. Engineered ionizable lipid siRNA conjugates enhance endosomal escape but induce toxicity in vivo. J Control Release. 2022 09; 349:831-843. PMID: 35917865.
      Citations: 2     Fields:    Translation:Animals
    32. Davis SM, Hariharan VN, Lo A, Turanov AA, Echeverria D, Sousa J, McHugh N, Biscans A, Alterman JF, Karumanchi SA, Moore MJ, Khvorova A. Chemical optimization of siRNA for safe and efficient silencing of placental sFLT1. Mol Ther Nucleic Acids. 2022 Sep 13; 29:135-149. PMID: 35847173.
    33. Godinho BMDC, Knox EG, Hildebrand S, Gilbert JW, Echeverria D, Kennedy Z, Haraszti RA, Ferguson CM, Coles AH, Biscans A, Caiazzi J, Alterman JF, Hassler MR, Khvorova A. PK-modifying anchors significantly alter clearance kinetics, tissue distribution, and efficacy of therapeutics siRNAs. Mol Ther Nucleic Acids. 2022 Sep 13; 29:116-132. PMID: 35795486.
    34. Khvorova A. Modulation of DNA transcription: The future of ASO therapeutics? Cell. 2022 06 09; 185(12):2011-2013. PMID: 35688130.
      Citations: 1     Fields:    Translation:Humans
    35. Bonaccorsi-Riani E, Gillooly AR, Iesari S, Br?ggenwirth IMA, Ferguson CM, Komuta M, Xhema D, Daumerie A, Maistriaux L, Leuvenink H, Kupiec-Weglinski J, Porte RJ, Khvorova A, Cave DR, Gianello P, Martins PN. Delivering siRNA Compounds During HOPE to Modulate Organ Function: A Proof-of-concept Study in a Rat Liver Transplant Model. Transplantation. 2022 08 01; 106(8):1565-1576. PMID: 35581683.
      Citations: 2     Fields:    Translation:HumansAnimals
    36. Tang Q, Sousa J, Echeverria D, Fan X, Hsueh YC, Afshari K, MeHugh N, Cooper DA, Vangjeli L, Monopoli K, Okamura K, Biscans A, Clauss A, Harris JE, Khvorova A. RNAi-based modulation of IFN-? signaling in skin. Mol Ther. 2022 08 03; 30(8):2709-2721. PMID: 35477658.
      Citations: 5     Fields:    Translation:Animals
    37. Ferguson CM, Godinho BM, Alterman JF, Coles AH, Hassler M, Echeverria D, Gilbert JW, Knox EG, Caiazzi J, Haraszti RA, King RM, Taghian T, Puri A, Moser RP, Gounis MJ, Aronin N, Gray-Edwards H, Khvorova A. Comparative route of administration studies using therapeutic siRNAs show widespread gene modulation in Dorset sheep. JCI Insight. 2021 12 22; 6(24). PMID: 34935646.
      Citations: 3     Fields:    Translation:Animals
    38. Yamada K, Hildebrand S, Davis SM, Miller R, Conroy F, Sapp E, Caiazzi J, Alterman JF, Roux L, Echeverria D, Hassler MR, Pfister EL, DiFiglia M, Aronin N, Khvorova A. Structurally constrained phosphonate internucleotide linkage impacts oligonucleotide-enzyme interaction, and modulates siRNA activity and allele specificity. Nucleic Acids Res. 2021 12 02; 49(21):12069-12088. PMID: 34850120.
      Citations: 2     Fields:    Translation:Humans
    39. Yenilmez B, Wetoska N, Kelly M, Echeverria D, Min K, Lifshitz L, Alterman JF, Hassler MR, Hildebrand S, DiMarzio C, McHugh N, Vangjeli L, Sousa J, Pan M, Han X, Brehm MA, Khvorova A, Czech MP. An RNAi therapeutic targeting hepatic DGAT2 in a genetically obese mouse model of nonalcoholic steatohepatitis. Mol Ther. 2022 03 02; 30(3):1329-1342. PMID: 34774753.
      Citations: 7     Fields:    Translation:Animals
    40. Lim WF, Forouhan M, Roberts TC, Dabney J, Ellerington R, Speciale AA, Manzano R, Lieto M, Sangha G, Banerjee S, Concei??o M, Cravo L, Biscans A, Roux L, Pourshafie N, Grunseich C, Duguez S, Khvorova A, Pennuto M, Cortes CJ, La Spada AR, Fischbeck KH, Wood MJA, Rinaldi C. Gene therapy with AR isoform 2 rescues spinal and bulbar muscular atrophy phenotype by modulating AR transcriptional activity. Sci Adv. 2021 08; 7(34). PMID: 34417184.
      Citations: 14     Fields:    Translation:Animals
    41. Fukuda K, Okamura K, Riding RL, Fan X, Afshari K, Haddadi NS, McCauley SM, Guney MH, Luban J, Funakoshi T, Yaguchi T, Kawakami Y, Khvorova A, Fitzgerald KA, Harris JE. AIM2 regulates anti-tumor immunity and is a viable therapeutic target for melanoma. J Exp Med. 2021 09 06; 218(9). PMID: 34325468.
      Citations: 24     Fields:    Translation:HumansAnimalsCells
    42. Saha K, Sontheimer EJ, Brooks PJ, Dwinell MR, Gersbach CA, Liu DR, Murray SA, Tsai SQ, Wilson RC, Anderson DG, Asokan A, Banfield JF, Bankiewicz KS, Bao G, Bulte JWM, Bursac N, Campbell JM, Carlson DF, Chaikof EL, Chen ZY, Cheng RH, Clark KJ, Curiel DT, Dahlman JE, Deverman BE, Dickinson ME, Doudna JA, Ekker SC, Emborg ME, Feng G, Freedman BS, Gamm DM, Gao G, Ghiran IC, Glazer PM, Gong S, Heaney JD, Hennebold JD, Hinson JT, Khvorova A, Kiani S, Lagor WR, Lam KS, Leong KW, Levine JE, Lewis JA, Lutz CM, Ly DH, Maragh S, McCray PB, McDevitt TC, Mirochnitchenko O, Morizane R, Murthy N, Prather RS, Ronald JA, Roy S, Roy S, Sabbisetti V, Saltzman WM, Santangelo PJ, Segal DJ, Shimoyama M, Skala MC, Tarantal AF, Tilton JC, Truskey GA, Vandsburger M, Watts JK, Wells KD, Wolfe SA, Xu Q, Xue W, Yi G, Zhou J. The NIH Somatic Cell Genome Editing program. Nature. 2021 04; 592(7853):195-204. PMID: 33828315.
      Citations: 45     Fields:    Translation:HumansAnimalsCells
    43. Biscans A, Caiazzi J, McHugh N, Hariharan V, Muhuri M, Khvorova A. Docosanoic acid conjugation to siRNA enables functional and safe delivery to skeletal and cardiac muscles. Mol Ther. 2021 04 07; 29(4):1382-1394. PMID: 33348054.
      Citations: 19     Fields:    Translation:HumansAnimals
    44. Biscans A, Caiazzi J, Davis S, McHugh N, Sousa J, Khvorova A. The chemical structure and phosphorothioate content of hydrophobically modified siRNAs impact extrahepatic distribution and efficacy. Nucleic Acids Res. 2020 08 20; 48(14):7665-7680. PMID: 32672813.
      Citations: 18     Fields:    Translation:Animals
    45. Ly S, Echeverria D, Sousa J, Khvorova A. Single-Stranded Phosphorothioated Regions Enhance Cellular Uptake of Cholesterol-Conjugated siRNA but Not Silencing Efficacy. Mol Ther Nucleic Acids. 2020 Sep 04; 21:991-1005. PMID: 32818923.
    46. Ferguson CM, Echeverria D, Hassler M, Ly S, Khvorova A. Cell Type Impacts Accessibility of mRNA to Silencing by RNA Interference. Mol Ther Nucleic Acids. 2020 Sep 04; 21:384-393. PMID: 32650236.
    47. Davis SM, Sousa J, Vangjeli L, Hassler MR, Echeverria D, Knox E, Turanov AA, Alterman JF, Khvorova A. 2'-O-Methyl at 20-mer Guide Strand 3' Termini May Negatively Affect Target Silencing Activity of Fully Chemically Modified siRNA. Mol Ther Nucleic Acids. 2020 Sep 04; 21:266-277. PMID: 32610253.
    48. Sava V, Fihurka O, Khvorova A, Sanchez-Ramos J. Data on enrichment of chitosan nanoparticles for intranasal delivery of oligonucleotides to the brain. Data Brief. 2020 Feb; 28:105093. PMID: 31970274.
    49. Segal M, Biscans A, Gilles ME, Anastasiadou E, De Luca R, Lim J, Khvorova A, Slack FJ. Hydrophobically Modified let-7b miRNA Enhances Biodistribution to NSCLC and Downregulates HMGA2 In?Vivo. Mol Ther Nucleic Acids. 2020 Mar 06; 19:267-277. PMID: 31855835.
    50. Sava V, Fihurka O, Khvorova A, Sanchez-Ramos J. Enriched chitosan nanoparticles loaded with siRNA are effective in lowering Huntington's disease gene expression following intranasal administration. Nanomedicine. 2020 02; 24:102119. PMID: 31666200.
      Citations: 26     Fields:    Translation:Animals
    51. McAdam RL, Morton A, Gordon SL, Alterman JF, Khvorova A, Cousin MA, Smillie KJ. Loss of huntingtin function slows synaptic vesicle endocytosis in striatal neurons from the httQ140/Q140 mouse model of Huntington's disease. Neurobiol Dis. 2020 02; 134:104637. PMID: 31614197.
      Citations: 11     Fields:    Translation:HumansAnimalsCells
    52. Alterman JF, Godinho BMDC, Hassler MR, Ferguson CM, Echeverria D, Sapp E, Haraszti RA, Coles AH, Conroy F, Miller R, Roux L, Yan P, Knox EG, Turanov AA, King RM, Gernoux G, Mueller C, Gray-Edwards HL, Moser RP, Bishop NC, Jaber SM, Gounis MJ, Sena-Esteves M, Pai AA, DiFiglia M, Aronin N, Khvorova A. A divalent siRNA chemical scaffold for potent and sustained modulation of gene expression throughout the central nervous system. Nat Biotechnol. 2019 08; 37(8):884-894. PMID: 31375812.
      Citations: 69     Fields:    Translation:Animals
    53. Haraszti RA, Miller R, Dubuke ML, Rockwell HE, Coles AH, Sapp E, Didiot MC, Echeverria D, Stoppato M, Sere YY, Leszyk J, Alterman JF, Godinho BMDC, Hassler MR, McDaniel J, Narain NR, Wollacott R, Wang Y, Shaffer SA, Kiebish MA, DiFiglia M, Aronin N, Khvorova A. Serum Deprivation of Mesenchymal Stem Cells Improves Exosome Activity and Alters Lipid and Protein Composition. iScience. 2019 Jun 28; 16:230-241. PMID: 31195240.
    54. Watts JK, Brown RH, Khvorova A. Nucleic Acid Therapeutics for Neurological Diseases. Neurotherapeutics. 2019 04; 16(2):245-247. PMID: 31037650.
      Citations: 11     Fields:    Translation:HumansAnimals
    55. Biscans A, Coles A, Echeverria D, Khvorova A. The valency of fatty acid conjugates impacts siRNA pharmacokinetics, distribution, and efficacy in vivo. J Control Release. 2019 05 28; 302:116-125. PMID: 30940496.
      Citations: 25     Fields:    Translation:HumansAnimalsCells
    56. Osborn MF, Coles AH, Biscans A, Haraszti RA, Roux L, Davis S, Ly S, Echeverria D, Hassler MR, Godinho BMDC, Nikan M, Khvorova A. Hydrophobicity drives the systemic distribution of lipid-conjugated siRNAs via lipid transport pathways. Nucleic Acids Res. 2019 02 20; 47(3):1070-1081. PMID: 30535404.
      Citations: 45     Fields:    Translation:HumansAnimalsCells
    57. Biscans A, Coles A, Haraszti R, Echeverria D, Hassler M, Osborn M, Khvorova A. Diverse lipid conjugates for functional extra-hepatic siRNA delivery in vivo. Nucleic Acids Res. 2019 02 20; 47(3):1082-1096. PMID: 30544191.
      Citations: 70     Fields:    Translation:Animals
    58. Thijssen MF, Br?ggenwirth IMA, Gillooly A, Khvorova A, Kowalik TF, Martins PN. Gene Silencing With siRNA (RNA Interference): A New Therapeutic Option During Ex Vivo Machine Liver Perfusion Preservation Liver Transpl. 2019 01; 25(1):140-151. PMID: 30561891.
      Citations: 15     Fields:    Translation:Humans
    59. Tousley A, Iuliano M, Weisman E, Sapp E, Zhang N, Vodicka P, Alexander J, Aviolat H, Gatune L, Reeves P, Li X, Khvorova A, Ellerby LM, Aronin N, DiFiglia M, Kegel-Gleason KB. Rac1 Activity Is Modulated by Huntingtin and Dysregulated in Models of Huntington's Disease. J Huntingtons Dis. 2019; 8(1):53-69. PMID: 30594931.
      Citations: 11     Fields:    Translation:HumansAnimalsCells
    60. Th?ry C, Witwer KW, Aikawa E, Alcaraz MJ, Anderson JD, Andriantsitohaina R, Antoniou A, Arab T, Archer F, Atkin-Smith GK, Ayre DC, Bach JM, Bachurski D, Baharvand H, Balaj L, Baldacchino S, Bauer NN, Baxter AA, Bebawy M, Beckham C, Bedina Zavec A, Benmoussa A, Berardi AC, Bergese P, Bielska E, Blenkiron C, Bobis-Wozowicz S, Boilard E, Boireau W, Bongiovanni A, Borr?s FE, Bosch S, Boulanger CM, Breakefield X, Breglio AM, Brennan M?, Brigstock DR, Brisson A, Broekman ML, Bromberg JF, Bryl-G?recka P, Buch S, Buck AH, Burger D, Busatto S, Buschmann D, Bussolati B, Buz?s EI, Byrd JB, Camussi G, Carter DR, Caruso S, Chamley LW, Chang YT, Chen C, Chen S, Cheng L, Chin AR, Clayton A, Clerici SP, Cocks A, Cocucci E, Coffey RJ, Cordeiro-da-Silva A, Couch Y, Coumans FA, Coyle B, Crescitelli R, Criado MF, D'Souza-Schorey C, Das S, Datta Chaudhuri A, de Candia P, De Santana EF, De Wever O, Del Portillo HA, Demaret T, Deville S, Devitt A, Dhondt B, Di Vizio D, Dieterich LC, Dolo V, Dominguez Rubio AP, Dominici M, Dourado MR, Driedonks TA, Duarte FV, Duncan HM, Eichenberger RM, Ekstr?m K, El Andaloussi S, Elie-Caille C, Erdbr?gger U, Falc?n-P?rez JM, Fatima F, Fish JE, Flores-Bellver M, F?rs?nits A, Frelet-Barrand A, Fricke F, Fuhrmann G, Gabrielsson S, G?mez-Valero A, Gardiner C, G?rtner K, Gaudin R, Gho YS, Giebel B, Gilbert C, Gimona M, Giusti I, Goberdhan DC, G?rgens A, Gorski SM, Greening DW, Gross JC, Gualerzi A, Gupta GN, Gustafson D, Handberg A, Haraszti RA, Harrison P, Hegyesi H, Hendrix A, Hill AF, Hochberg FH, Hoffmann KF, Holder B, Holthofer H, Hosseinkhani B, Hu G, Huang Y, Huber V, Hunt S, Ibrahim AG, Ikezu T, Inal JM, Isin M, Ivanova A, Jackson HK, Jacobsen S, Jay SM, Jayachandran M, Jenster G, Jiang L, Johnson SM, Jones JC, Jong A, Jovanovic-Talisman T, Jung S, Kalluri R, Kano SI, Kaur S, Kawamura Y, Keller ET, Khamari D, Khomyakova E, Khvorova A, Kierulf P, Kim KP, Kislinger T, Klingeborn M, Klinke DJ, Kornek M, Kosanovic MM, Kov?cs ?F, Kr?mer-Albers EM, Krasemann S, Krause M, Kurochkin IV, Kusuma GD, Kuypers S, Laitinen S, Langevin SM, Languino LR, Lannigan J, L?sser C, Laurent LC, Lavieu G, L?zaro-Ib??ez E, Le Lay S, Lee MS, Lee YXF, Lemos DS, Lenassi M, Leszczynska A, Li IT, Liao K, Libregts SF, Ligeti E, Lim R, Lim SK, Line A, Linnemannst?ns K, Llorente A, Lombard CA, Lorenowicz MJ, L?rincz ?M, L?tvall J, Lovett J, Lowry MC, Loyer X, Lu Q, Lukomska B, Lunavat TR, Maas SL, Malhi H, Marcilla A, Mariani J, Mariscal J, Martens-Uzunova ES, Martin-Jaular L, Martinez MC, Martins VR, Mathieu M, Mathivanan S, Maugeri M, McGinnis LK, McVey MJ, Meckes DG, Meehan KL, Mertens I, Minciacchi VR, M?ller A, M?ller J?rgensen M, Morales-Kastresana A, Morhayim J, Mullier F, Muraca M, Musante L, Mussack V, Muth DC, Myburgh KH, Najrana T, Nawaz M, Nazarenko I, Nejsum P, Neri C, Neri T, Nieuwland R, Nimrichter L, Nolan JP, Nolte-'t Hoen EN, Noren Hooten N, O'Driscoll L, O'Grady T, O'Loghlen A, Ochiya T, Olivier M, Ortiz A, Ortiz LA, Osteikoetxea X, ?stergaard O, Ostrowski M, Park J, Pegtel DM, Peinado H, Perut F, Pfaffl MW, Phinney DG, Pieters BC, Pink RC, Pisetsky DS, Pogge von Strandmann E, Polakovicova I, Poon IK, Powell BH, Prada I, Pulliam L, Quesenberry P, Radeghieri A, Raffai RL, Raimondo S, Rak J, Ramirez MI, Raposo G, Rayyan MS, Regev-Rudzki N, Ricklefs FL, Robbins PD, Roberts DD, Rodrigues SC, Rohde E, Rome S, Rouschop KM, Rughetti A, Russell AE, Sa? P, Sahoo S, Salas-Huenuleo E, S?nchez C, Saugstad JA, Saul MJ, Schiffelers RM, Schneider R, Sch?yen TH, Scott A, Shahaj E, Sharma S, Shatnyeva O, Shekari F, Shelke GV, Shetty AK, Shiba K, Siljander PR, Silva AM, Skowronek A, Snyder OL, Soares RP, S?dar BW, Soekmadji C, Sotillo J, Stahl PD, Stoorvogel W, Stott SL, Strasser EF, Swift S, Tahara H, Tewari M, Timms K, Tiwari S, Tixeira R, Tkach M, Toh WS, Tomasini R, Torrecilhas AC, Tosar JP, Toxavidis V, Urbanelli L, Vader P, van Balkom BW, van der Grein SG, Van Deun J, van Herwijnen MJ, Van Keuren-Jensen K, van Niel G, et al. 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. J Extracell Vesicles. 2018; 7(1):1535750. PMID: 30637094.
      Citations: 4263     Fields:    
    61. Turanov AA, Lo A, Hassler MR, Makris A, Ashar-Patel A, Alterman JF, Coles AH, Haraszti RA, Roux L, Godinho BMDC, Echeverria D, Pears S, Iliopoulos J, Shanmugalingam R, Ogle R, Zsengeller ZK, Hennessy A, Karumanchi SA, Moore MJ, Khvorova A. RNAi modulation of placental sFLT1 for the treatment of preeclampsia. Nat Biotechnol. 2018 Nov 19. PMID: 30451990.
      Citations: 55     Fields:    
    62. Shmushkovich T, Monopoli KR, Homsy D, Leyfer D, Betancur-Boissel M, Khvorova A, Wolfson AD. Functional features defining the efficacy of cholesterol-conjugated, self-deliverable, chemically modified siRNAs. Nucleic Acids Res. 2018 11 16; 46(20):10905-10916. PMID: 30169779.
      Citations: 26     Fields:    Translation:HumansCells
    63. Haraszti RA, Miller R, Stoppato M, Sere YY, Coles A, Didiot MC, Wollacott R, Sapp E, Dubuke ML, Li X, Shaffer SA, DiFiglia M, Wang Y, Aronin N, Khvorova A. Exosomes Produced from 3D Cultures of MSCs by Tangential Flow Filtration Show Higher Yield and Improved Activity. Mol Ther. 2018 12 05; 26(12):2838-2847. PMID: 30341012.
      Citations: 161     Fields:    Translation:AnimalsCells
    64. Didiot MC, Ferguson CM, Ly S, Coles AH, Smith AO, Bicknell AA, Hall LM, Sapp E, Echeverria D, Pai AA, DiFiglia M, Moore MJ, Hayward LJ, Aronin N, Khvorova A. Nuclear Localization of Huntingtin mRNA Is Specific to Cells of Neuronal Origin. Cell Rep. 2018 09 04; 24(10):2553-2560.e5. PMID: 30184490.
      Citations: 22     Fields:    Translation:HumansAnimalsCells
    65. Godinho BMDC, Henninger N, Bouley J, Alterman JF, Haraszti RA, Gilbert JW, Sapp E, Coles AH, Biscans A, Nikan M, Echeverria D, DiFiglia M, Aronin N, Khvorova A. Transvascular Delivery of Hydrophobically Modified siRNAs: Gene Silencing in the Rat Brain upon Disruption of the Blood-Brain Barrier. Mol Ther. 2018 11 07; 26(11):2580-2591. PMID: 30143435.
      Citations: 19     Fields:    Translation:HumansAnimalsCells
    66. Mir A, Alterman JF, Hassler MR, Debacker AJ, Hudgens E, Echeverria D, Brodsky MH, Khvorova A, Watts JK, Sontheimer EJ. Heavily and fully modified RNAs guide efficient SpyCas9-mediated genome editing. Nat Commun. 2018 Jul 06; 9(1):2641. PMID: 29980686.
      Citations: 36     Fields:    Translation:HumansCells
    67. Sharma VK, Osborn MF, Hassler MR, Echeverria D, Ly S, Ulashchik EA, Martynenko-Makaev YV, Shmanai VV, Zatsepin TS, Khvorova A, Watts JK. Novel Cluster and Monomer-Based GalNAc Structures Induce Effective Uptake of siRNAs in Vitro and in Vivo. Bioconjug Chem. 2018 07 18; 29(7):2478-2488. PMID: 29898368.
      Citations: 15     Fields:    Translation:AnimalsCells
    68. Haraszti RA, Miller R, Didiot MC, Biscans A, Alterman JF, Hassler MR, Roux L, Echeverria D, Sapp E, DiFiglia M, Aronin N, Khvorova A. Optimized Cholesterol-siRNA Chemistry Improves Productive Loading onto Extracellular Vesicles. Mol Ther. 2018 08 01; 26(8):1973-1982. PMID: 29937418.
      Citations: 36     Fields:    Translation:HumansAnimalsCells
    69. Osborn MF, Khvorova A. Improving siRNA Delivery In Vivo Through Lipid Conjugation. Nucleic Acid Ther. 2018 06; 28(3):128-136. PMID: 29746209.
      Citations: 54     Fields:    Translation:HumansAnimals
    70. Osborn MF, Coles AH, Golebiowski D, Echeverria D, Moazami MP, Watts JK, Sena-Esteves M, Khvorova A. Efficient Gene Silencing in Brain Tumors with Hydrophobically Modified siRNAs. Mol Cancer Ther. 2018 06; 17(6):1251-1258. PMID: 29654062.
      Citations: 6     Fields:    Translation:HumansAnimalsCells
    71. Biscans A, Haraszti RA, Echeverria D, Miller R, Didiot MC, Nikan M, Roux L, Aronin N, Khvorova A. Hydrophobicity of Lipid-Conjugated siRNAs Predicts Productive Loading to Small Extracellular Vesicles. Mol Ther. 2018 06 06; 26(6):1520-1528. PMID: 29699940.
      Citations: 14     Fields:    Translation:HumansCells
    72. Hassler MR, Turanov AA, Alterman JF, Haraszti RA, Coles AH, Osborn MF, Echeverria D, Nikan M, Salomon WE, Roux L, Godinho BMDC, Davis SM, Morrissey DV, Zamore PD, Karumanchi SA, Moore MJ, Aronin N, Khvorova A. Comparison of partially and fully chemically-modified siRNA in conjugate-mediated delivery in vivo. Nucleic Acids Res. 2018 03 16; 46(5):2185-2196. PMID: 29432571.
      Citations: 71     Fields:    Translation:HumansAnimalsCells
    73. Stoddard BL, Khvorova A, Corey DR, Dynan WS, Fox KR. Editorial: Nucleic Acids Research and Nucleic Acid Therapeutics. Nucleic Acids Res. 2018 02 28; 46(4):1563-1564. PMID: 29506274.
      Citations: 1     Fields:    
    74. Didiot MC, Haraszti RA, Aronin N, Khvorova A. Loading of Extracellular Vesicles with Hydrophobically Modified siRNAs. Methods Mol Biol. 2018; 1740:199-214. PMID: 29388146.
      Citations: 6     Fields:    Translation:HumansAnimalsCells
    75. Sanchez-Ramos J, Song S, Kong X, Foroutan P, Martinez G, Dominguez-Viqueria W, Mohapatra S, Mohapatra S, Haraszti RA, Khvorova A, Aronin N, Sava V. Chitosan-Mangafodipir nanoparticles designed for intranasal delivery of siRNA and DNA to brain. J Drug Deliv Sci Technol. 2018 Feb; 43:453-460. PMID: 29805475.
    76. Godinho BMDC, Gilbert JW, Haraszti RA, Coles AH, Biscans A, Roux L, Nikan M, Echeverria D, Hassler M, Khvorova A. Pharmacokinetic Profiling of Conjugated Therapeutic Oligonucleotides: A High-Throughput Method Based Upon Serial Blood Microsampling Coupled to Peptide Nucleic Acid Hybridization Assay. Nucleic Acid Ther. 2017 Dec; 27(6):323-334. PMID: 29022758.
      Citations: 27     Fields:    Translation:AnimalsCells
    77. Alterman JF, Coles AH, Hall LM, Aronin N, Khvorova A, Didiot MC. A High-throughput Assay for mRNA Silencing in Primary Cortical Neurons in vitro with Oligonucleotide Therapeutics. Bio Protoc. 2017 Aug 20; 7(16). PMID: 28966945.
    78. Sharma VK, Singh SK, Krishnamurthy PM, Alterman JF, Haraszti RA, Khvorova A, Prasad AK, Watts JK. Synthesis and biological properties of triazole-linked locked nucleic acid. Chem Commun (Camb). 2017 Aug 03; 53(63):8906-8909. PMID: 28736781.
      Citations: 13     Fields:    Translation:Cells
    79. Haraszti RA, Roux L, Coles AH, Turanov AA, Alterman JF, Echeverria D, Godinho BMDC, Aronin N, Khvorova A. 5?-Vinylphosphonate improves tissue accumulation and efficacy of conjugated siRNAs in vivo. Nucleic Acids Res. 2017 Jul 27; 45(13):7581-7592. PMID: 28591791.
      Citations: 46     Fields:    Translation:HumansAnimalsCells
    80. Haraszti RA, Coles A, Aronin N, Khvorova A, Didiot MC. Loading of Extracellular Vesicles with Chemically Stabilized Hydrophobic siRNAs for the Treatment of Disease in the Central Nervous System. Bio Protoc. 2017 Jun 20; 7(20). PMID: 28868334.
    81. Nikan M, Osborn MF, Coles AH, Biscans A, Godinho BMDC, Haraszti RA, Sapp E, Echeverria D, DiFiglia M, Aronin N, Khvorova A. Synthesis and Evaluation of Parenchymal Retention and Efficacy of a Metabolically Stable O-Phosphocholine-N-docosahexaenoyl-l-serine siRNA Conjugate in Mouse Brain. Bioconjug Chem. 2017 06 21; 28(6):1758-1766. PMID: 28462988.
      Citations: 21     Fields:    Translation:Animals
    82. Khvorova A, Watts JK. The chemical evolution of oligonucleotide therapies of clinical utility. Nat Biotechnol. 2017 Mar; 35(3):238-248. PMID: 28244990.
      Citations: 353     Fields:    Translation:HumansAnimals
    83. Khvorova A. Oligonucleotide Therapeutics - A New Class of Cholesterol-Lowering Drugs. N Engl J Med. 2017 Jan 05; 376(1):4-7. PMID: 28052224.
      Citations: 49     Fields:    Translation:HumansCells
    84. Ly S, Navaroli DM, Didiot MC, Cardia J, Pandarinathan L, Alterman JF, Fogarty K, Standley C, Lifshitz LM, Bellve KD, Prot M, Echeverria D, Corvera S, Khvorova A. Visualization of self-delivering hydrophobically modified siRNA cellular internalization. Nucleic Acids Res. 2017 01 09; 45(1):15-25. PMID: 27899655.
      Citations: 47     Fields:    Translation:HumansAnimalsCells
    85. Haraszti RA, Didiot MC, Sapp E, Leszyk J, Shaffer SA, Rockwell HE, Gao F, Narain NR, DiFiglia M, Kiebish MA, Aronin N, Khvorova A. High-resolution proteomic and lipidomic analysis of exosomes and microvesicles from different cell sources. J Extracell Vesicles. 2016; 5:32570. PMID: 27863537.
      Citations: 297     Fields:    
    86. Nikan M, Osborn MF, Coles AH, Godinho BM, Hall LM, Haraszti RA, Hassler MR, Echeverria D, Aronin N, Khvorova A. Docosahexaenoic Acid Conjugation Enhances Distribution and Safety of siRNA upon Local Administration in Mouse Brain. Mol Ther Nucleic Acids. 2016 Aug 09; 5(8):e344. PMID: 27504598.
    87. Didiot MC, Hall LM, Coles AH, Haraszti RA, Godinho BM, Chase K, Sapp E, Ly S, Alterman JF, Hassler MR, Echeverria D, Raj L, Morrissey DV, DiFiglia M, Aronin N, Khvorova A. Exosome-mediated Delivery of Hydrophobically Modified siRNA for Huntingtin mRNA Silencing. Mol Ther. 2016 10; 24(10):1836-1847. PMID: 27506293.
      Citations: 179     Fields:    Translation:HumansAnimalsCells
    88. Amit I, Baker D, Barker R, Berger B, Bertozzi C, Bhatia S, Biffi A, Demichelis F, Doudna J, Dowdy SF, Endy D, Helmstaedter M, Junca H, June C, Kamb S, Khvorova A, Kim DH, Kim JS, Krishnan Y, Lakadamyali M, Lappalainen T, Lewin S, Liao J, Loman N, Lundberg E, Lynd L, Martin C, Mellman I, Miyawaki A, Mummery C, Nelson K, Paz J, Peralta-Yahya P, Picotti P, Polyak K, Prather K, Qin J, Quake S, Regev A, Rogers JA, Shetty R, Sommer M, Stevens M, Stolovitzky G, Takahashi M, Tang F, Teichmann S, Torres-Padilla ME, Tripathi L, Vemula P, Verdine G, Vollmer F, Wang J, Ying JY, Zhang F, Zhang T. Voices of biotech. Nat Biotechnol. 2016 Mar; 34(3):270-5. PMID: 26963549.
      Citations: 2     Fields:    Translation:Humans
    89. Alterman JF, Hall LM, Coles AH, Hassler MR, Didiot MC, Chase K, Abraham J, Sottosanti E, Johnson E, Sapp E, Osborn MF, Difiglia M, Aronin N, Khvorova A. Hydrophobically Modified siRNAs Silence Huntingtin mRNA in Primary Neurons and Mouse Brain. Mol Ther Nucleic Acids. 2015 Dec 01; 4:e266. PMID: 26623938.
    90. Coles AH, Osborn MF, Alterman JF, Turanov AA, Godinho BM, Kennington L, Chase K, Aronin N, Khvorova A. A High-Throughput Method for Direct Detection of Therapeutic Oligonucleotide-Induced Gene Silencing In Vivo. Nucleic Acid Ther. 2016 Apr; 26(2):86-92. PMID: 26595721.
      Citations: 28     Fields:    Translation:Animals
    91. Osborn MF, Alterman JF, Nikan M, Cao H, Didiot MC, Hassler MR, Coles AH, Khvorova A. Guanabenz (Wytensin?) selectively enhances uptake and efficacy of hydrophobically modified siRNAs. Nucleic Acids Res. 2015 Oct 15; 43(18):8664-72. PMID: 26400165.
      Citations: 22     Fields:    Translation:HumansCells
    92. Khvorova A, Osborn MF, Hassler MR. Corrigendum: Taking charge of siRNA delivery. Nat Biotechnol. 2015 May; 33(5):563. PMID: 25965761.
      Citations:    Fields:    
    93. Khvorova A, Osborn MF, Hassler MR. Taking charge of siRNA delivery. Nat Biotechnol. 2014 Dec; 32(12):1197-8. PMID: 25489835.
      Citations: 8     Fields:    Translation:HumansAnimals
    94. Byrne M, Tzekov R, Wang Y, Rodgers A, Cardia J, Ford G, Holton K, Pandarinathan L, Lapierre J, Stanney W, Bulock K, Shaw S, Libertine L, Fettes K, Khvorova A, Kaushal S, Pavco P. Novel hydrophobically modified asymmetric RNAi compounds (sd-rxRNA) demonstrate robust efficacy in the eye. J Ocul Pharmacol Ther. 2013 Dec; 29(10):855-64. PMID: 24180627.
      Citations: 40     Fields:    Translation:HumansAnimalsCells
    95. Khvorova A, Wolfson A. New competition in RNA regulation. Nat Biotechnol. 2012 Jan 09; 30(1):58-9. PMID: 22231098.
      Citations: 12     Fields:    Translation:HumansCells
    96. Lapierre J, Salomon W, Cardia J, Bulock K, Lam JT, Stanney WJ, Ford G, Smith-Anzures B, Woolf T, Kamens J, Khvorova A, Samarsky D. Potent and systematic RNAi mediated silencing with single oligonucleotide compounds. RNA. 2011 Jun; 17(6):1032-7. PMID: 21493786.
      Citations: 7     Fields:    Translation:HumansCells
    97. Robertson B, Dalby AB, Karpilow J, Khvorova A, Leake D, Vermeulen A. Specificity and functionality of microRNA inhibitors. Silence. 2010 Apr 01; 1(1):10. PMID: 20359337.
    98. Simpson KJ, Selfors LM, Bui J, Reynolds A, Leake D, Khvorova A, Brugge JS. Identification of genes that regulate epithelial cell migration using an siRNA screening approach. Nat Cell Biol. 2008 Sep; 10(9):1027-38. PMID: 19160483.
      Citations: 221     Fields:    Translation:HumansCells
    99. Anderson EM, Birmingham A, Baskerville S, Reynolds A, Maksimova E, Leake D, Fedorov Y, Karpilow J, Khvorova A. Experimental validation of the importance of seed complement frequency to siRNA specificity. RNA. 2008 May; 14(5):853-61. PMID: 18367722.
      Citations: 68     Fields:    Translation:HumansCells
    100. Anderson E, Boese Q, Khvorova A, Karpilow J. Identifying siRNA-induced off-targets by microarray analysis. Methods Mol Biol. 2008; 442:45-63. PMID: 18369777.
      Citations: 12     Fields:    Translation:Humans
    101. Li L, Lin X, Khvorova A, Fesik SW, Shen Y. Defining the optimal parameters for hairpin-based knockdown constructs. RNA. 2007 Oct; 13(10):1765-74. PMID: 17698642.
      Citations: 47     Fields:    Translation:HumansCells
    102. Vermeulen A, Robertson B, Dalby AB, Marshall WS, Karpilow J, Leake D, Khvorova A, Baskerville S. Double-stranded regions are essential design components of potent inhibitors of RISC function. RNA. 2007 May; 13(5):723-30. PMID: 17400817.
      Citations: 63     Fields:    Translation:HumansCells
    103. Birmingham A, Anderson E, Sullivan K, Reynolds A, Boese Q, Leake D, Karpilow J, Khvorova A. A protocol for designing siRNAs with high functionality and specificity. Nat Protoc. 2007; 2(9):2068-78. PMID: 17853862.
      Citations: 81     Fields:    Translation:Humans
    104. Jackson AL, Burchard J, Leake D, Reynolds A, Schelter J, Guo J, Johnson JM, Lim L, Karpilow J, Nichols K, Marshall W, Khvorova A, Linsley PS. Position-specific chemical modification of siRNAs reduces "off-target" transcript silencing. RNA. 2006 Jul; 12(7):1197-205. PMID: 16682562.
      Citations: 301     Fields:    Translation:HumansCells
    105. Fedorov Y, Anderson EM, Birmingham A, Reynolds A, Karpilow J, Robinson K, Leake D, Marshall WS, Khvorova A. Off-target effects by siRNA can induce toxic phenotype. RNA. 2006 Jul; 12(7):1188-96. PMID: 16682561.
      Citations: 183     Fields:    Translation:HumansCells
    106. Reynolds A, Anderson EM, Vermeulen A, Fedorov Y, Robinson K, Leake D, Karpilow J, Marshall WS, Khvorova A. Induction of the interferon response by siRNA is cell type- and duplex length-dependent. RNA. 2006 Jun; 12(6):988-93. PMID: 16611941.
      Citations: 123     Fields:    Translation:HumansCells
    107. Birmingham A, Anderson EM, Reynolds A, Ilsley-Tyree D, Leake D, Fedorov Y, Baskerville S, Maksimova E, Robinson K, Karpilow J, Marshall WS, Khvorova A. 3' UTR seed matches, but not overall identity, are associated with RNAi off-targets. Nat Methods. 2006 Mar; 3(3):199-204. PMID: 16489337.
      Citations: 358     Fields:    Translation:HumansCells
    108. Benimetskaya L, Lai JC, Khvorova A, Wu S, Miller P, Stein CA. Induction of apoptosis by G3139 in melanoma cells. Ann N Y Acad Sci. 2005 Nov; 1058:235-45. PMID: 16394140.
      Citations:    Fields:    Translation:HumansAnimalsCells
    109. Vermeulen A, Behlen L, Reynolds A, Wolfson A, Marshall WS, Karpilow J, Khvorova A. The contributions of dsRNA structure to Dicer specificity and efficiency. RNA. 2005 May; 11(5):674-82. PMID: 15811921.
      Citations: 116     Fields:    Translation:HumansCells
    110. Fedorov Y, King A, Anderson E, Karpilow J, Ilsley D, Marshall W, Khvorova A. Different delivery methods-different expression profiles. Nat Methods. 2005 Apr; 2(4):241. PMID: 15782213.
      Citations: 24     Fields:    
    111. Lai JC, Benimetskaya L, Khvorova A, Wu S, Hua E, Miller P, Stein CA. Phosphorothioate oligodeoxynucleotides and G3139 induce apoptosis in 518A2 melanoma cells. Mol Cancer Ther. 2005 Feb; 4(2):305-15. PMID: 15713901.
      Citations: 8     Fields:    Translation:HumansCells
    112. Kisseleva N, Khvorova A, Westhof E, Schiemann O. Binding of manganese(II) to a tertiary stabilized hammerhead ribozyme as studied by electron paramagnetic resonance spectroscopy. RNA. 2005 Jan; 11(1):1-6. PMID: 15611296.
      Citations: 19     Fields:    Translation:Cells
    113. Boese Q, Leake D, Reynolds A, Read S, Scaringe SA, Marshall WS, Khvorova A. Mechanistic insights aid computational short interfering RNA design. Methods Enzymol. 2005; 392:73-96. PMID: 15644176.
      Citations: 26     Fields:    Translation:HumansAnimalsCells
    114. Benimetskaya L, Lai JC, Khvorova A, Wu S, Hua E, Miller P, Zhang LM, Stein CA. Relative Bcl-2 independence of drug-induced cytotoxicity and resistance in 518A2 melanoma cells. Clin Cancer Res. 2004 Dec 15; 10(24):8371-9. PMID: 15623615.
      Citations: 9     Fields:    Translation:HumansCells
    115. Saksmerprome V, Roychowdhury-Saha M, Jayasena S, Khvorova A, Burke DH. Artificial tertiary motifs stabilize trans-cleaving hammerhead ribozymes under conditions of submillimolar divalent ions and high temperatures. RNA. 2004 Dec; 10(12):1916-24. PMID: 15547137.
      Citations: 36     Fields:    Translation:Cells
    116. Canny MD, Jucker FM, Kellogg E, Khvorova A, Jayasena SD, Pardi A. Fast cleavage kinetics of a natural hammerhead ribozyme. J Am Chem Soc. 2004 Sep 08; 126(35):10848-9. PMID: 15339162.
      Citations: 82     Fields:    Translation:AnimalsCells
    117. Miller JT, Khvorova A, Scaringe SA, Le Grice SF. Synthetic tRNALys,3 as the replication primer for the HIV-1HXB2 and HIV-1Mal genomes. Nucleic Acids Res. 2004; 32(15):4687-95. PMID: 15342789.
      Citations: 3     Fields:    Translation:Cells
    118. Raffo A, Lai JC, Stein CA, Miller P, Scaringe S, Khvorova A, Benimetskaya L. Antisense RNA down-regulation of bcl-2 expression in DU145 prostate cancer cells does not diminish the cytostatic effects of G3139 (Oblimersen). Clin Cancer Res. 2004 May 01; 10(9):3195-206. PMID: 15131061.
      Citations: 8     Fields:    Translation:HumansCells
    119. Penedo JC, Wilson TJ, Jayasena SD, Khvorova A, Lilley DM. Folding of the natural hammerhead ribozyme is enhanced by interaction of auxiliary elements. RNA. 2004 May; 10(5):880-8. PMID: 15100442.
      Citations: 66     Fields:    Translation:Cells
    120. Huang F, Khvorova A, Marshall W, Sorkin A. Analysis of clathrin-mediated endocytosis of epidermal growth factor receptor by RNA interference. J Biol Chem. 2004 Apr 16; 279(16):16657-61. PMID: 14985334.
      Citations: 223     Fields:    Translation:HumansCells
    121. Hsieh AC, Bo R, Manola J, Vazquez F, Bare O, Khvorova A, Scaringe S, Sellers WR. A library of siRNA duplexes targeting the phosphoinositide 3-kinase pathway: determinants of gene silencing for use in cell-based screens. Nucleic Acids Res. 2004; 32(3):893-901. PMID: 14769947.
      Citations: 71     Fields:    Translation:HumansCells
    122. Li T, Chang CY, Jin DY, Lin PJ, Khvorova A, Stafford DW. Identification of the gene for vitamin K epoxide reductase. Nature. 2004 Feb 05; 427(6974):541-4. PMID: 14765195.
      Citations: 207     Fields:    Translation:HumansAnimalsCells
    123. Reynolds A, Leake D, Boese Q, Scaringe S, Marshall WS, Khvorova A. Rational siRNA design for RNA interference. Nat Biotechnol. 2004 Mar; 22(3):326-30. PMID: 14758366.
      Citations: 755     Fields:    Translation:HumansAnimalsCells
    124. Khvorova A, Reynolds A, Jayasena SD. Functional siRNAs and miRNAs exhibit strand bias. Cell. 2003 Oct 17; 115(2):209-16. PMID: 14567918.
      Citations: 985     Fields:    Translation:HumansAnimalsCells
    125. Khvorova A, Lescoute A, Westhof E, Jayasena SD. Sequence elements outside the hammerhead ribozyme catalytic core enable intracellular activity. Nat Struct Biol. 2003 Sep; 10(9):708-12. PMID: 12881719.
      Citations: 191     Fields:    Translation:Cells
    126. Wilson JA, Jayasena S, Khvorova A, Sabatinos S, Rodrigue-Gervais IG, Arya S, Sarangi F, Harris-Brandts M, Beaulieu S, Richardson CD. RNA interference blocks gene expression and RNA synthesis from hepatitis C replicons propagated in human liver cells. Proc Natl Acad Sci U S A. 2003 Mar 04; 100(5):2783-8. PMID: 12594341.
      Citations: 92     Fields:    Translation:HumansCells
    127. Wolfson AD, Khvorova AM, Sauter C, Florentz C, Gieg? R. Mimics of yeast tRNAAsp and their recognition by aspartyl-tRNA synthetase. Biochemistry. 1999 Sep 14; 38(37):11926-32. PMID: 10508395.
      Citations: 7     Fields:    Translation:AnimalsCells
    128. Khvorova AM, Motorin IuA, Vol'fson AD. [Mechanism of discrimination of tRNA(Phe) from E. coli by yeast phenylalanine-tRNA-synthetase]. Biokhimiia. 1993 Apr; 58(4):613-9. PMID: 8389606.
      Citations:    Fields:    Translation:AnimalsCells
    129. Khvorova AM, Wolfson AD, Gladilin KL. Anticodon-dependent aminoacylation of RNA minisubstrate by lysyl-tRNA synthetase. FEBS Lett. 1992 Dec 21; 314(3):256-8. PMID: 1281788.
      Citations: 4     Fields:    Translation:AnimalsCells
    130. Khvorova AM, Motorin IuA, Vol'fson AD. [Pyrophosphate-dependent inactivation of tyrosyl-tRNA synthetase during aminoacylation of heterologous tRNA]. Biokhimiia. 1992 Dec; 57(12):1913-6. PMID: 1338185.
      Citations:    Fields:    Translation:AnimalsCells
    131. Khvorova AM, Wolfson AD. Crucial role of pyrophosphate in the aminoacylation of E. coli tRNA(Phe) by yeast phenylalanyl-tRNA synthetase. FEBS Lett. 1992 Oct 19; 311(2):139-42. PMID: 1383036.
      Citations: 2     Fields:    Translation:AnimalsCells
    132. Khvorova AM, Motorin IuA, Vol'fson AD, Gladilin KL. [Specific aminoacylation of oligo U-CCA lysyl-tRNA-synthetase molecules]. Dokl Akad Nauk. 1992; 325(1):179-82. PMID: 1425198.
      Citations:    Fields:    Translation:AnimalsCells
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