Publications

Industrial Publications

Sanhita Ghosh, Sayeed Mohammad Firdous and Anirban Nath. siRNA could be a potential therapy for COVID-19. EXCLI J. 2020 Apr 22;19:528-531. DOI:10.17179/excli2020-1328

Hasan Uludağ, Kylie Parent, Hamidreza Montazeri Aliabadi and Azita Haddadi. Prospects for RNAi Therapy of COVID-19. Front. Bioeng. Biotechnol. 30 July 2020. DOI: 10.3389/fbioe.2020.00916

Cardia J, Samarsky D, Woolf T. RNAi Delivery – Self-delivering RNAi compounds. Drug Delivery Technology .2010.

Peer-Reviewed Publications

Edward F.Boumil, NileymaCastro, Andrew T.Phillips, Jon E.Chatterton, Sean M.McCauley, Alexey D.Wolfson, Taisia Shmushkovich, Marc Ridilla, Audrey M.Bernstein. USP10 Targeted Self-Deliverable siRNA to Prevent Scarring in the Cornea. ScienceDirect. Volume 21, P1029-1043, September 04, 2020. DOI : https://doi.org/10.1016/j.omtn.2020.07.032

Alterman, J.F., Godinho, B.M.D.C., Hassler, M.R., Khvorova A.et al. A divalent siRNA chemical scaffold for potent and sustained modulation of gene expression throughout the central nervous system. Nature Biotechnology. 37, pages 884–894(2019). DOI: https://doi.org/10.1038

Osborn MF, Khvorova A. Improving siRNA Delivery In Vivo Through Lipid Conjugation. Nucleic Acid Ther. 2018 Jun;28(3):128-136. DOI: https://doi.org/10.1089/nat.2018.0725

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 Sep 4;24(10):2553-2560.e5. DOI: https://doi.org/10.1016/j.celrep.2018.07.106

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 Research. Volume 46, Issue 20, 16 November 2018, Pages 10905–10916. DOI: https://doi.org/10.1093/nar/gky745

Ligtenberg M, Pico de Coaña Y, Shmushkovich T, Yoshimoto Y, Truxova I, Yang Y, Betancur-Boissel M, Eliseev A, Wolfson A, Kiessling R. Self-delivering RNA interference targeting PD-1 improves tumor specific T cell functionality for adoptive cell therapy of malignant melanoma. Molecular Therapy. 2018 Jun 6;26(6):1482-1493. DOI : https://doi.org/10.1016/j.ymthe.2018.04.015

Basova LV, Tang X, Umasume T, Gromova A, Zyrianova T, Shmushkovich T, Wolfson A, Hawley D, Zoukhri D, Shestopalov VI, Makarenkova HP. Manipulation of Panx1 Activity Increases the Engraftment of Transplanted Lacrimal Gland Epithelial Progenitor Cells. Invest Ophthalmol Vis Sci. 2017 Nov 1;58(13):5654-5665. DOI: https://doi.org/10.1167/iovs.17-22071.

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 on Upon Serial Blood Microsampling Coupled to Peptide Nucleic Acid Hybridization Assay Nucleic Acid Therapeutics. 2017 Dec;27(6):323-334. DOI : https://doi.org/10.1089/nat.2017.0690.

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):e2501. DOI: https://doi.org/10.21769/BioProtoc.2501.

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):e2338. DOI: https://doi.org/10.21769/BioProtoc.2338.

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. Bioconjugate Chemistry. 2017 Aug 3;53(63):8906-8909. DOI: https://doi.org/10.1021/acs.bioconjchem.

Khvorova A, Watts JK. The chemical evolution of oligonucleotide therapies of clinical utility. Nat Biotechnol. 2017 Mar;35(3):238-248. DOI: 10.1038/nbt.3765.The chemical evolution of oligonucleotide therapies of clinical utility. Nature Biotechnology. 2017 Mar;35(3):238-248. DOI: https://doi.org/10.1038/nbt.3765.

Khvorova A. (2017) New England Journal of Medicine.Oligonucleotide Therapeutics - A New Class of Cholesterol-Lowering Drugs. New England Journal of Medicine. 2017; 376:4-7. DOI: https://doi.org/10.1056/NEJMp1614154.

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., Prot, M., Echeverria, D., Corvera, S., & Khvorova, A. (2017). Visualization of self-delivering hydrophobically modified siRNA cellular internalization. Nucleic acids research vol. 45,1 (2017): 15-25. DOI: https://doi.org/10.1093/nar/gkw1005

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 Oct;24(10):1836-1847. DOI: https://doi.org/10.1038/mt.2016.126.

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 9;5(8):e344. DOI: https://doi.org/10.1038/mtna.2016.50.

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 1;4(12):e266. DOI: https://doi.org/10.1038/mtna.2015.38.

Shtam TA, Kovalev RA, Varfolomeeva EY, Makarov EM, Kil YV, Filatov MV. Exosomes are natural carriers of exogenous siRNA to human cells in vitro. Cell Commun Signal. 2013 Nov 18;11:88. DOI: https://doi.org/10.1186/1478-811X-11-88.

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. DOI: https://doi.org/10.1089/jop.2013.0148.

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. DOI: https://doi.org/10.1089/nat.2015.0578.

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. DOI: https://doi.org/10.1093/nar/gkv942.