Industry Publications

RNAi Delivery – Self-delivering RNAi compounds
Cardia J, Samarsky D, Woolf T. (2010) Drug Delivery Technology.
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Peer-Reviewed Publications

A divalent siRNA chemical scaffold for potent and sustained modulation of gene expression throughout the central nervous system
Alterman JF, Godinho BMDC, Hassler MR, Ferguson CM, Echeverria C, 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. (2019) Nature Biotechnology.
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Functional features defining the efficacy of cholesterol-conjugated, self-deliverable, chemically modified siRNAs
Shmushkovich T, Monopoli KR, Homsy D, Leyfer D, Betancur-Boissel M, Khvorova A, Wolfson AD. (2018) Nucleic Acids Research.
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Self-delivering RNA interference targeting PD-1 improves tumor specific T cell functionality for adoptive cell therapy of malignant melanoma
Ligtenberg M, Pico de Coaña Y, Shmushkovich T, Yoshimoto Y, Truxova I, Yang Y, Betancur-Boissel M, Eliseev A, Wolfson A, Kiessling R. (2018) Molecular Therapy.
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Manipulation of Panx1 Activity Increases the Engraftment of Transplanted Lacrimal Gland Epithelial Progenitor Cells
Basova LV, Tang X, Umasume T, Gromova A, Zyrianova T, Shmushkovich T, Wolfson A, Hawley D, Zoukhri D, Shestopalov VI, Makarenkova HP. (2017) Investigative Ophthalmology & Visual Science. 
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Pharmacokinetic Profiling of Conjugated Therapeutic Oligonucleotides: A High-Throughput Method Based on Upon Serial Blood Microsampling Coupled to Peptide Nucleic Acid Hybridization Assay
Godinho BMDC, Gilbert JW, Haraszti RA, Coles AH, Biscans A, Roux L, Nikan M, Echeverria D, Hassler M, Khvorova A. (2017) Nucleic Acid Therapeutics.
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A High-throughput Assay for mRNA Silencing in Primary Cortical Neurons in vitro with Oligonucleotide Therapeutics
Alterman JF, Coles AH, Hall LM, Aronin N, Khvorova A, Didiot MC. (2017) Bio Protocol.
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Loading of Extracellular Vesicles with Chemically Stabilized Hydrophobic siRNAs for the Treatent of Disease in the Central Nervous System
Haraszti RA, Coles A, Aronin N, Khvorova A, Didiot MC. (2017) Bio Protocol.
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Synthesis and biological properties of triazole-linked locked nucleic acid
Sharma VK, Singh SK, Krishnamurthy PM, Alterman JF, Haraszti RA, Khvorova A, Prasad AK, Watts JK. (2017) Chemical Communications.
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5'-Vinylphosphate improves tissue accumulation and efficacy of conjugated siRNAs in vivo
Haraszti RA, Roux L, Coles AH, Turanov AA, Alterman JF, Echeverria D, Godinho BMDC, Aronin N, Khvorova A. (2017) Nucleic Acids Research.
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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 MF, Coles AH, Biscans A, Godinho BMDC, Haraszti RA, Sapp E, Echeverria D, DiFiglia M, Aronin N, Khvorova A. (2017) Bioconjugate Chemistry.  
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The chemical evolution of oligonucleotide therapies of clinical utility
Khvorova A, Watts JK. (2017) Nature Biotechnology.  
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Oligonucleotide Therapeutics - A New Class of Cholesterol-Lowering Drugs
Khvorova A. (2017) New England Journal of Medicine.
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Visualization of self-delivering hydrophobically modified siRNA cellular internalization
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. (2017) Nucleic Acids Research.
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Exosome-mediated Delivery of Hydrophobically Modified siRNA for Huntingtin mRNA Silencing
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. (2016) Molecular Therapy
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Docosahexaenoic Acid Conjugation Enhances Distribution and Safety of siRNA upon Local Administration in Mouse Brain
Nikan M, Osborn MF, Coles AH, Godinho BM, Hall LM, Haraszti RA, Hassler MR, Echeverria D, Aronin N, Khvorova A. (2016) Molecular Therapy - Nucleic Acids
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Hydrophobically Modified siRNAs Silence Huntingtin mRNA in Primary Neurons and Mouse Brain 
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. (2015) Molecular Therapy Nucleic Acids. 
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Exosomes are natural carriers of exogenous siRNA to human cells in vitro
Shtam TA, Kovalev RA, Varfolomeeva EY, Makarov EM, Kil YV, Filatov MV. (2013) Cell Communication and Signaling. 
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Novel hydrophobically modified asymmetric RNAi compounds (sd-rxRNA) demonstrate robust efficacy in the eye
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. (2013) Journal of Ocular Pharmacology and Therapeutics.
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Guanabenz (Wytensin™) selectively enhances uptake and efficacy of hydrophobically modified siRNAs 
Osborn MF, Alterman JF, Nikan M, Cao, H, Didiot MC, Hassler MR, Coles AH, Khvorova A. (2015) Nucleic Acids Research.
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A High-Throughput Method for Direct Detection of Therapeutic Oligonucleotide-Induced Gene Silencing In Vivo
Coles AH, Osborn MF, Alterman JF, Turanov AA, Godinho BM, Kennington L, Chase K, Aronin N, Khvorova A. (2016) Nucleic Acid Therapeutics.
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