Industry Publications

RNAi Delivery – Self-delivering RNAi compounds
Cardia J, Samarsky D, Woolf T. (2010) Drug Delivery Technology.
   > View article

Peer-Reviewed Publications

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. 
   > Link to article

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.
   > Link to article

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.
   > Link to article

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.
   > Link to article

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.
   > Link to article

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.
   > Link to article

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.  
   > Link to article

The chemical evolution of oligonucleotide therapies of clinical utility
Khvorova A, Watts JK. (2017) Nature Biotechnology.  
   > Link to article

Oligonucleotide Therapeutics - A New Class of Cholesterol-Lowering Drugs
Khvorova A. (2017) New England Journal of Medicine.
   > Link to article

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.
   > Link to article

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
   > Link to article

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
   > Link to article

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. 
   > Link to article

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. 
   > Link to article

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.
   > Link to article

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.
   > Link to article

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.
   > Link to article