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Yu M, Qin J, Liu X, Ramsden D, williams B, Zlatev I, Guenther D, Matsuda S, Tymon R, Darcy J, wong C, Tsung J, Zawaneh P, Chong S,Theile CS, Taneja N, Rogers A, Liu J, Castellanos-Rizaldos E, Bond S, So K, Denoncourt J, Castoreno A, Manoharan M, Wu JT, Fitzgerald K, Maier MA, Jadhav V, Nair JK

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PR1-002 2'-O-4'-C-Locked-A(Bz) phosphoramidite PR2-005 2'-O-4'-C-Locked-G(dmf) Phosphoramidite PR3-009 2'-O-4'-C-Locked-5-Me-C(Bz) Phosphoramidite PR4-001 2'-O-4'-C-Locked-T Phosphoramidite

Res Sq.

Jun 01, 2023

Extended Nucleic Acid (exNA): A Novel, Biologically Compatible Backbone that Significantly Enhances Oligonucleotide Efficacy in vivo

Hariharan VN, Caiazzi J, MillerR, Ferguson c, Sapp E, FakihH, Tang Q, Yamada N, FurgalR, 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

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PR5-001 DMT-2'-O-Me-U-CE Phosphoramidite

PLoS Genet.

Jan 13, 2011

Histone H4 Lysine 12 Acetylation Regulates Telomeric Heterochromatin Plasticity in Saccharomyces cerevisiae

Zhou BO, Wang SS, Zhang Y, Fu XH, Dang W, Lenzmeier BA, Zhou JQ

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D3100 dCMP·H2 D3-005 5-Me-dCMP·Na2

Nat Commun.

Jul 21, 2025

Identification of a key nucleotide influencing Cas12a crRNA activity for universal photo-controlled CRISPR diagnostics

Tian T, Xiao H, Guo X, Chen Y, Qiu Z, Zhang T, Chen M, Qi W, Cai P, Cheng M, Zhou X

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PR1-008 DMT-2'-O-TBDMS-A(Bz)-CE-Phosphoramidite PR2-007 DMT-2'-O-TBDMS-G(iBu)-CE-Phosphoramidite PR3-008 DMT-2'-O-TBDMS-C(Ac)-CE-Phosphoramidite PR5-003 DMT-2'-O-TBDMS-U-CE-Phosphoramidite

Mol Ther Nucleic Acids

Jul 31, 2024

Identification of selective and non-selective C9ORF72 targeting in vivo active siRNAs

Gilbert Jw, Kennedy Z, Godinho BMDC, Summers A, Weiss A, Echeverria D, Bramato B, McHugh N, Cooper D, Yamada K, Hassler M, Tran H, Gao FB, Brown RH Jr, Khvorova A

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PD1-001 DMT-2'-F-dA(Bz)-CE-Phosphoramidite PD2-001 DMT-2'-F-dG(dmf)-CE-Phosphoramidite PD2-002 DMT-2'-F-dG(iBu)-CE-Phosphoramidite PD3-001 DMT-2'-F-dC(Ac)-CE Phosphoramidite PD5-001 DMT-2'-F-dU-CE Phosphoramidite PR1-001 DMT-2'-O-Me-A(Bz)-CE Phosphoramidite PR2-001 DMT-2'-O-Me-G(dmf)-CE Phosphoramidite PR2-002 DMT-2'-O-Me-G(iBu)-CE Phosphoramidite PR3-001 DMT-2'-OMe-C(Ac)-CE-Phosphoramidite PR5-001 DMT-2'-O-Me-U-CE Phosphoramidite

Life Sci Alliance.

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Immunogenicity and effectiveness of an mRNA therapeutic vaccine for HPV-related malignancies

Wang J, Ma L, Chen Y, Zhou R, Wang Q, Zhang T, Yi D, Liu Q, Zhang Y, Zhang W, Dong Y, Cen S

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R5-027 N1-Me-pUTP, 100mM solution

Signal Transduct Target Ther.

Jul 17, 2023

Incorporation of a Toll-like receptor 2/6 agonist potentiates mRNA vaccines against cancer and infectious diseases

Gu Y, Yang J, He C, Zhao T, Lu R, Liu J, Mo X, Wen F, Shi H

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ON-124 BspQI

Sci Rep.

Mar 28, 2024

Intravenous administration of IL-12 encoding self-replicating RNA-lipid nanoparticle complex leads to safe and effective antitumor responses

Wang Z, Chen Y, Wu H, Wang M, Mao L, Guo X, Zhu J, Ye Z, Luo X, Yang X, Liu X, Yang J, Sheng Z, Lee J, Guo ZZ, Liu Y

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ON-062 10X IVT Reaction Buffer RN31 NTP 25mM Mix ON-025 Pyrophosphatase Inorganic (yeast) ON-039 RNase Inhibitor ON-004-C050 T7 RNA polymerase ON-028 Vaccinia Capping Enzyme ON-073 10X Capping Buffer ON-074 S-adenosylmethionine (SAM) ON-014 mRNA Cap-2'-O-Methyltransferase R2331 GTP 100mM solution

J Transl Med.

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Ligand-based adoptive T cell targeting CA125 in ovarian cancer

Zhao H, Wu L, Dai J, Sun K, ZiZ, Guan J, Zhang L

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ON-257 Capped RNA Synthesis Kit ON-269 Capped RNA Synthesis Kit (with tailing)

ACS Synth Biol.

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Low Resource Integrated Platform for Production and Analysis of Capped mRNA

Nwokeoji AO, Chou T, Nwokeoji EA

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ON-028 Vaccinia Capping Enzyme ON-073 10X Capping Buffer

PLoS Pathog.

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Mycobacterial MazG Safeguards Genetic Stability via Housecleaning of 5-OH-dCTP

Lyu LD, Tang BK, Fan XY, Ma H, Zhao GP

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R5-046 5-OMe-UTP 100mM solution

Nature Commuications

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Optimized inhaled LNP formulation for enhanced treatment of idiopathic pulmonary fibrosis via mRNA-mediated antibody therapy

Bai X, Chen Q, Li F, Teng Y, Tang M, Huang J, Xu X, Zhang XQ

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R5-022 pUTP, 100mM solution

bioRxiv

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Preventing acute neurotoxicity of CNS therapeutic oligonucleotides with the addition of Ca2+ and Mg2+ in the formulation

Miller R, Paquette J, Barker A, Sapp E, McHugh N, BramatoB, Yamada N, Alterman J, Echeveria D, Yamada k, Watts J, Anaclet C, DiFiglia M, Khvorova A, Aronin N

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PD1-001 DMT-2'-F-dA(Bz)-CE-Phosphoramidite PD2-001 DMT-2'-F-dG(dmf)-CE-Phosphoramidite PD2-002 DMT-2'-F-dG(iBu)-CE-Phosphoramidite PD3-001 DMT-2'-F-dC(Ac)-CE Phosphoramidite PD5-001 DMT-2'-F-dU-CE Phosphoramidite PR1-001 DMT-2'-O-Me-A(Bz)-CE Phosphoramidite PR2-001 DMT-2'-O-Me-G(dmf)-CE Phosphoramidite PR2-002 DMT-2'-O-Me-G(iBu)-CE Phosphoramidite PR3-001 DMT-2'-OMe-C(Ac)-CE-Phosphoramidite PR5-001 DMT-2'-O-Me-U-CE Phosphoramidite

Front Mol Biosci.

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Purification of linearized template plasmid DNA decreases double-stranded RNA formation during IVT reaction

Martinez J, Lampaya V, Larraga A, Magallón H, Casabona D

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R5-027 N1-Me-pUTP, 100mM solution

Front Microbiol.

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Quantification of T4-Like and T7-Like Cyanophages Using the Polony Method Show They Are Significant Members of the Virioplankton in the North Pacific Subtropical Gyre

Goldin S, Hulata Y, Baran N, Lindell D

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DN34 dNTP 2.5mM Mix

NPJ Vaccines

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Rational development of a combined mRNA vaccine against COVID-19 and influenza

Ye Q, Wu M, Zhou C, Lu X, Huang B, Zhang N, Zhao H, Chi H, Zhang X, Ling D, Zhang RR, Li Z, Luo D, Huang YI, Qiu HY, Song H, Tan W, Xu K, Ying B, Qin CF

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ON-028 Vaccinia Capping Enzyme ON-014 mRNA Cap-2'-O-Methyltransferase

Nat Commun.

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RNAi-mediated rheostat for dynamic control of AAV-delivered transgenes

Subramanian M, McIninch J, Zlatev I, Schlegel MK, Kaittanis C, Nguyen T, Agarwal S, Racie T, AlvaradoMA, Wassarman K, Collins TSChickering T, Brown CR, Schmidt K, Castoreno AB, Shulga-Morskaya S, Stamenova E, Buckowing K, Berman D, Barry JD, Bisbe A, Maier MA, Fitzgerald K, Jadhav V

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PR1-002 2'-O-4'-C-Locked-A(Bz) phosphoramidite PR2-005 2'-O-4'-C-Locked-G(dmf) Phosphoramidite PR2-003 2'-O-4'-C-Locked-G(iBu)-CE Phosphoramidite PR3-009 2'-O-4'-C-Locked-5-Me-C(Bz) Phosphoramidite PR5-006 5'-O-DMTr-2'-O-4'-C-Locked-U Phosphoramidite PR4-001 2'-O-4'-C-Locked-T Phosphoramidite D1-004 2'-F-dA D3-003 2'-F-dC R2-001 2'-OMe-Gr R5-002 2'-OMe-Ur

bioRxiv

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RNAi-mediated silencing of SOD1 profoundly extends survival and functional outcomes in ALS mice

Weiss A, Gilbert JW, Flores IVR, Belgrad J, Ferguson C, Dogan EO, Wightman N, Mocarski K, Echeverria D, Summers A, Bramato B, McHugh N, Furgal R, Yamada N, Cooper D, Monopoli K, Godinho BMDC, Hassler MR, Yamada K, Greer P, Henninger N, Brown RH Jr, Khvorova A

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ON-418 Cholesterol tetraethylene glycol (TetraEG) (plant source)

Nature Communications

Aug 27, 2024

RNA nanotherapeutics with fibrosis overexpression and retention for MASH treatment

Shan X, Zhao Z, Lai P, Liu Y, Li B, Ke Y, Jiang H, Zhou Y, Li W, Wang Q, Qin P, Xue Y, Zhang Z, Wei c, Ma B, Liu W, Luo C, Lu X, Lin J, Shu L, Jie Y Xian X, Delcassian D, Ge Y, Miao L

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ON-004-C050 T7 RNA polymerase R5-027 N1-Me-pUTP, 100mM solution

Nucleic Acids Res.

Nov 30, 2023

Self-delivering, chemically modified CRISPR RNAs for AAV co-delivery and genome editing in vivo

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

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ON-133 GalNAc-Cluster-COONa

Nucleic Acids Res.

Nov 25, 2021

Structurally constrained phosphonate internucleotide linkage impacts oligonucleotide-enzyme interaction, and modulates siRNA activity and allele specificity

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

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R5-003 DMT-2'-OMe-Ur D5-010 DMT-2'-F-dU

bioRxiv

Nov 04, 2024

Suppression of Huntington’s Disease Somatic Instability by Transcriptional Repression and Direct CAG Repeat Binding

Mathews EW, Coffey SR, Gärtner A, Belgrad J, Bragg RM, O'Reilly D, Cantle JP, McHugh C, Summers A, Fentz J, Schwagarus T, Cornelius A, Lingos I, Burch Z, KovalenkoM, Andrew MA, Frank Bennett C, Kordasiewicz HB, Marchionini DM, Wilkinson H, Vogt TF, Pinto RM, KhvorovaA, Howland D, wheeler VC, Carroll JB

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PD1-001 DMT-2'-F-dA(Bz)-CE-Phosphoramidite PD2-001 DMT-2'-F-dG(dmf)-CE-Phosphoramidite PD2-002 DMT-2'-F-dG(iBu)-CE-Phosphoramidite PD3-001 DMT-2'-F-dC(Ac)-CE Phosphoramidite PD5-001 DMT-2'-F-dU-CE Phosphoramidite PR1-001 DMT-2'-O-Me-A(Bz)-CE Phosphoramidite PR2-001 DMT-2'-O-Me-G(dmf)-CE Phosphoramidite PR2-002 DMT-2'-O-Me-G(iBu)-CE Phosphoramidite PR3-001 DMT-2'-OMe-C(Ac)-CE-Phosphoramidite PR5-001 DMT-2'-O-Me-U-CE Phosphoramidite

Ann Neurol.

Aug 13, 2021

Synergistic Deoxynucleoside and Gene Therapies for Thymidine Kinase 2 Deficiency

Lopez-Gomez C, Sanchez-Quintero MJ, Lee EJ, KleinerG, Tadesse S, Xie J, Akman HO, Gao G, Hirano M

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PD3-003 DMT-dC(Bz)-CE-Phosphoramidite PD3-007 DMT-dC(Ac)-CE-Phosphoramidite PD4-002 DMT-dT-CE-Phosphoramidite

Pharmaceutics

Nov 11, 2024

Towards Aptamer-Targeted Drug Delivery to Brain Tumors: The Synthesis of Ramified Conjugates of an EGFR-Specific Aptamer with MMAE on a Cathepsin B-Cleavable Linker

Brylev VA, Ryabukhina EV, Nazarova EV, Samoylenkova NS, Gulyak EL, Sapozhnikova KA, Dzarieva FM, Ustinov AV, Pronin IN, Usachev DY, Kopylov AM, Golovin AV, Pavlova GV, Ryazantsev DY, Korshun VA

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PD1-004 DMT-dA(Bz)-CE-Phosphoramidite PD2-004 DMT-dG(iBu)-CE-Phosphoramidite PD2-006 DMT-dG(dmf)-CE-Phosphoramidite PD3-003 DMT-dC(Bz)-CE-Phosphoramidite PD3-007 DMT-dC(Ac)-CE-Phosphoramidite PD4-002 DMT-dT-CE-Phosphoramidite

Scientific Publications

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