Scientific Publications

Explore research articles from leading scientific journals featuring our innovative products and technologies.

Nucleic Acids Res.

Aug 26, 2016

Evoking picomolar binding in RNA by a single phosphorodithioate linkage

Abeydeera ND, Egli M, Cox N, Mercier K, Conde JN, Pallan PS, Mizurini DM, Sierant M, Hibti FE, Hassell T, Wang T, Liu FW, Liu HM, Martinez C, Sood AK, Lybrand TP, Frydman C, Monteiro RQ, Gomer RH, Nawrot B, Yang X.
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Nat Commun.

Feb 19, 2018

Selection of GalNAc-conjugated siRNAs with limited off-target-driven rat hepatotoxicity

Janas MM, Schlegel MK, Harbison CE, Yilmaz VO, Jiang Y, Parmar R, Zlatev I, Castoreno A, Xu H, Shulga-Morskaya S, Rajeev KG, Manoharan M, Keirstead ND, Maier MA, Jadhav V.
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Nucleic Acids Res.

Apr 19, 2018

Crystal structures of thrombin in complex with chemically modified thrombin DNA aptamers reveal the origins of enhanced affinity

Dolot R, Lam CH, Sierant M, Zhao Q, Liu FW, Nawrot B, Egli M, Yang X.
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J Thromb Haemost.

Mar 05, 2020

The development and characterization of a long acting anti‐thrombotic von Willebrand factor (VWF) aptamer

Zhu S, Gilbert JC, Hatala P, Harvey W, Liang Z, Gao S, Kang D, Jilma B.
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Nucleic Acids Res.

Jul 15, 2021

Chirality matters: stereo-defined phosphorothioate linkages at the termini of small interfering RNAs improve pharmacology in vivo

Jahns H, Taneja N, Willoughby JLS, Akabane-Nakata M, Brown CR, Nguyen T, Bisbe A, Matsuda S, Hettinger M, Manoharan RM, Rajeev KG, Maier MA, Zlatev I, Charisse K, Egli M, Manoharan M.
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Nucleic Acids Res.

Sep 11, 2021

Small circular interfering RNAs (sciRNAs) as a potent therapeutic platform for gene-silencing

Jahns H, Taneja N, Willoughby JLS, Akabane-Nakata M, Brown CR, Nguyen T, Bisbe A, Matsuda S, Hettinger M, Manoharan RM, Rajeev KG, Maier MA, Zlatev I, Charisse K, Egli M, Manoharan M.
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Biochemistry.

Apr 18, 2022

Phosphonoacetate Modifications Enhance the Stability and Editing Yields of Guide RNAs for Cas9 Editors

Ryan DE, Diamant-Levi T, Steinfeld I, Taussig D, Visal-Shah S, Thakker S, Lunstad BD, Kaiser RJ, McCaffrey R, Ortiz M, Townsend J, Welch WRW, Singh M, Curry B, Dellinger DJ, Bruhn L.
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Ann Neurol.

Oct 03, 2022

Chemical engineering of therapeutic siRNAs for allele-specific gene silencing in Huntington's disease models

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

Oct 19, 2023

Two Birds with One Stone: A Novel Dithiomaleimide-Based GalNAc-siRNA Conjugate Enabling Good siRNA Delivery and Traceability

Kong S, Gao X, Wang Q, Lin J, Qiu L, Xie M.
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Nucleic Acids Res.

Nov 16, 2023

Synthesis of peptide–siRNA conjugates via internal sulfonylphosphoramidate modifications and evaluation of their in vitro activity

Smidt JM, Lykke L, Stidsen CE, Pristovšek N, Gothelf KV.
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Mol Ther Nucleic Acids

Dec 05, 2023

Single intravitreal administration of a tetravalent siRNA exhibits robust and efficient gene silencing in mouse and pig photoreceptors

Cheng SY, Caiazzi J, Biscans A, Alterman JF, Echeverria D, McHugh N, Hassler M, Jolly S, Giguere D, Cipi J, Khvorova A, Punzo C.
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Comput Struct Biotechnol J.

Jul 30, 2024

A statistical-physics approach for codon usage optimisation

Luna-Cerralbo D, Blasco-Machin I, Adame-Pérez S, Lampaya V, Larraga A, AlejoT, Martinez-Oliván J, Broset E, Bruscolini P.
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Brief Bioinform

Oct 24, 2024

AntigenBoost: enhanced mRNA-based antigen expression through rational amino acid substitution

Gao Y, Zhu S, Li H, Hao X, Chen W, Pan D, Qian Z
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Int J Mol Sci.

Oct 01, 2024

Comprehensive Optimization of a Freeze-Drying Process Achieving Enhanced Long-Term Stability and In Vivo Performance of Lyophilized mRNA-LNPs

Alejo T, Toro-Córdova A, Fernández L, Rivero A, StoianAM, Pérez L, Navarro V, Martinez-Oliván J, de Miguel
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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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Nature Commuications

Aug 10, 2024

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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J Transl Med.

Mar 13, 2024

CD19/CD20 dual-targeted chimeric antigen receptor-engineered natural killer cells exhibit improved cytotoxicity against acute lymphoblastic leukemia

Yang N, Zhang C, Zhang Y, Fan Y, Zhang J, Lin X, Guo T, Gu Y, Wu J, Gao J, Zhao X, He Z
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Life Sci Alliance.

Mar 21, 2024

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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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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bioRxiv

Jun 08, 2024

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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PLoS Pathog.

Apr 01, 2024

Broad-spectrum Delta-BA.2 tandem-fused heterodimer mRNA vaccine delivered by lipopolyplex

Du P, Huang L, Fang Y, Zhao F, Li Q, Ma X, Li R, Chen Q, Shen H, Wang Q, Li H, Gao GF
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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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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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Nature

May 02, 2023

Algorithm for optimized mRNA design improves stability and immunogenicity

Zhang H, Zhang L, Lin A, Xu C, Li Z, Liu K, Liu B, Ma X, Zhao F, Jiang H, Chen C, Shen H, Li H, Mathews DH, ZhangY, Huang L
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NPJ Vaccines

Jul 26, 2022

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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Nat Biotechnol.

Nov 10, 2022

An engineered T7 RNA polymerase that produces mRNA free of immunostimulatory byproducts

Dousis A, Ravichandran K, Hobert EM, Moore MJ, Rabideau AE
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ACS Synth Biol.

Dec 10, 2022

Low Resource Integrated Platform for Production and Analysis of Capped mRNA

Nwokeoji AO, Chou T, Nwokeoji EA
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Nat Commun.

Apr 08, 2023

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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Pharmaceutics

Apr 20, 2023

Multicomponent Lipid Nanoparticles for RNA Transfection

Gretskaya N, Akimov M, Andreev D, Zalygin A, Belitskaya E, Zinchenko GFomina-Ageeva E, MikhalyovI, Vodovozova E, Bezuglov V
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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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J Transl Med.

Sep 05, 2023

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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Front Mol Biosci.

Sep 29, 2023

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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Front Immunol.

Nov 26, 2023

An mRNA vaccine against rabies provides strong and durable protection in mice

Li M, Fang E, Wang Y, Shi L, Li J, Peng Q, Li X, Zhao D, Liu X, Liu X, Liu J, Xu H, Wang H, Huang Y, Yang R, Yue G, Suo Y, Wu X, Cao S, Li Y
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Nucleic Acids Res.

May 14, 2024

Evaluating the oral delivery of GalNAc-conjugated siRNAs in rodents and non-human primates

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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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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Front Cell Infect Microbiol.

Aug 08, 2024

An accurate and convenient method for Mycoplasma pneumoniae via one-step LAMP-CRISPR/Cas12b detection platform

Liu T, Liu Q, Chen F, Shi Y, Maimaiti G, Yang Z, Zheng S, Lu X, Li H, Chen Z
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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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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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Mol Ther Nucleic Acids

May 24, 2025

Albumin-binding dendritic siRNA improves delivery and efficacy to solid tumors in a melanoma model

Fakih HH, Tang Q, Summers A, Gross KY, Rachid MO, Okamura K, Martinez N,Sleiman HF, Harris JE, Khvorova A
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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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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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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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Front Microbiol.

Jun 16, 2020

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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eBioMedicine

Aug 02, 2019

Bioavailability and cytosolic kinases modulate response to deoxynucleoside therapy in TK2 deficiency

Lopez-Gomez C, Hewan H, Sierra C, Akman HO, Sanchez-Quintero MJ, Juanola-Falgarona M, Tadesse S, Tanji K, Konofagou EE, Hirano M
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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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PLoS Pathog.

Dec 05, 2013

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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EMBO Mol Med.

Jun 26, 2014

Deoxypyrimidine monophosphate bypass therapy for thymidine kinase 2 deficiency

Garone C, Garcia-Diaz B, Emmanuele V, Lopez LC, Tadesse S, Akman HO, Tanji K, Quinzii CM, Hirano M
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Protein Cell

Jul 14, 2016

An unusual UMP C-5 methylase in nucleoside antibiotic polyoxin biosynthesis

Chen W, Li Y, Li J, Wu L, Li Y, Wang R, Deng Z, Zhou J
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Appl Environ Microbiol.

May 01, 2017

Biosynthesis of 2′-Chloropentostatin and 2′-Amino-2′-Deoxyadenosine Highlights a Single Gene Cluster Responsible for Two Independent Pathways in Actinomadura sp. Strain ATCC 39365

Gao Y, Xu G, Wu P, Liu J, Cai YS, Deng Z, Chen W
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Ann Neurol.

May 04, 2017

Deoxycytidine and deoxythymidine treatment for thymidine kinase 2 deficiency

Lopez-Gomez C, Levy RJ, Sanchez-Quintero MJ, Juanola-Falgarona M, Barca E, Garcia-Diaz B, Tadesse S,Garone C, Hirano M
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bioRxiv

Jun 25, 2024

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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Nat Commun.

Jul 14, 2025

Boosting RNA nanotherapeutics with V-ATPase activating non-inflammatory lipid nanoparticles to treat chronic lung injury

Zhao Z, Shan X, Ding J, Ma B, Li B, Huang W, Yang Q, Fang Y, Chen J, Song C, Wei C, Liu S, Cheng X, Zhang S, Liu Y, Wu H, Luo C, Shu S, Qiao X, Wang Z, Lu X, Miao L.
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Cell Rep Med.

Jul 01, 2025

Cardiolipin-mimic lipid nanoparticles without antibody modification delivered senolytic in vivo CAR-T therapy for inflamm-aging

Zhang Z, Ma B, Li B, Li Z, Gao M, Zhao H, Peng R, Hu J, Wang Y, You W, Gui X, Wang R, Hu X, Chen B, Zhang Y, Hao Y, Sun X, Rao P, Zhang L, Lu M, Zhou D, Yang Y, Deng M, Miao L.
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Mol Ther Methods Clin Dev.

Jun 16, 2025

A systemically deliverable lipid-conjugated siRNA targeting DUX4 as an facioscapulohumeral muscular dystrophy therapeutic

Daman K, Yan J, Biscans A, Echeverria D, Shmushkovich T, Wolfson A, Alterman JF, Khvorova A, Emerson CP Jr.
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Vaccines (Basel).

Jun 11, 2025

Development of an Influenza/COVID-19 Combination mRNA Vaccine Containing a Novel Multivalent Antigen Design That Enhances Immunogenicity of Influenza Virus B Hemagglutinins

Thornhill-Wadolowski E, Ruter DL, Yan F, Gajera M, Kurt E, Samanta L, Leigh K, Zhu J, Guo Z, Wang Z, Liu Y, Lee J, Bugno M.
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Front Cell Infect Microbiol.

May 29, 2025

Innovative nucleic acid detection of Clostridioides difficile utilizing the PAM-unconventional, one-step LAMP/CRISPR-Cas12b detection platforms

Zhang Y, Lv L, Xu S, Chen Y.
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Adv Sci (Weinh).

Apr 25, 2025

Novel Bivalent mRNA‐LNP Vaccine for Highly Effective Protection against Pneumonic Plague

Elia U, Levy Y, Cohen H, Zauberman A, Gur D, Hazan-Halevy I, Aftalion M, Benarroch S, Bar-Haim E, Redy-Keisar O, Cohen O, Peer D, Mamroud E.
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Nucleic Acids Res.

May 10, 2024

A programmable dual-targeting siRNA scaffold supports potent two-gene modulation in the central nervous system

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.
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Hypertension.

May 01, 2024

Counteracting Angiotensinogen Small-Interfering RNA-Mediated Antihypertensive Effects With REVERSIR

Ye D, Cruz-López EO, Veghel RV, Garrelds IM, Kasper A, Wassarman K, Tu HC, Zlatev I, Danser AHJ.
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bioRxiv

Apr 28, 2024

mRNA nuclear clustering leads to a difference in mutant huntingtin mRNA and protein silencing by siRNAs in vivo

Allen S, O'Reilly D, Miller R, Sapp E, Summers A, Paquette J, Moreno DE, Bramato B, McHugh N, Yamada K, Aronin N, DiFiglia M, Khvorova A.
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eLife.

Apr 02, 2024

Lactate transporter MCT1 in hepatic stellate cells promotes fibrotic collagen expression in nonalcoholic steatohepatitis

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.
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