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Oligos in Action

2024 

INT-1B3, an LNP formulated miR-193a-3p mimic, promotes anti-tumor immunity by enhancing T cell mediated immune responses via modulation of the tumor microenvironment and induction of immunogenic cell death

Duurland, C. L., de Gunst, T., den Boer, H. C., van den Bosch, M. T. J., Telford, B. J., Vos, R. M., Xie, X., Zang, M., Wang, F., Shao, Y., An, X., Wang, J., Cai, J., Bourré, L., van Pinxteren, L. A. H., Schaapveld, R. Q. J., Janicot, M., Yahyanejad, S.

Oncotarget (2024)

 

Role of the soluble epoxide hydrolase in keratinocyte proliferation and sensitivity of skin to inflammatory stimuli

Naeem, Z., Zukunft, S., Huard, A., Hu, J., Hammock, B. D., Weigert, A., Frömel, T., Fleming, I.

Biomedicine & Pharmacotherapy Vol. 171 (2024)

 

Profiling the interactome of oligonucleotide drugs by proximity biotinylation

Hanswillemenke, A., Hofacker, D. T., Sorgenfrei, M., Fruhner, C., Franz-Wachtel, M., Schwarzer, D., Maček, B., Stafforst, T.

Nat Chem Biol (2024) 

 

A pipeline for identifying guide RNA sequences that promote RNA editing of nonsense mutations that cause inherited retinal diseases

Schneider, N., Steinberg, R., Ben-David, A., Valensi, J., David-Kadoch, G., Rosenwasser, Z., Banin, E., Levanon, E. Y., Sharon, D., Ben-Aroya, S.

Molecular Therapy - Nucleic Acids (2024)

 

GNTI-122: an autologous antigen-specific engineered Treg cell therapy for type 1 diabetes

Uenishi, G. I., Repic, M., Yam, J. Y., Landuyt, A., Saikumar-Lakshmi, P., Guo, T., Zarin, P., Sassone-Corsi, M., Chicoine, A., Kellogg, H., Hunt, M., Drow, T., Tewari, R., Cook, P. J., Yang, S. J., Cerosaletti, K., Schweinoch, D., Guiastrennec, B., James, E., Patel, C., Chen, T. F., Buckner, J. H., Rawlings, D. J., Wickham, T. J., Mueller, K. T.

JCI Insight (2024)

 

Add-on multiple submucosal injections of the RNA oligonucleotide GUT-1 to anti-TNF antibody treatment in patients with moderate-to-severe ulcerative colitis: an open-label, proof-of concept study

Suzuki, K., Sameshima, Y., Yokoyama, J., Terai, S., Yoneyama, H., Atreya, R., Neurath, M. F., Hibi, T., Asakura, H.

Inflamm Regener (2024)

 

 

2023

The tumor suppressor 5A2, a synthetic miR-7-5p mimic, targets oncogenic and metabolic pathways, as revealed by transcriptome-wide analysis

van den Bosch, M. T. J., Telford, B. J., Yahyanejad, S., de Gunst, T., den Boer, H. C., Vos, R. M., Duurland, C. L., Biemans, R., Dubois, L. J., van Pinxteren, L. A. H., Schaapveld, R. Q. J., Janicot, M.

Frontiers in Drug Discovery Vol. 3 (2023) 

 

Cationic LNP-formulated mRNA expressing Tie2-agonist in the lung endothelium prevents pulmonary vascular leakage

Radloff, K., Gutbier, B., Dunne, C. M., Moradian, H., Schwestka, M., Gossen, M., Ahrens, K., Kneller, L., Wang, Y., Moga, A., Gkionis, L., Keil, O., Fehring, V., Tondera, D., Giese, K., Santel, A., Kaufmann, J., Witzenrath, M.

Molecular Therapy: Nucleic Acids Vol. 34 (2023)

 

Targeted siRNA lipid nanoparticles for the treatment of KRAS-mutant tumors

Anthiya, S., Öztürk, S.C., Yanik, H., Tavukcuoglu, E., Şahin, A., Datta, D., Charisse, K., Álvarez, D.M., Loza, M.I., Calvo, A., & Sulheim, E.

Journal of Controlled Release (2023)

 

 ADAR1-mediated RNA editing promotes B cell lymphomagenesis

Pecori, R., Ren, W., Pirmoradian, M., Wang, X., Liu, D., Berglund, M., Li, W., Tasakis, R. N., Di Giorgio, S., Ye, X., Li, X., Arnold, A., Wüst, S., Schneider, M., Selvasaravanan, K.-D., Fuell, Y., Stafforst, T., Amini, R.-M., Sonnevi, K., Enblad, G., Sander, B., Wahlin, B. E., Wu, K., Zhang, H., Helm, D., Binder, M., Papavasiliou, F. N., Pan-Hammarström, Q.

iScience Volume 26 (2023)

 

Combination of protein and cell internalization SELEX identifies a potential RNA therapeutic and delivery platform to treat EphA2-expressing tumors

Santana-Viera, L., Dassie, J. P., Rosàs-Lapeña, M., Garcia-Monclús, S., Chicón-Bosch, M., Pérez-Capó, M., del Pozo, L., Sanchez-Serra, S., Almacellas-Rabaiget, O., Maqueda-Marcos, S., López-Alemany, R., Thiel, W. H., Giangrande, P. H., Tirado, O. M.

ACS Nano (2023)

 

Positron Emission Tomography Studies of the Biodistribution, Translocation, and Fate of Poly Allyl Amine-Based Carriers for siRNA Delivery by Systemic and Intratumoral Administration

Simo, C., Salvador, C., Andreozzi, P., Gomez-Vallejo, V., Romero, G., Dupin, D., Llop, J., Moya, S. E.

Small (2023)

 

Spacer Fidelity Assessments of Guide RNA by Top-Down Mass Spectrometry

Macias, L. A., Garcia, S. P., Back, K. M., Wu, Y., Johnson, G. H., Kathiresan, S., Bellinger, A. M., Rohde, E., Freitas, M. A., Madsen, J. A.

ACS Cent. Sci. (2023)

 

An Aptamer against MNK1 for Non-Small Cell Lung Cancer Treatment

Carrión-Marchante, R., Pinto-Díez, C., Klett-Mingo, J. I., Palacios, E., Barragán-Usero, M., Pérez-Morgado, M. I., Pascual-Mellado, M., Alcalá, S., Ruiz-Cañas, L., Sainz, B., Jr., González, V. M., Martín, M. E.

Pharmaceutics (2023)

 

Correlating the Structure and Gene Silencing Activity of Oligonucleotide-Loaded Lipid Nanoparticles Using Small-Angle X-ray Scattering

Hammel, M., Fan, Y., Sarode, A., Byrnes, A. E., Zang, N., Kou, P., Nagapudi, K., Leung, D., Hoogenraad, C. C., Chen, T., Yen, C.-W., Hura, G. L.

ACS Nano (2023)

2022

Herpes simplex virus PCR in 2230 explanted corneal buttons

Tóth, G., Berkó-Göttel, B., Seitz, B., Langenbucher, A., Stachon, T., Pluzsik, M.T., Nagy, Z.Z., Smola, S., & Szentmáry, N.

Acta Ophthalmologica (2022)

 

Selective Targeting and Eradication of Various Human Non-Small Cell Lung Cancer Cell Lines Using Self-Assembled Aptamer-Decorated Nanoparticles

Barak, D., Engelberg, S., Assaraf, Y. G., Livney, Y. D.

Pharmaceutics (2022)

 

Targeting TGF-ß in the Central Nervous System: Assessment of Cynomolgus Monkey—Toxicity and Pharmacokinetics for an LNA-Antisense Oligonucleotide

Peters, S., Wirkert, E., Kuespert, S., Heydn, R., Korte, S., Mecklenburg, L., Aigner, L., Johannesen, S., Bruun, T.-H., Bogdahn, U.

Appl. Sci. (2022)

 

Predictive high-throughput screening of PEGylated lipids in oligonucleotide-loaded lipid nanoparticles for neuronal gene silencing

Sarode, A., Fan, Y., Byrnes, A. E., Hammel, M., Hura, G. L., Fu, Y., Kou, P., Hu, C., Hinz, F. I., Roberts, J., Koenig, S. G., Nagapudi, K., Hoogenraad, C. C., Chen, T., Leung, D., Yen, C.-W.

Nanoscale Adv. (2022)

 

Enhancement of protective efficacy of innate immunostimulant based formulations against yolk sac infection in young chicks

Nguyen, T. T. T., Shahin, K., Allan, B., Sarfraz, M., Wheler, C., Gerdts, V., Köster, W., Dar, A.

Poultry Science (2022)

 

Functional Downregulation of PD-L1 and PD-L2 by CpG and non-CpG Oligonucleotides in Melanoma Cells

Kleemann, J., Steinhorst, K., König, V., Zöller, N., Cinatl, J., Jr., Özistanbullu, D., Kaufmann, R., Meissner, M., Kippenberger, S.

Cancers (2022)

 

SAMHD1 controls innate immunity by regulating condensation of immunogenic self RNA

Maharana, S., Kretschmer, S., Hunger, S., Yan, X., Kuster, D., Traikov, S., Zillinger, T., Gentzel, M., Elangovan, S., Dasgupta, P., Chappidi, N., Lucas, N., Maser, K. I., Maatz, H., Rapp, A., Marchand, V., Chang, Y.-T., Motorin, Y., Hubner, N., Hartmann, G., Hyman, A. A., Alberti, S., Lee-Kirsch, M. A.

Molecular Cell (2022)

 

Optimized Hemolysin Type 1 Secretion System in Escherichia coli by Directed Evolution of the Hly Enhancer Fragment and Including a Terminator Region

Pourhassan N., Z., Cui, H., Khosa, S., Davari, M. D., Jaeger, K.-E., Smits, S. H. J., Schwaneberg, U., Schmitt, L.

ChemBioChem (2022)

 

In vivo base editing rescues cone photoreceptors in a mouse model of early-onset inherited retinal degeneration

Choi, E. H., Suh, S., Foik, A. T., Leinonen, H., Newby, G. A., Gao, X. D., Banskota, S., Hoang, T., Du, S. W., Dong, Z., Raguram, A., Kohli, S., Blackshaw, S., Lyon, D. C., Liu, D. R., Palczewski, K.

Nat Commun (2022)

 

RNA-Cholesterol Nanoparticles Function as Potent Immune Activators via TLR7 and TLR8

Obermann, H.-L., Lederbogen, I. I., Steele, J., Dorna, J., Sander, L. E., Engelhardt, K., Bakowsky, U., Kaufmann, A., Bauer, S.

Front. Immunol. (2022)

 

 

2021

Base editing of hematopoietic stem cells rescues sickle cell disease in mice

Newby, G. A., Yen, J. S., Woodard, K. J., Mayuranathan, T., Lazzarotto, C. R., Li, Y., Sheppard-Tillman, H., Porter, S. N., Yao, Y., Mayberry, K., Everette, K. A., Jang, Y., Podracky, C. J., Thaman, E., Lechauve, C., Sharma, A., Henderson, J. M., Richter, M. F., Zhao, K. T., Miller, S. M., Wang, T., Koblan, L. W., McCaffrey, A. P., Tisdale, J. F., Kalfa, T. A., Pruett-Miller, S. M., Tsai, S. Q., Weiss, M. J., Liu, D. R.

Nature (2021)

 

Aptamer BC 007’s affinity to specific and less-specific anti-Sars-CoV-2 neutralizing antibodies

Haberland, A., Krylova, O., Nikolenko, H., Göttel, P., Dallmann, A., Müller, J., & Weisshoff, H.

Viruses (2021)

 

The consequences of soluble epoxide hydrolase deletion on tumorigenesis and metastasis in a mouse model of breast cancer

Kesavan, R., Frömel, T., Zukunft, S., Brüne, B., Weigert, A., Wittig, I., Popp, R., & Fleming, I.

International Journal of Molecular Sciences (2021)

 

Regulation of GC box activity by 8-oxoguanine.

Müller, N. & Khobta, A.

Redox Biology (2021)

 

Targeted Nanoparticles Harboring Jasmine-Oil-Entrapped Paclitaxel for Elimination of Lung Cancer Cells

Engelberg, S., Lin, Y., Assaraf, Y. G., Livney, Y. D.

Int. J. Mol. Sci. (2021)

 

Overcoming PD-1 Blockade Resistance with CpG-A Toll-Like Receptor 9 Agonist Vidutolimod in Patients with Metastatic Melanoma

Ribas, A., Medina, T., Kirkwood, J. M., Zakharia, Y., Gonzalez, R., Davar, D., Chmielowski, B., Campbell, K. M., Bao, R., Kelley, H., Morris, A., Mauro, D., Wooldridge, J. E., Luke, J. J., Weiner, G. J., Krieg, A. M., Milhem, M. M.

Cancer Discov (2021)

 

Multi-modal effects of 1B3, a novel synthetic miR-193a-3p mimic, support strong potential for therapeutic intervention in oncology

Telford, B. J., Yahyanejad, S., de Gunst, T., den Boer, H. C., Vos, R. M., Stegink, M., van den Bosch, M. T. J., Alemdehy, M. F., van Pinxteren, L. A. H., Schaapveld, R. Q. J., Janicot, M.

Oncotarget. (2021)

 

Transcriptome-wide analysis reveals insight into tumor suppressor functions of 1B3, a novel synthetic miR-193a-3p mimic

van den Bosch, M. T. J., Yahyanejad, S., Alemdehy, M. F., Telford, B. J., de Gunst, T., den Boer, H. C., Vos, R. M., Stegink, M., van Pinxteren, L. A. H., Schaapveld, R. Q. J., Janicot, M.

Molecular Therapy: Nucleic Acids (2021)

 

CroSR391, an ortholog of the λ Cro repressor, plays a major role in suppressing polVR391-dependent mutagenesis

McDonald, J. P., Quiros, D. R., Vaisman, A., Mendez, A. R., Reyelt, J., Schmidt, M., Gonzalez, M., Woodgate, R.

Molecular Microbiology (2021)

 

Direct and Base Excision Repair-Mediated Regulation of a GC-Rich cis-Element in Response to 5-Formylcytosine and 5-Carboxycytosine

Müller, N., Ponkkonen, E., Carell, T., Khobta, A.

Int. J. Mol. Sci. (2021)

 

Induced dendritic cells co-expressing GM-CSF/IFN-α/tWT1 priming T and B cells and automated manufacturing to boost GvL

Bialek-Waldmann, J. K., Domning, S., Esser, R., Glienke, W., Mertens, M., Aleksandrova, K., Arseniev, L., Kumar, S., Schneider, A., Koenig, J., Theobald, S. J., Tsay, H.-C., Cornelius, A. D. A., Bonifacius, A., Eiz-Vesper, B., Figueiredo, C., Schaudien, D., Talbot, S. R., Bleich, A., Spineli, L. M., von Kaisenberg, C., Clark, C., Blasczyk, R., Heuser, M., Ganser, A., Köhl, U., Farzaneh, F., Stripecke, R.

Molecular Therapy: Methods & Clinical Development (2021)

 

Sulfonated Amphiphilic Poly(α)glutamate Amine—A Potential siRNA Nanocarrier for the Treatment of Both Chemo-Sensitive and Chemo-Resistant Glioblastoma Tumors

Krivitsky, A., Pozzi, S., Yeini, E., Israeli Dangoor, S., Zur, T., Golan, S., Krivitsky, V., Albeck, N., Pisarevsky, E., Ofek, P., Madi, A., Satchi-Fainaro, R.

Pharmaceutics (2021)

 

Embryonic stem cells are devoid of macropinocytosis, a trafficking pathway for activin A in differentiated cells

Kostopoulou, N., Bellou, S., Bagli, E., Markou, M., Kostaras, E., Hyvönen, M., Kalaidzidis, Y., Papadopoulos, A., Chalmantzi, V., Kyrkou, A., Panopoulou, E., Fotsis, T., Murphy, C.

J Cell Sci (2021)

 

Endothelial Tpl2 regulates vascular barrier function via JNK-mediated degradation of claudin-5 promoting neuroinflammation or tumor metastasis

Nanou, A., Bourbouli, M., Vetrano, S., Schaeper, U., Ley, S., Kollias, G.

Cell Reports (2021)

2020

A Bovine Enteric Mycobacterium Infection Model to Analyze Parenteral Vaccine-Induced Mucosal Immunity and Accelerate Vaccine Discovery

Facciuolo, A., Lee, A.H., Trimble, M.J., Rawlyk, N., Townsend, H.G., Bains, M., Arsic, N., Mutharia, L.M., Potter, A., Gerdts, V., & Napper, S.

Frontiers in Immunology (2020)

 

Antisense oligonucleotide in LNA-gapmer design targeting TGFBR2—a key single gene target for safe and effective inhibition of TGFβ signaling

Kuespert, S., Heydn, R., Peters, S., Wirkert, E., Meyer, A.L., Siebörger, M., Johannesen, S., Aigner, L., Bogdahn, U. and Bruun, T.H.

International Journal of Molecular Sciences (2020)

 

A three-part, randomised study to investigate the safety, tolerability, pharmacokinetics and mode of action of BC 007, neutraliser of pathogenic autoantibodies against G-protein coupled receptors in healthy, young and elderly subjects.

Becker, N.P., Haberland, A., Wenzel, K., Göttel, P., Wallukat, G., Davideit, H., Schulze-Rothe, S., Hönicke, A.S., Schimke, I., Bartel, S. & Grossmann, M.

Clinical Drug Investigation (2020)

 

EGFP reporters for direct and sensitive detection of mutagenic bypass of DNA lesions

Rodriguez-Alvarez, M., Kim, D., & Khobta, A.

Biomolecules (2020)

 

Macrophages produce and functionally respond to interleukin-34 in colon cancer

Franzè, E., Laudisi, F., Di Grazia, A., Marônek, M., Bellato, V., Sica, G., Monteleone, G.

Cell Death Discov. (2020)

 

Radiolabeling and PET–MRI microdosing of the experimental cancer therapeutic, MN-anti-miR10b, demonstrates delivery to metastatic lesions in a murine model of metastatic breast cancer

Le Fur, M., Ross, A., Pantazopoulos, P., Rotile, N., Zhou, I., Caravan, P., Medarova, Z., Yoo, B.

Cancer Nano (2021)

 

Aptamer BC 007 - Efficient binder of spreading-crucial SARS-CoV-2 proteins

Weisshoff, H., Krylova, O., Nikolenko, H., Düngen, H.-D., Dallmann, A., Becker, S., Göttel, P., Müller, J., Haberland, A.

Heliyon (2020)

 

Dose-Finding Study and Pharmacokinetics Profile of the Novel 13-Mer Antisense miR-221 Inhibitor in Sprague-Dawley Rats

Di Martino, M. T., Arbitrio, M., Caracciolo, D., Scionti, F., Tagliaferri, P., Tassone, P.

Molecular Therapy: Nucleic Acids (2020)

 

MRI-based molecular imaging of epicardium-derived stromal cells (EpiSC) by peptide-mediated active targeting

Straub, T., Nave, J., Bouvain, P., Akbarzadeh, M., Dasa, S. S. K., Kistner, J., Ding, Z., Marzoq, A., Stepanow, S., Becker, K., Hesse, J., Köhrer, K., Flögel, U., Ahmadian, M. R., French, B. A., Schrader, J., Temme, S.

Sci Rep (2020)

 

RNA and DNA Binding Epitopes of the Cold Shock Protein TmCsp from the Hyperthermophile Thermotoga maritima

von König, K., Kachel, N., Kalbitzer, H. R., Kremer, W.

Protein J (2020)

 

 

2019

Allometric scaling approaches for predicting human pharmacokinetic of a locked nucleic acid oligonucleotide targeting cancer-associated miR-221

Di Martino, M.T., Arbitrio, M., Fonsi, M., Erratico, C.A., Scionti, F., Caracciolo, D., Tagliaferri, P. & Tassone, P.

Cancers (2019)

 

Effects of siRNA-mediated knockdown of GSK3β on retinal ganglion cell survival and neurite/axon growth

Ahmed, Z., Morgan-Warren, P.J., Berry, M., Scott, R.A.H., & Logan, A.

Cells (2019)

 

Comparison of PEGylated and non-PEGylated proticles: An in vitro and in vivo study

Fresacher, K., Helbok, A., Reiser, M., Blass, S., Rangger, C., Mair, C., von Guggenberg, E., Decristoforo, C., Andreae, F., & Zimmer, A.

European Journal of Pharmaceutical Sciences (2019)

 

Nucleotide excision repair of abasic DNA lesions

Kitsera, N., Rodriguez-Alvarez, M., Emmert, S., Carell, T., & Khobta, A.

Nucleic Acids Research (2019)

 

Effect of an intravitreal antisense oligonucleotide on vision in Leber congenital amaurosis due to a photoreceptor cilium defect

Cideciyan, A.V., Jacobson, S.G., Drack, A.V., Ho, A.C., Charng, J., Garafalo, A.V., Roman, A.J., Sumaroka, A., Han, I.C., Hochstedler, M.D. & Pfeifer, W.L.

Nature Medicine (2019)

 

Selective eradication of human non-small cell lung cancer cells using aptamer-decorated nanoparticles harboring a cytotoxic drug cargo

Engelberg, S., Netzer, E., Assaraf, Y. G., Livney, Y. D.

Cell Death Discov. (2019)

 

EnanDIM - a novel family of L-nucleotide-protected TLR9 agonists for cancer immunotherapy

Kapp, K., Volz, B., Curran, M. A., Oswald, D., Wittig, B., Schmidt, M.

j. immunotherapy cancer (2019)

 

Macrophages Are a Potent Source of Streptococcus-Induced IFN-β

Feuerstein, R., Gres, V., Elias Perdigó, N., Baasch, S., Freudenhammer, M., Elling, R., Henneke, P.

J Immunol (2019)

 

Evaluation of eluforsen, a novel RNA oligonucleotide for restoration of CFTR function in in vitro and murine models of p.Phe508del cystic fibrosis

Beumer, W., Swildens, J., Leal, T., Noel, S., Anthonijsz, H., van der Horst, G., Kuiperij-Boersma, H., Potman, M., van Putten, C., Biasutto, P., Platenburg, G., de Jonge, H., Henig, N., Ritsema, T.

PLOS ONE (2019)

 

Enzyme-free ligation of dimers and trimers to RNA primers

Sosson, M., Pfeffer, D., Richert, C.

Nucleic Acids Research (2019)

 

Epigenetic control of the angiotensin-converting enzyme in endothelial cells during inflammation

Mudersbach, T., Siuda, D., Kohlstedt, K., Fleming, I.

PLOS ONE (2019)

 

UGCG influences glutamine metabolism of breast cancer cells

Schömel, N., Hancock, S. E., Gruber, L., Olzomer, E. M., Byrne, F. L., Shah, D., Hoehn, K. L., Turner, N., Grösch, S., Geisslinger, G., Wegner, M.-S.

Sci Rep (2019)

 

Site-Specific Cleavage of RNAs Derived from the PIM1 3′-UTR by a Metal-Free Artificial Ribonuclease

Zellmann, F., Thomas, L., Scheffer, U., Hartmann, R. K., Göbel, M. W.

Molecules (2019)

 

Myeloid-Specific Deletion of the AMPKα2 Subunit Alters Monocyte Protein Expression and Atherogenesis

Fisslthaler, B., Zippel, N., Abdel Malik, R., Delgado Lagos, F., Zukunft, S., Thoele, J., Siuda, D., Soehnlein, O., Wittig, I., Heidler, J., Weigert, A., Fleming, I.

Int. J. Mol. Sci. (2019)

 

VEGF-Trap is a potent modulator of vasoregenerative responses and protects dopaminergic amacrine network integrity in degenerative ischemic neovascular retinopathy

Rojo Arias, J. E., Economopoulou, M., Juárez López, D. A., Kurzbach, A., Au Yeung, K. H., Englmaier, V., Merdausl, M., Schaarschmidt, M., Ader, M., Morawietz, H., Funk, R. H. W., Jászai, J.

J. Neurochem. (2019)

 

Stable depletion of RUNX1-ETO in Kasumi-1 cells induces expression and enhanced proteolytic activity of Cathepsin G and Neutrophil Elastase

Schoenherr, C., Wohlan, K., Dallmann, I., Pich, A., Hegermann, J., Ganser, A., Hilfiker-Kleiner, D., Heidenreich, O., Scherr, M., Eder, M.

PLOS ONE (2019)

 

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