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Knowledge Base

Knowledge Base

BioSping's Oligos In Action

BioSpring is at the forefront of therapeutic nucleic acid manufacturing and analysis, providing comprehensive solutions for drug developers with advanced manufacturing technologies and high-quality, scalable production​.

Discover the diverse applications of nucleic acids, learn how BioSpring supports researchers worldwide, and get an overview of approved oligonucleotide therapeutics.

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Areas of Application of Nucleic Acids

Therapeutic Applications

Nucleic acids have revolutionized medicine by serving as the foundation for therapies targeting genetic conditions, cancers, and more. siRNA, guide RNA, and mRNA-based therapies are gaining significant attention for their precise ability to edit the genome, silence specific genes, and more. These nucleic acid therapeutics, and more, provide targeted approaches to treat diseases that were once considered untreatable.

Cancer Treatment

Therapeutic nucleic acids, such as antisense oligonucleotides (ASOs) and small interfering RNAs (siRNAs), have been widely applied in cancer therapies to silence genes responsible for tumor growth and progression. In addition to gene-silencing approaches, TLRmers like CpG oligonucleotides activate the immune response by targeting Toll-like receptor 9 (TLR9), which can stimulate the body’s immune cells to attack cancerous cells​​.

Genome Editing

Guide RNAs (gRNAs) are essential in precision genome-editing technologies like CRISPR/Cas systems. These guide RNAs enable targeting of specific genetic sequences, allowing for accurate gene editing to treat genetic disorders such as muscular dystrophy, cystic fibrosis, or certain cancers.

Vaccines and Immunotherapies

Nucleic acids are becoming a cornerstone in the development of next-generation vaccines. mRNA-based vaccines have revolutionized immunization strategies by delivering instructions to cells for producing antigens that trigger protective immune responses. Additionally, CpG oligonucleotides, like CpG 7909, act as potent vaccine adjuvants by boosting the immune response. By triggering cytokine release and enhancing antigen-specific antibody production, nucleic acid-based immunotherapies help provide enhanced protection against diseases​.

Genetic Disorders and Rare Diseases

With advancements in therapeutic nucleic acids, treatments for rare genetic disorders are becoming a reality. Antisense oligonucleotides (ASOs) and siRNAs are designed to silence or modify disease-causing genes, offering hope for the eradication of diseases that were previously untreatable.

Cardiovascular and Neurological Diseases

Therapeutic nucleic acids are being applied in the treatment of cardiovascular and neurological diseases. RNA-based therapies, such as mRNAs and antisense RNAs, are developed to target genes associated with heart disease or neurodegenerative conditions like Alzheimer’s​.

Early-Stage Research and Development Projects

In addition to well-established diagnostic applications, nucleic acids are pivotal in early-stage R&D for novel diagnostic methods. As personalized medicine advances, nucleic acids are driving the development of companion diagnostics – tests that identify the patients most likely to benefit from a specific therapy. This approach allows for more targeted and effective treatments based on individual genetic profiles.

At BioSpring, we partner with diagnostic companies to provide custom oligonucleotides that meet the specific needs of early-stage research, offering flexibility in design, length, and modification​. Our capabilities enable the creation of innovative diagnostic solutions, driving forward the next generation of precision medicine.

See how other researchers have partnered with BioSpring for their research and clinical studies in our Featured in Research section below.

Diagnostics

Nucleic acids have transformed the field of diagnostics by enabling highly specific, sensitive, and rapid detection of a wide array of diseases and conditions. At BioSpring, we support the growing demand for oligonucleotides in diagnostics by offering tailored manufacturing services for commercial diagnostic applications such as forensic science, HLA-typing, microbial and viral testing, and more. A closer examination reveals how nucleic acids are shaping key diagnostic areas:

Forensic Science

Nucleic acid-based technologies have revolutionized forensic science, where DNA and RNA are used to identify individuals, analyze biological evidence, and even uncover historical data from ancient remains. Techniques such as polymerase chain reaction (PCR) and short tandem repeat (STR) analysis rely on the precise amplification of nucleic acids, allowing forensic experts to generate DNA profiles from tiny biological samples (such as hair, blood, or skin cells). These profiles can be compared against criminal databases, making it a powerful tool in criminal investigations.

Genomics and Ancestry Testing

Beyond criminal investigations, nucleic acid-based methods are also widely used in genomic ancestry testing. By analyzing mitochondrial DNA (mtDNA) or Y-chromosome sequences, scientists can trace an individual's lineage, revealing genetic relationships that span generations. This has applications in anthropology, genealogy, and population studies.

HLA-Typing (Human Leukocyte Antigen Typing)

HLA-typing is a critical diagnostic tool in organ transplantation and immune-related disease detection. Nucleic acids are used to identify specific HLA gene variants, which are essential for matching organ donors with recipients. Accurate HLA-typing minimizes the risk of organ rejection and improves the success rate of transplants.

Microbial and Viral Testing

Nucleic acids have become indispensable in microbial and viral detection, especially in the diagnosis of infectious diseases. Techniques such as real-time PCR (qPCR), next-generation sequencing (NGS), and isothermal amplification allow for the rapid identification of pathogens like bacteria and viruses with high specificity and sensitivity. The COVID-19 pandemic highlighted the importance of nucleic acids in diagnostics, as PCR-based testing became the gold standard for detecting the SARS-CoV-2 virus. 

Other Applications

Agricultural Biotechnology

In agriculture, nucleic acids are driving innovations in crop protection and improvement. Techniques like RNA interference (RNAi) are being utilized to develop crops with improved resistance to pests and diseases, reduced need for chemical treatments, and enhanced nutritional profiles.

Environmental and Industrial Applications

Beyond health and agriculture, nucleic acids are used in environmental monitoring and industrial biotechnology. Biosensors based on nucleic acids can detect pollutants and pathogens in water, soil, and air, contributing to safer environments. Additionally, nucleic acids serve in the development of biocatalysts and enzymes used in industrial processes, enhancing efficiency and reducing reliance on harmful chemicals.

At BioSpring, we are dedicated to driving scientific progress by providing high-quality nucleic acid services. Our products and solutions have supported pioneering research in various fields - helping scientists push the boundaries of innovation. From early-stage discovery to clinical development, BioSpring materials and expertise have played a crucial role in numerous peer-reviewed publications and breakthrough studies. Explore a selection of research where our contributions have helped shape the future of nucleic acid-based science.

2026 

  • Cystine import and oxidative catabolism fuel vascular growth and repair via nutrient-responsive histone acetylation
    Drekolia, M.-K., Mettner, J., Wang, D., Delgado Lagos, F., Koch, C., Hecker, D., Eresch, J., Mao, Y., Bähr, M., Weichenhan, D., Cordero, J., Wittig, J., Zhang, B., Cui, H., Li, X., Oo, J. A., Weigert, A., Siragusa, M., Klatt, S., Fleming, I., Günther, S., Looso, M., Brandes, R. P., Langer, H. F., Papapetropoulos, A., Singhal, M., Schulz, M. H., Plass, C., Heineke, J., Dobreva, G., Hu, J., Bibli, S.-I.
    Cell Metabolism (2026)


  • Intestinal organoids as a platform for functional evaluation of ASO-mediated splicing modulation in cystic fibrosis
    Stanleigh, N., Irony-Tur Sinai, M., Oren, Y., Ozeri-Galai, E., Hart, G., Grunewald, M., Birimberg-Schwartz, L., Beekman, J. M., Kerem, E., Wilschanski, M., Kerem, B.
    Journal of Cystic Fibrosis (2026)

  • Sensitive and unbiased genome-wide profiling of base-editor-induced off-target activity using CHANGE-seq-BE
    Lazzarotto, C. R., Katta, V., Li, Y., Manquen, G., Wood, R. K., Chyr, J., Urbina, E., Matsubara, A., Lee, G., Wu, X., De Ravin, S. S., Tsai, S. Q.
    Nature Biotechnology (2026)

  • Therapeutic suppression of Tubb4a rescues H-ABC leukodystrophy
    Sase, S., Hacker, J. L., Napit, P. R., Bhagavatula, A., Woidill, S., D’Alessandro, A., Jeffries, M. A., Almad, A., Takanohashi, A., Padiath, Q. S., Grinspan, J. B., Marsh, E. D., Vanderver, A. 
    Molecular Therapy (2026) 

 

2025

  • Lipopolymers as the Basis of Non-Viral Delivery of Therapeutic siRNA Nanoparticles in a Leukemia (MOLM-13) Model
    Yotsomnuk, P., Rajendran, A. P., Sundaram, D. N. M., Morales, L. C., Kucharski, C., Nasrullah, M., Skolpap, W., Jiang, X., Gibson, S. B., Brandwein, J., Uludağ, H.
    Biomolecules (2025)
     

  • FORCE platform overcomes barriers of oligonucleotide delivery to muscle and corrects myotonic dystrophy features in preclinical models
    Weeden, T., Picariello, T., Quinn, B., Spring, S., Shen, P.-Y., Qiu, Q., Vieira, B. F., Schlaefke, L., Russo, R. J., Chang, Y.-A., Cui, J., Yao, M., Wen, A., Hsia, N., Evron, T., Ovington, K., Tsai, P.-N., Yoder, N., Lan, B., Venkatesan, R., Hall, J., Desjardins, C. A., Qatanani, M., Hilderbrand, S., Najim, J., Tang, Z., Tanner, M. K., Subramanian, R., Thornton, C. A., Ibraghimov-Beskrovnaya, O., Zanotti, S.
    Commun Med (2025) 

  • Targeting a Novel Site in Exon 51 with Antisense Oligonucleotides Induces Enhanced Exon Skipping in a Mouse Model of Duchenne Muscular Dystrophy
    Oppeneer, T., Qi, Y., Henshaw, J., Larimore, K., Melton, A., Puoliväli, J., Carter, C., Fant, P., Brennan, S., Wetzel, L. A., Sigg, M. A., Crawford, B. E., Magat, J., Froelich, S., Woloszynek, J. C., O’Neill, C. A. Nucleic Acid Therapeutics (2025)
  • BMN 351-Induced Exon Skipping and Dystrophin Expression in Skeletal and Cardiac Muscle Lead to Preservation of Motor Function in a Mouse Model of Exon 51 Skip-Amenable Duchenne Muscular Dystrophy
    Oppeneer, T., Qi, Y., Henshaw, J., Larimore, K., Puoliväli, J., Carter, C., Fant, P., Brennan, S., Wetzel, L. A., Sigg, M. A., O’Neill, C. A.
    Nucleic Acid Therapeutics (2025)

  • Assessment of native mass spectrometry as a screening method to identify and characterize RNA-targeting small molecules
    Sternicki, L. M., Klose, J. W., Poulsen, S.-A.
    bioRxiv (2025)

  • A potent epigenetic editor targeting human PCSK9 for durable reduction of low-density lipoprotein cholesterol levels
    Tremblay, F., Xiong, Q., Shah, S. S., Ko, C.-W., Kelly, K., Morrison, M. S., Giancarlo, C., Ramirez, R. N., Hildebrand, E. M., Voytek, S. B., El Sebae, G. K., Wright, S. H., Lofgren, L., Clarkson, S., Waters, C., Linder, S. J., Liu, S., Eom, T., Parikh, S., Weber, Y., Martinez, S., Malyala, P., Abubucker, S., Friedland, A. E., Maeder, M. L., Lombardo, A., Myer, V. E., Jaffe, A. B.
    Nat Med (2025)

  • A Simplified Guide RNA Synthesis Protocol for SNAP- and Halo-Tag-Based RNA Editing Tools
    Hofacker, D. T., Kalkuhl, S., Schmid, J. F., Singh, S., Stafforst, T.
    Molecules (2025)

  • BCL11A-deficient human erythropoiesis is impaired in vitro and after xenotransplantation into mice
    Jang, Y., Feng, R., Palmer, L. E., Mayuranathan, T., Yao, Y., Mayberry, K., Zhou, S., Xu, J., Gossett, J., Kang, G., Cheng, Y., Yen, J. S., Weiss, M. J.
    Blood Adv (2025)


  • Cationic lipopolymer based siRNA delivery for experimental lung cancer treatment
    Remant, K., Nasrullah, M., Sandhu, G., Kucharski, C., Lavasanifar, A., Uludag, H.
    Blood Adv (2025)

2024

  • A regulatory loop involving the cytochrome P450-soluble epoxide hydrolase axis and TGF-β signaling
    Li, X., Kempf, S., Delgado Lagos, F., Ukan, Ü., Popp, R., Hu, J., Frömel, T., Günther, S., Weigert, A., Fleming, I.
    iScience (2024)

  • Ionizable nanoemulsions for RNA delivery into the central nervous system – importance of diffusivity
    Borrajo, M. L., Quijano, A., Lapuhs, P., Rodriguez-Perez, A. I., Anthiya, S., Labandeira-Garcia, J. L., Valenzuela, R., Alonso, M. J.
    Journal of Controlled Release (2024)'

  • Introducing a hemoglobin G-Makassar variant in HSCs by in vivo base editing treats sickle cell disease in mice
    Li, C., Georgakopoulou, A., Paschoudi, K., Anderson, A. K., Huang, L., Gil, S., Giannaki, M., Vlachaki, E., Newby, G. A., Liu, D. R., Yannaki, E., Kiem, H.-P., Lieber, A.
    Molecular Therapy (2024)

  • An improved SNAP-ADAR tool enables efficient RNA base editing to interfere with post-translational protein modification
    Kiran Kumar, K. D., Singh, S., Schmelzle, S. M., Vogel, P., Fruhner, C., Hanswillemenke, A., Brun, A., Wettengel, J., Füll, Y., Funk, L., Mast, V., Botsch, J. J., Reautschnig, P., Li, J. B., Stafforst, T.
    Nat Commun (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)

  • Antithrombotic but not anticoagulant activity of the thrombin-binding RNA aptamer Apta-1
    Jedlina, L., Paramel, G., Soboleva, S., Chutna Olin, O., Haug, M., Fransén, K., Lindstam, M., Brewinska-Olchowik, M., Piwocka, K., Grenegård, M.
    British Journal of Pharmacology (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.
    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)

giving hope - changing lifes

Approved Oligonucleotide Therapeutics

Nucleic acid-based therapeutics have emerged as a powerful class of medicines, revolutionizing the treatment of genetic disorders, cancers, and other diseases. To date, 27 oligonucleotide-based therapies have received regulatory approval, highlighting the growing impact of these innovative treatments. Notably, half of these approvals were granted in the last 5 years, reflecting significant advancements in the field and an accelerated pace of clinical translation.

The following list provides an overview of all currently approved nucleic acid-based therapies, demonstrating the versatility and potential of oligonucleotides to address diverse medical challenges.

Trade Name (Drug name)

Year of Approval FDA/EMA

Modality

Disease Indication

REDEMPLO (Plozasiran)

2025/NA

Small Interfering RNA (siRNA)

Familial Chylomicronemia Syndrome (FCS) 

Dawnzera (Donidalorsen)

2025/2026

Antisense Oligonucleotide (ASO) 

Hereditary angioedema (HAE) 

QFITLIA (Fitusiran) 

2025/NA

Small Interfering RNA (siRNA) 

Hemophilia A and B 

Rytelo (Imetelstat)

2024/2025

Antisense Oligonucleotide (ASO)

Transfusion-dependent anaemia of MDS patients 

Tryngolza (Olezarsen)

2024/2025

Antisense Oligonucleotide (ASO)

Familial chylomicronemia syndrome (FCS) 

Wainua (Eplontersen)

2023/2025

Antisense Oligonucleotide (ASO) 

TTR-mediated polyneuropathy/TTR-mediated cardiomyopathy 

Casgevy (Exagamglogene autotemcel)

2023/2024

CRISPR-Cas9 Gene Editing

Sickle cell anemia

Rivfloza (Nedosiran)

2023/NA

Small Interfering RNA (siRNA) 

Primary hyperoxaluria 

Cyfendus (Anthrax Vaccine Absorbed, Adjuvanted) 

 2023/NA 

 CpG ODN 

Postexposure prophylaxis for anthrax  

Izervay (Avacincaptad pegol)

2023/2023

Aptamer

 Geographic atrophy of age-related macular degeneration 

Qualsody (Tofersen)

2023/2022

Antisense Oligonucleotide (ASO)

Amyotrophic lateral sclerosis 

Amvuttra (Vutrisiran)

2022/2022

Small Interfering RNA (siRNA)

Polyneuropathy of hereditary transthyretin amyloidosis 

Leqvio (Inclisiran)

2021/2020

Small Interfering RNA (siRNA) 

Hypercholesteremia, primary hyperlipidemia  

Amondys 45 (Casimersen) 

2021/NA

ASO (PMO) (Phosphorodiamidate morpholino oligomer)

Duchenne muscular dystrophy

Oxlumo (Lumasiran)

2020/2020

Small Interfering RNA (siRNA)

Primary hyperoxaluria type 1

Viltepso (Viltolarsen)

2020/2020

ASO (PMO) (Phosphorodiamidate morpholino oligomer) 

Duchenne muscular dystrophy

Vyondys 53 (Golodirsen)

2019/NA

ASO (PMO) (Phosphorodiamidate morpholino oligomer) 

Duchenne muscular dystrophy

Givlaari (Givosiran)

2019/2020

Small Interfering RNA (siRNA)

Acute hepatic porphyria

Waylivra (Volanesorsen)

NA/2019

Antisense Oligonucleotide (ASO)

Familial chylomicronemia syndrome

Tagsedi (Inotersen)

2018/2018

Antisense Oligonucleotide (ASO)

Polyneuropathy of hereditary transthyretin amyloidosis

Onpattro (Patisiran)

2018/2018

Small Interfering RNA (siRNA)

Polyneuropathy of hereditary transthyretin amyloidosis

Hepislav-B

2017/2021

CpG ODN

Hepatitis B

Spinraza (Nusinersen)

2016/2017

Antisense Oligonucleotide (ASO)

Spinal muscular atrophy

Exondys 51 (Eteplirsen)

2016/NA

ASO (PMO) (Phosphorodiamidate morpholino oligomer)

Duchenne muscular dystrophy

Kynamro (Mipomersen)(withdrawn) 

2013/NA

Antisense Oligonucleotide (ASO)

Homozygous familial hypercholesteremia 

Macugen (Pegaptanib)(withdrawn) 

2004/2006

Aptamer

 Age-related macular degeneration 

Vitravene (Fomivirsen)  (withdrawn)

1998/1999

Antisense Oligonucleotide (ASO)

 CMV retinitis 

 

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