From bench side to clinic: Potential and challenges of RNA vaccines and therapeutics in infectious diseases

Paving the Road for RNA Therapeutics

Dammes, N., & Peer, D. (2020) · Trends in Pharmacological Sciences · doi:10.1016/j.tips.2020.08.004 · Cited by 223

A Dual Targeting Dendrimer-Mediated siRNA Delivery System for Effective Gene Silencing in Cancer Therapy

Dong, Y., Yu, T., Ding, L., Laurini, E., Huang, Y., Zhang, M., Weng, Y., Lin, S., Chen, P., Marson, D., et al. (2018) · Journal of the American Chemical Society · doi:10.1021/jacs.8b10021 · Cited by 221

Current Development of siRNA Bioconjugates: From Research to the Clinic

Chernikov, I. V., Vlassov, V. V., & Chernolovskaya, E. L. (2019) · Frontiers in Pharmacology · doi:10.3389/fphar.2019.00444 · Cited by 221

Modified mRNA Vaccines Protect against Zika Virus Infection

Richner, J. M., Himansu, S., Dowd, K. A., Butler, S. L., Salazar, V., Fox, J. M., Julander, J. G., Tang, W. W., Shresta, S., Pierson, T. C., et al. (2017) · Cell · doi:10.1016/j.cell.2017.03.016 · Cited by 221

Systemic delivery of siRNA to tumors using a lipid nanoparticle containing a tumor-specific cleavable PEG-lipid

Hatakeyama, H., Akita, H., Ito, E., Hayashi, Y., Oishi, M., Nagasaki, Y., Danev, R., Nagayama, K., Kaji, N., Kikuchi, H., et al. (2011) · Biomaterials · doi:10.1016/j.biomaterials.2011.02.045 · Cited by 219

Intrathecal Injections in Children With Spinal Muscular Atrophy

Haché, M., Swoboda, K. J., Sethna, N. F., Farrow-Gillespie, A. C., Khandji, A. G., Xia, S., & Bishop, K. M. (2016) · Journal of Child Neurology · doi:10.1177/0883073815627882 · Cited by 215

Genome editing of CXCR4 by CRISPR/cas9 confers cells resistant to HIV-1 infection

Hou, P., Chen, S., Wang, S., Yu, X., Chen, Y. Z., Jiang, M., Zhuang, K., Ho, W., Hou, W., Huang, J. Y., et al. (2015) · Scientific Reports · doi:10.1038/srep15577 · Cited by 214

Advantages of 2'-O-methyl oligoribonucleotide probes for detecting RNA targets

Majlessi, M. (1998) · Nucleic Acids Research · doi:10.1093/nar/26.9.2224 · Cited by 213

Excision of HIV-1 DNA by gene editing: a proof-of-concept in vivo study

Kaminski, R., Bella, R., Yin, C., Otte, J., Ferrante, P., Gendelman, H. E., Li, H., Booze, R. M., Gordon, J., Hu, W., et al. (2016) · Gene Therapy · doi:10.1038/gt.2016.41 · Cited by 213

Exploring Chemical Modifications for siRNA Therapeutics: A Structural and Functional Outlook

Shukla, S., Sumaria, C. S., & Pradeepkumar, P. I. (2009) · ChemMedChem · doi:10.1002/cmdc.200900444 · Cited by 212

Long-term safety and efficacy of microRNA-targeted therapy in chronic hepatitis C patients

van der Ree, M. H., van der Meer, A. J., de Bruijne, J., Maan, R., van Vliet, A., Welzel, T. M., Zeuzem, S., Lawitz, E., Rodriguez‐Torres, M., Kupčová, V., et al. (2014) · Antiviral Research · doi:10.1016/j.antiviral.2014.08.015 · Cited by 208

Induction of an IFN-mediated antiviral response by a self-amplifying RNA vaccine: implications for vaccine design

Pepini, T., Pulichino, A.-M., Carsillo, T., Carlson, A. L., Sari-Sarraf, F., Ramsauer, K., Debasitis, J. C., Maruggi, G., Otten, G. R., Geall, A. J., et al. (2017) · The Journal of Immunology · doi:10.4049/jimmunol.1601877 · Cited by 208

Emergence of synthetic mRNA: In vitro synthesis of mRNA and its applications in regenerative medicine

Kwon, H., Kim, M., Seo, Y., Moon, Y. S., Lee, H. J., Lee, K., & Lee, H. (2018) · Biomaterials · doi:10.1016/j.biomaterials.2017.11.034 · Cited by 208

How mRNA therapeutics are entering the monoclonal antibody field

Van Hoecke, L., & Roose, K. (2019) · Journal of Translational Medicine · doi:10.1186/s12967-019-1804-8 · Cited by 205

Translational Efficiency Is Regulated by the Length of the 3′ Untranslated Region

Tanguay, R. L., & Gallie, D. R. (1996) · Molecular and Cellular Biology · doi:10.1128/mcb.16.1.146 · Cited by 204

A lipid-encapsulated mRNA encoding a potently neutralizing human monoclonal antibody protects against chikungunya infection

Kose, N., Fox, J. M., Sapparapu, G., Bombardi, R., Tennekoon, R. N., de Silva, A. D., Elbashir, S. M., Theisen, M. A., Humphris-Narayanan, E., Ciaramella, G., et al. (2019) · Science Immunology · doi:10.1126/sciimmunol.aaw6647 · Cited by 202

Potent inhibition of microRNA in vivo without degradation

Davis, S. P., Propp, S. S., Freier, S. M., Jones, L., Serra, M. J., Kinberger, G. A., Bhat, B., Swayze, E. E., Bennett, C. F., & Esau, C. (2008) · Nucleic Acids Research · doi:10.1093/nar/gkn904 · Cited by 201

Long-circulating siRNA nanoparticles for validating Prohibitin1-targeted non-small cell lung cancer treatment

Zhu, X., Xu, Y., Solis, L. M., Wei, T., Wang, L., Behrens, C., Xu, X., Zhao, L., Liu, D. X., Wu, J., et al. (2015) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1505629112 · Cited by 200

Telomerase Inhibitor Imetelstat in Patients with Essential Thrombocythemia

Baerlocher, G. M., Leibundgut, E. O., Ottmann, O. G., Spitzer, G., Odenike, O. M., McDevitt, M. A., Röth, A., Daskalakis, M., Burington, B. E., Stuart, M. J., et al. (2015) · New England Journal of Medicine · doi:10.1056/nejmoa1503479 · Cited by 199

Human Clinical Trials of Plasmid DNA Vaccines

Liu, M. A., & Ulmer, J. B. (2005) · Advances in genetics · doi:10.1016/s0065-2660(05)55002-8 · Cited by 198

Self-replicating Semliki Forest virus RNA as recombinant vaccine

Zhou, X., Berglund, P., Rhodes, G., Parker, S. E., Jondal, M., & Liljeström, P. (1994) · Vaccine · doi:10.1016/0264-410x(94)90074-4 · Cited by 193

The packaging of siRNA within the mesoporous structure of silica nanoparticles

Xu, L., Xie, Q. R., Zhang, J., Xia, W., & Gu, H. (2011) · Biomaterials · doi:10.1016/j.biomaterials.2011.08.068 · Cited by 193

Selective uptake of naked vaccine RNA by dendritic cells is driven by macropinocytosis and abrogated upon DC maturation

Diken, M., Kreiter, S., Selmi, A., Britten, C. M., Huber, C. G., Türeci, Ö., & Şahin, U. (2011) · Gene Therapy · doi:10.1038/gt.2011.17 · Cited by 192

Distinct Uptake Routes of Cell-Penetrating Peptide Conjugates

Lundin, P., Johansson, H. J., Guterstam, P., Holm, T., Hansen, M., Langel, Ü., & Andaloussi, S. E. (2008) · Bioconjugate Chemistry · doi:10.1021/bc800212j · Cited by 192

A single dose of self-transcribing and replicating RNA-based SARS-CoV-2 vaccine produces protective adaptive immunity in mice

de Alwis, R., Gan, E. S., Chen, S., Leong, Y. S., Tan, H. C., Zhang, S. L., Yau, C., Low, J. G. H., Kalimuddin, S., Matsuda, D., et al. (2021) · Molecular Therapy · doi:10.1016/j.ymthe.2021.04.001 · Cited by 191

miRNAs reshape immunity and inflammatory responses in bacterial infection

Zhou, X., Li, X., & Wu, M. (2018) · Signal Transduction and Targeted Therapy · doi:10.1038/s41392-018-0006-9 · Cited by 190

Fomivirsen – a phosphorothioate oligonucleotide for the treatment of CMV retinitis

de Smet, M. D., Meenken, C., & van den Horn, G. J. (1999) · Ocular Immunology and Inflammation · doi:10.1076/ocii.7.3.189.4007 · Cited by 189

Development of CRISPR-Cas systems for genome editing and beyond

Zhang, F. (2019) · Quarterly Reviews of Biophysics · doi:10.1017/s0033583519000052 · Cited by 188

CpG Still Rocks! Update on an Accidental Drug

Krieg, A. Μ. (2012) · Nucleic Acid Therapeutics · doi:10.1089/nat.2012.0340 · Cited by 184

Development of Exon Skipping Therapies for Duchenne Muscular Dystrophy: A Critical Review and a Perspective on the Outstanding Issues

Aartsma-Rus, A. M., Straub, V. W., Hemmings, R., Haas, M., Schlosser‐Weber, G., Stoyanova-Beninska, V. V., Mercuri, E. M., Francesco Muntoni, A., Sepodes, B. M. N., Vroom, E., et al. (2017) · Nucleic Acid Therapeutics · doi:10.1089/nat.2017.0682 · Cited by 182

A Single siRNA Suppresses Fatal Encephalitis Induced by Two Different Flaviviruses

Kumar, P., Lee, S. K., Shankar, P., & Manjunath, N. (2006) · PLoS Medicine · doi:10.1371/journal.pmed.0030096 · Cited by 180

mRNA-Based Protein Replacement Therapy for the Heart

Magadum, A., Kaur, K., & Zangi, L. (2019) · Molecular Therapy · doi:10.1016/j.ymthe.2018.11.018 · Cited by 176

Delivery of self-amplifying mRNA vaccines by cationic lipid nanoparticles: The impact of cationic lipid selection

Lou, G., Anderluzzi, G., Schmidt, S., Woods, S., Gallorini, S., Brazzoli, M., Giusti, F., Ferlenghi, I., Johnson, R. N., Roberts, C. W., et al. (2020) · Journal of Controlled Release · doi:10.1016/j.jconrel.2020.06.027 · Cited by 176

The Clinical Application of MicroRNAs in Infectious Disease

Drury, R. E., O’Connor, D., & Pollard, A. J. (2017) · Frontiers in Immunology · doi:10.3389/fimmu.2017.01182 · Cited by 172

Antisense-mediated lowering of plasma apolipoprotein C-III by volanesorsen improves dyslipidemia and insulin sensitivity in type 2 diabetes

Digenio, A., Dunbar, R. L., Alexander, V. J., Hompesch, M., Morrow, L., Lee, R. G., Graham, M. J., Hughes, S. G., Yu, R., Singleton, W., et al. (2016) · Diabetes Care · doi:10.2337/dc16-0126 · Cited by 170

ALN-RSV01 for prevention of bronchiolitis obliterans syndrome after respiratory syncytial virus infection in lung transplant recipients

Gottlieb, J., Zamora, M. R., Hodges, T. N., Musk, A. W., Sommerwerck, U., Dilling, D. F., Arcasoy, S. M., DeVincenzo, J. P., Karsten, V., Shah, S. D., et al. (2015) · The Journal of Heart and Lung Transplantation · doi:10.1016/j.healun.2015.08.012 · Cited by 168

Crystal structure of NF-κB (p50) 2 complexed to a high-affinity RNA aptamer

Huang, D.-B., Vu, D., Cassiday, L. A., Zimmerman, J. M., Maher, L. J., & Ghosh, G. (2003) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1632011100 · Cited by 167

Clinical and Preclinical Pharmacokinetics and Pharmacodynamics of Mipomersen (Kynamro®): A Second-Generation Antisense Oligonucleotide Inhibitor of Apolipoprotein B

Geary, R. S., Baker, B. F., & Crooke, S. T. (2015) · Clinical Pharmacokinetics · doi:10.1007/s40262-014-0224-4 · Cited by 165

Experimental Treatment of Ebola Virus Disease with TKM-130803: A Single-Arm Phase 2 Clinical Trial

Dunning, J. W., Sahr, F., Rojek, A. M., Gannon, F., Carson, G., Idriss, B. R., Massaquoi, T. A., Gandi, R., Joseph, S., Osman, H. K., et al. (2016) · PLoS Medicine · doi:10.1371/journal.pmed.1001997 · Cited by 165

The human cytomegalovirus microRNA miR-UL112 acts synergistically with a cellular microRNA to escape immune elimination

Nachmani, D., Lankry, D., Wolf, D. G., & Mandelboim, O. (2010) · Nature Immunology · doi:10.1038/ni.1916 · Cited by 164

Chemical modifications of nucleic acid drugs and their delivery systems for gene‐based therapy

Chen, C., Yang, Z., & Tang, X. (2018) · Medicinal Research Reviews · doi:10.1002/med.21479 · Cited by 164

Cobomarsen, an Oligonucleotide Inhibitor of miR-155, Slows DLBCL Tumor Cell Growth In Vitro and In Vivo

Anastasiadou, E., Seto, A. G., Beatty, X., Hermreck, M., Gilles, M.-E., Stroopinsky, D., Pinter‐Brown, L. C., Pestano, L. A., Marchese, C., Avigan, D., et al. (2020) · Clinical Cancer Research · doi:10.1158/1078-0432.ccr-20-3139 · Cited by 163

Cell-Penetrating Peptides to Enhance Delivery of Oligonucleotide-Based Therapeutics

McClorey, G., & Banerjee, S. (2018) · Biomedicines · doi:10.3390/biomedicines6020051 · Cited by 161

Dendrimers for siRNA Delivery

Biswas, S., & Torchilin, V. P. (2013) · Pharmaceuticals · doi:10.3390/ph6020161 · Cited by 158

Long-term safety and efficacy of patisiran for hereditary transthyretin-mediated amyloidosis with polyneuropathy: 12-month results of an open-label extension study

Adams, D., Polydefkis, M., González-Duarte, A., Wixner, J., Kristen, A. V., Schmidt, H. H., Berk, J. L., Losada López, I. A., Dispenzieri, A., Quan, D., et al. (2021) · The Lancet Neurology · doi:10.1016/s1474-4422(20)30368-9 · Cited by 158

[4] Synthesis of long, capped transcripts in Vitro by SP6 and T7 RNA polymerases

Yisraeli, J. K., & Melton, D. A. (1989) · Methods in enzymology on CD-ROM/Methods in enzymology · doi:10.1016/0076-6879(89)80090-4 · Cited by 156

Rapid progression of familial amyloidotic polyneuropathy

Adams, D., Coelho, T., Obici, L. P., Merlini, G., Mincheva, Z., Suanprasert, N., Bettencourt, B. R., Gollob, J., Gandhi, P. J., Litchy, W. J., et al. (2015) · Neurology · doi:10.1212/wnl.0000000000001870 · Cited by 155

Big Is Beautiful: Enhanced saRNA Delivery and Immunogenicity by a Higher Molecular Weight, Bioreducible, Cationic Polymer

Blakney, A. K., Zhu, Y., McKay, P. F., Bouton, C. R., Yeow, J., Tang, J., Hu, K., Samnuan, K., Grigsby, C. L., Shattock, R. J., et al. (2020) · ACS Nano · doi:10.1021/acsnano.0c00326 · Cited by 155

Induction of Broad-Based Immunity and Protective Efficacy by Self-amplifying mRNA Vaccines Encoding Influenza Virus Hemagglutinin

Brazzoli, M., Magini, D., Bonci, A., Buccato, S., Giovani, C., Kratzer, R., Zurli, V., Mangiavacchi, S., Casini, D., Brito, L. M., et al. (2016) · Journal of Virology · doi:10.1128/jvi.01786-15 · Cited by 154

Comparative Analysis of Antisense Oligonucleotide Sequences for Targeted Skipping of Exon 51 During Dystrophin Pre-mRNA Splicing in Human Muscle

Arechavala‐Gomeza, V., Graham, I. R., Popplewell, L. J., Adams, A. M., Aartsma-Rus, A. M., Kinali, M., Morgan, J. E., van Deutekom, J. C., Wilton, S. D., Dickson, G., et al. (2007) · Human Gene Therapy · doi:10.1089/hum.2006.061 · Cited by 152

Long-term efficacy and safety of mipomersen in patients with familial hypercholesterolaemia: 2-year interim results of an open-label extension

Santos, R. D., Duell, P. B., East, C. A., Guyton, J. R., Moriarty, P. M., Chin, W., & Mittleman, R. S. (2013) · European Heart Journal · doi:10.1093/eurheartj/eht549 · Cited by 149

Immunogenicity and protective efficacy induced by self-amplifying mRNA vaccines encoding bacterial antigens

Maruggi, G., Chiarot, E., Giovani, C., Buccato, S., Bonacci, S., Frigimelica, E., Margarit, I., Geall, A., Bensi, G., & Maione, D. (2017) · Vaccine · doi:10.1016/j.vaccine.2016.11.040 · Cited by 149

MicroRNA-mediated species-specific attenuation of influenza A virus

Perez, J. T., Pham, A. M., Lorini, M. H., Chua, M. A., Steel, J., & tenOever, B. R. (2009) · Nature Biotechnology · doi:10.1038/nbt.1542 · Cited by 146

FDA approves patisiran to treat hereditary transthyretin amyloidosis

Wood, H. (2018) · Nature Reviews Neurology · doi:10.1038/s41582-018-0065-0 · Cited by 145

Engineering Nanoparticles for Targeted Delivery of Nucleic Acid Therapeutics in Tumor

Xiao, Y., Shi, K. Y., Qu, Y., Chu, B., & Qian, Z. (2018) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2018.09.002 · Cited by 144

Suppressing transthyretin production in mice, monkeys and humans using 2nd-Generation antisense oligonucleotides

Ackermann, E. J., Guo, S., Benson, M. D., Booten, S. L., Freier, S., Hughes, S. G., Kim, T., Kwoh, T. J., Matson, J. E., Norris, D., et al. (2016) · Amyloid · doi:10.1080/13506129.2016.1191458 · Cited by 143

Fomivirsen approved for CMV retinitis.

Roehr, B. (1998) · PubMed · Cited by 140

Anti-Human Immunodeficiency Virus Hematopoietic Progenitor Cell-Delivered Ribozyme in a Phase I Study: Myeloid and Lymphoid Reconstitution in Human Immunodeficiency Virus Type-1–Infected Patients

Amado, R. G., Mitsuyasu, R. T., Rosenblatt, J. D., Ngok, F. K., Bakker, A., Cole, S. W., Chorn, N., Lin, L.-S., Bristol, G. C., Boyd, M. P., et al. (2004) · Human Gene Therapy · doi:10.1089/104303404322886101 · Cited by 139

Aptamers in Diagnostics and Treatment of Viral Infections

Wandtke, T., Woźniak, J., & Kopiński, P. (2015) · Viruses · doi:10.3390/v7020751 · Cited by 138

Monoclonal antibody-based molecular imaging strategies and theranostic opportunities

Dammes, N., & Peer, D. (2020) · Theranostics · doi:10.7150/thno.37443 · Cited by 138

Oligodeoxynucleotides lacking CpG dinucleotides mediate Toll‐like receptor 9 dependent T helper type 2 biased immune stimulation

Vollmer, J., Weeratna, R. D., Jurk, M., Samulowitz, U., McCluskie, M. J., Payette, P., Davis, H. L., Schetter, C., & Krieg, A. Μ. (2004) · Immunology · doi:10.1111/j.1365-2567.2004.01962.x · Cited by 137

CRISPR-Cas9 modified bacteriophage for treatment of Staphylococcus aureus induced osteomyelitis and soft tissue infection

Cobb, L. H., Park, J., Swanson, E. A., Beard, M. C., McCabe, E. M., Rourke-Funderburg, A. S., Seo, K. S., Olivier, A. K., & Priddy, L. B. (2019) · PLoS ONE · doi:10.1371/journal.pone.0220421 · Cited by 135

Highly efficient siRNA delivery from core–shell mesoporous silica nanoparticles with multifunctional polymer caps

Möller, K., Müller, K., Engelke, H., Bräuchle, C., Wagner, E., & Bein, T. (2015) · Nanoscale · doi:10.1039/c5nr06246b · Cited by 133

Long-term mipomersen treatment is associated with a reduction in cardiovascular events in patients with familial hypercholesterolemia

Duell, P. B., Santos, R. D., Kirwan, B., Witztum, J. L., Tsimikas, S., & Kastelein, J. J. P. (2016) · Journal of clinical lipidology · doi:10.1016/j.jacl.2016.04.013 · Cited by 132

Removal of Integrated Hepatitis B Virus DNA Using CRISPR-Cas9

Li, H., Sheng, C., Wang, S., Yang, L., Liang, Y., Huang, Y., Liu, H., Li, P., Yang, C., Yang, X., et al. (2017) · Frontiers in Cellular and Infection Microbiology · doi:10.3389/fcimb.2017.00091 · Cited by 131

The Principles of MiRNA-Masking Antisense Oligonucleotides Technology

Wang, Z. (2010) · Methods in molecular biology · doi:10.1007/978-1-60761-863-8_3 · Cited by 129

Inhibition of HSV-1 Replication by Gene Editing Strategy

Roehm, P. C., Shekarabi, M., Wollebo, H. S., Bellizzi, A., He, L., Salkind, J., & Khalili, K. (2016) · Scientific Reports · doi:10.1038/srep23146 · Cited by 128

Targeted delivery of anti-miR-155 by functionalized mesoporous silica nanoparticles for colorectal cancer therapy

Li, Y., Duo, Y., Bi, J., Zeng, X., Mei, L., Bao, S., He, L., Shan, A., Zhang, Y., & Yu, X. (2018) · International Journal of Nanomedicine · doi:10.2147/ijn.s158290 · Cited by 128

RUNNING INTERFERENCE: Prospects and Obstacles to Using Small Interfering RNAs as Small Molecule Drugs

Dykxhoorn, D. M., & Lieberman, J. (2006) · Annual Review of Biomedical Engineering · doi:10.1146/annurev.bioeng.8.061505.095848 · Cited by 127

Self-Amplifying mRNA Vaccines Expressing Multiple Conserved Influenza Antigens Confer Protection against Homologous and Heterosubtypic Viral Challenge

Magini, D., Giovani, C., Mangiavacchi, S., Maccari, S., Cecchi, R., Ulmer, J. B., Gregorio, E. D., Geall, A. J., Brazzoli, M., & Bertholet, S. (2016) · PLoS ONE · doi:10.1371/journal.pone.0161193 · Cited by 127

A Randomized, Double-Masked, Placebo-Controlled Study of Alicaforsen, an Antisense Inhibitor of Intercellular Adhesion Molecule 1, for the Treatment of Subjects With Active Crohn’s Disease

Yacyshyn, B., Chey, W. Y., Wedel, M. K., Yu, R. Z., Paul, D., & Chuang, E. (2007) · Clinical Gastroenterology and Hepatology · doi:10.1016/j.cgh.2006.11.001 · Cited by 125

Progress and perspective of inorganic nanoparticle-based siRNA delivery systems

Jiang, Y. Y., Huo, S., Hardie, J. M., Liang, X., & Rotello, V. M. (2016) · Expert Opinion on Drug Delivery · doi:10.1517/17425247.2016.1134486 · Cited by 125

Targeted delivery of CRISPR/Cas9 to prostate cancer by modified gRNA using a flexible aptamer-cationic liposome

Zhen, S., Takahashi, Y., Narita, S., Yang, Y., & Xu, L. (2016) · Oncotarget · doi:10.18632/oncotarget.14072 · Cited by 125

Imetelstat Achieves Meaningful and Durable Transfusion Independence in High Transfusion–Burden Patients With Lower-Risk Myelodysplastic Syndromes in a Phase II Study

Steensma, D. P., Fenaux, P., Eygen, K. V., Raza, A., Santini, V., Germing, U., Font, P., Díez‐Campelo, M., Thépot, S., Vellenga, E., et al. (2020) · Journal of Clinical Oncology · doi:10.1200/jco.20.01895 · Cited by 125

RNA Modifications Modulate Activation of Innate Toll-Like Receptors

Freund, I., Eigenbrod, T., Helm, M., & Dalpke, A. H. (2019) · Genes · doi:10.3390/genes10020092 · Cited by 123

Potent lipoprotein(a) lowering following apolipoprotein(a) antisense treatment reduces the pro-inflammatory activation of circulating monocytes in patients with elevated lipoprotein(a)

Stiekema, L. C. A., Prange, K. H. M., Hoogeveen, R. M., Verweij, S. L., Kroon, J., Schnitzler, J. G., Dzobo, K. E., Cupido, A. J., Tsimikas, S., Stroes, E. S. G., et al. (2020) · European Heart Journal · doi:10.1093/eurheartj/ehaa171 · Cited by 119

Therapeutic potential of chemically modified si RNA : Recent trends

Selvam, C., Mutisya, D., Prakash, S., Ranganna, K., & Thilagavathi, R. (2017) · Chemical Biology & Drug Design · doi:10.1111/cbdd.12993 · Cited by 117

The Expanding View of RNA and DNA Function

Breaker, R. R., & Joyce, G. F. (2014) · Chemistry & Biology · doi:10.1016/j.chembiol.2014.07.008 · Cited by 114

Functional high-throughput screening identifies the miR-15 microRNA family as cellular restriction factors for Salmonella infection

Maudet, C., Mano, M., Ushasree, S., Sharan, M., Giacca, M., Förstner, K. U., & Eulálio, A. (2014) · Nature Communications · doi:10.1038/ncomms5718 · Cited by 113

Nucleolin Interacts with the Dengue Virus Capsid Protein and Plays a Role in Formation of Infectious Virus Particles

Balinsky, C. A., Schmeisser, H., Ganesan, S., Singh, K., Pierson, T. C., & Zoon, K. C. (2013) · Journal of Virology · doi:10.1128/jvi.00704-13 · Cited by 113

Novel antisense inhibition of diacylglycerol O-acyltransferase 2 for treatment of non-alcoholic fatty liver disease: a multicentre, double-blind, randomised, placebo-controlled phase 2 trial

Loomba, R. S., Morgan, E. S., Watts, L. M., Xia, S., Hannan, L. A., Geary, R. S., Baker, B. F., & Bhanot, S. (2020) · The Lancet. Gastroenterology & hepatology · doi:10.1016/s2468-1253(20)30186-2 · Cited by 113

Selective tissue targeting of synthetic nucleic acid drugs

Seth, P. P., Tanowitz, M., & Bennett, C. F. (2019) · Journal of Clinical Investigation · doi:10.1172/jci125228 · Cited by 112

Rotavirus gene silencing by small interfering RNAs

Déctor, M. A., Romero, P. A., López, S., & Arias, C. F. (2002) · EMBO Reports · doi:10.1093/embo-reports/kvf234 · Cited by 111

Safety and Efficacy of a Lentiviral Vector Containing Three Anti-HIV Genes—CCR5 Ribozyme, Tat-rev siRNA, and TAR Decoy—in SCID-hu Mouse–Derived T Cells

Anderson, J. S., Li, M.-J., Palmer, B. E., Remling‐Mulder, L., Li, S. X., Yam, P. Y., Yee, J., Rossi, J. J., Zaia, J. A., & Akkina, R. (2007) · Molecular Therapy · doi:10.1038/sj.mt.6300157 · Cited by 111

Improved Nucleic Acid Therapy with Advanced Nanoscale Biotechnology

Weng, Y., Huang, Q., Li, C., Yang, Y., Wang, X., Yu, J., Huang, Y., & Liang, X. (2019) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2019.12.004 · Cited by 110

Probing the Influence of Stereoelectronic Effects on the Biophysical Properties of Oligonucleotides: Comprehensive Analysis of the RNA Affinity, Nuclease Resistance, and Crystal Structure of Ten 2‘-O-Ribonucleic Acid Modifications,

Egli, M., Minasov, G., Tereshko, V. A., Pallan, P. S., Teplova, M., Inamati, G. B., Lesnik, E. A., Owens, S. R., Ross, B. S., Prakash, T. P., et al. (2005) · Biochemistry · doi:10.1021/bi050574m · Cited by 109

A phase I dose-escalation study of apatorsen (OGX-427), an antisense inhibitor targeting heat shock protein 27 (Hsp27), in patients with castration-resistant prostate cancer and other advanced cancers

Chi, K. N., Yu, E. Y., Jacobs, C. A., Bazov, J., Kollmannsberger, C., Higano, C. S., Mukherjee, S. D., Gleave, M. E., STEWART, P. S., & Hotte, S. J. (2016) · Annals of Oncology · doi:10.1093/annonc/mdw068 · Cited by 109

Role of microRNAs in antiviral responses to dengue infection

Wong, R. R., Abd-Aziz, N., Affendi, S., & Poh, C. L. (2020) · Journal of Biomedical Science · doi:10.1186/s12929-019-0614-x · Cited by 109

Unique quadruplex structure and interaction of an RNA aptamer against bovine prion protein

Mashima, T., Matsugami, A., Nishikawa, F., Nishikawa, S., & Katahira, M. (2009) · Nucleic Acids Research · doi:10.1093/nar/gkp647 · Cited by 107

Pharmacokinetics and Pharmacodynamics of the Small Interfering Ribonucleic Acid, Givosiran, in Patients With Acute Hepatic Porphyria

Agarwal, S., Simon, A. R., Goel, V., Habtemariam, B. A., Clausen, V. A., Kim, J. B., & Robbie, G. J. (2020) · Clinical Pharmacology & Therapeutics · doi:10.1002/cpt.1802 · Cited by 104

Safety and Efficacy Clinical Trials for SYL1001, a Novel Short Interfering RNA for the Treatment of Dry Eye Disease

Benitez-del-Castillo, J. M., Moreno‐Montañés, J., Jiménez-Alfaro, I., Muñoz-Negrete, F. J., Turman, K., Palumaa, K., Sádaba, B., González, M. V., Ruz, V., Vargas, B., et al. (2016) · Investigative Ophthalmology & Visual Science · doi:10.1167/iovs.16-20303 · Cited by 102

Targeted Delivery of Small Interfering RNA to Human Dendritic Cells To Suppress Dengue Virus Infection and Associated Proinflammatory Cytokine Production

Subramanya, S., Kim, S. S., Abraham, S., Yao, J., Kumar, M., Kumar, P., Haridas, V., Lee, S., Shultz, L. D., Greiner, D. L., et al. (2009) · Journal of Virology · doi:10.1128/jvi.02105-08 · Cited by 100

Therapeutic Potential of Small Molecules and Engineered Proteins

Gurevich, E. V., & Gurevich, V. V. (2013) · Handbook of experimental pharmacology · doi:10.1007/978-3-642-41199-1_1 · Cited by 99

A sindbis virus mRNA polynucleotide vector achieves prolonged and high level heterologous gene expression in vivo

Johanning, F. W., Conry, R. M., LoBuglio, A. F., Wright, M., Sumerel, L. A., Pike, M. J., & Curiel, D. T. (1995) · Nucleic Acids Research · doi:10.1093/nar/23.9.1495 · Cited by 99

Inclisiran: a new promising agent in the management of hypercholesterolemia

Kosmas, C. E., Muñoz Estrella, A., Sourlas, A., Silverio, D., Hilario, E., Montan, P. D., & Guzman, E. (2018) · Diseases · doi:10.3390/diseases6030063 · Cited by 99

Antisense PNA tridecamers targeted to the coding region of ha-ras mRNA arrest polypeptide chain elongation

Dias, N., Dheur, S., Nielsen, P. E., Gryaznov, S. M., Aerschot, A. A. V., Herdewijn, P. A., Hélène, C., & Saison-Behmoaras, T. E. (1999) · Journal of Molecular Biology · doi:10.1006/jmbi.1999.3277 · Cited by 96

Clinical Applications of Single-Stranded Oligonucleotides: Current Landscape of Approved and In-Development Therapeutics

Scharner, J., & Aznarez, I. (2020) · Molecular Therapy · doi:10.1016/j.ymthe.2020.12.022 · Cited by 96

Effects of diamond-FET-based RNA aptamer sensing for detection of real sample of HIV-1 Tat protein

Ruslinda, A. R., Tanabe, K., Ibori, S., Wang, X., & Kawarada, H. (2012) · Biosensors and Bioelectronics · doi:10.1016/j.bios.2012.07.048 · Cited by 95

Eteplirsen Treatment Attenuates Respiratory Decline in Ambulatory and Non-Ambulatory Patients with Duchenne Muscular Dystrophy

Khan, N., Eliopoulos, H., Han, L., Kinane, T. B., Lowes, L. P., Mendell, J. R., Gordish‐Dressman, H., Henricson, E., McDonald, C. M., & on behalf of the Eteplirsen Investigators and the CINRG DNHS Investigators (2019) · Journal of Neuromuscular Diseases · doi:10.3233/jnd-180351 · Cited by 95

VEGF siRNA Delivery System Using Arginine-Grafted Bioreducible Poly(disulfide amine)

Kim, S. H., Jeong, J. H., Kim, T., Kim, S. W., & Bull, D. A. (2008) · Molecular Pharmaceutics · doi:10.1021/mp800161e · Cited by 94

Mipomersen and its use in familial hypercholesterolemia

Parham, J. S., & Goldberg, A. C. (2019) · Expert Opinion on Pharmacotherapy · doi:10.1080/14656566.2018.1550071 · Cited by 94

Enlisting the mRNA Vaccine Platform to Combat Parasitic Infections

Versteeg, L., Almutairi, M. M., Hotez, P. J., & Pollet, J. B. K. (2019) · Vaccines · doi:10.3390/vaccines7040122 · Cited by 94

Viral Vectors for COVID-19 Vaccine Development

Lundstrom, K. H. (2021) · Viruses · doi:10.3390/v13020317 · Cited by 94

Tivanisiran, a novel siRNA for the treatment of dry eye disease

Moreno‐Montañés, J., Bleau, A., & Jiménez, A. I. (2018) · Expert Opinion on Investigational Drugs · doi:10.1080/13543784.2018.1457647 · Cited by 93

Exon-Skipping Antisense Oligonucleotides to Correct Missplicing in Neurogenetic Diseases

Siva, K., Covello, G., & Denti, M. A. (2014) · Nucleic Acid Therapeutics · doi:10.1089/nat.2013.0461 · Cited by 92

Inotersen: new promise for the treatment of hereditary transthyretin amyloidosis

Mathew, V., & Wang, A. K. (2019) · Drug Design Development and Therapy · doi:10.2147/dddt.s162913 · Cited by 91

Removal of HIV DNA by CRISPR from Patient Blood Engrafts in Humanized Mice

Bella, R., Kaminski, R., Mancuso, P., Young, W., Chen, C., Sariyer, R., Fischer, T., Amini, S., Ferrante, P., Jacobson, J. M., et al. (2018) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2018.05.021 · Cited by 90

How COVID unlocked the power of RNA vaccines

Dolgin, E. (2021) · Nature · doi:10.1038/d41586-021-00019-w · Cited by 90

Toxicological and Pharmacokinetic Properties of QPI-1007, a Chemically Modified Synthetic siRNA Targeting Caspase 2 mRNA, Following Intravitreal Injection

Solano, E. C. R., Kornbrust, D. J., Beaudry, A. A., Foy, J. W.-D., Schneider, D. J., & Thompson, J. D. (2014) · Nucleic Acid Therapeutics · doi:10.1089/nat.2014.0489 · Cited by 87

Acute hepatic porphyrias: Current diagnosis & management

Anderson, K. E. (2019) · Molecular Genetics and Metabolism · doi:10.1016/j.ymgme.2019.07.002 · Cited by 87

Replicon RNA Viral Vectors as Vaccines

Lundstrom, K. H. (2016) · Vaccines · doi:10.3390/vaccines4040039 · Cited by 86

CRISPR-Cas based antiviral strategies against HIV-1

Wang, G., Zhao, N., Berkhout, B., & Das, A. T. (2017) · Virus Research · doi:10.1016/j.virusres.2017.07.020 · Cited by 86

Lipid Polymer Hybrid Nanomaterials for mRNA Delivery

Zhao, W., Zhang, C., Li, B., Zhang, X., Luo, X. L., Zeng, C., Li, W., Gao, M., & Dong, Y. (2018) · Cellular and Molecular Bioengineering · doi:10.1007/s12195-018-0536-9 · Cited by 86

Tumor Regression in Mice by Delivery of Bcl-2 Small Interfering RNA with Pegylated Cationic Liposomes

Sonoke, S., Ueda, T., Fujiwara, K., Sato, Y., Takagaki, K., Hirabayashi, K., Ohgi, T., & Yano, J. (2008) · Cancer Research · doi:10.1158/0008-5472.can-08-0127 · Cited by 85

Long-term treatment with eteplirsen in nonambulatory patients with Duchenne muscular dystrophy

Alfano, L. N., Charleston, J. S., Connolly, A. M., Cripe, L. H., Donoghue, C., Dracker, R. A., Dworzak, J., Eliopoulos, H., Frank, D. E., Lewis, S., et al. (2019) · Medicine · doi:10.1097/md.0000000000015858 · Cited by 85

Comparison of the efficacy of MOE and PMO modifications of systemic antisense oligonucleotides in a severe SMA mouse model

Sheng, L., Rigo, F., Bennett, C. F., Krainer, A. R., & Hua, Y. (2020) · Nucleic Acids Research · doi:10.1093/nar/gkaa126 · Cited by 84

CRISPR-Cas13a Inhibits HIV-1 Infection

Yin, L., Zhao, F., Sun, H., Wang, Z., Huang, Y., Zhu, W., Xu, F., Mei, S., Liu, X., Zhang, D., et al. (2020) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2020.05.030 · Cited by 82

Sampling a Biomarker of the Human Immunodeficiency Virus across a Synthetic Nanopore

Niedzwiecki, D. J., Iyer, R., Borer, P. N., & Movileanu, L. (2013) · ACS Nano · doi:10.1021/nn400125c · Cited by 81

Tegsedi (Inotersen): An Antisense Oligonucleotide Approved for the Treatment of Adult Patients with Hereditary Transthyretin Amyloidosis

Gales, L. (2019) · Pharmaceuticals · doi:10.3390/ph12020078 · Cited by 80

Volanesorsen in the Treatment of Familial Chylomicronemia Syndrome or Hypertriglyceridaemia: Design, Development and Place in Therapy

Esan, O., & Wierzbicki, A. S. (2020) · Drug Design Development and Therapy · doi:10.2147/dddt.s224771 · Cited by 80

Peptides Used in the Delivery of Small Noncoding RNA

Shukla, R., Qin, B. Y., & Cheng, K. (2014) · Molecular Pharmaceutics · doi:10.1021/mp500426r · Cited by 79

CRISPR/Cas9 System as an Agent for Eliminating Polyomavirus JC Infection

Wollebo, H. S., Bellizzi, A., Kaminski, R., Hu, W., White, M. K., & Khalili, K. (2015) · PLoS ONE · doi:10.1371/journal.pone.0136046 · Cited by 78

Targeted lipid nanoparticles for RNA therapeutics and immunomodulation in leukocytes

Veiga, N., Diesendruck, Y., & Peer, D. (2020) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2020.04.002 · Cited by 78

Kunjin Virus Replicon Vaccine Vectors Induce Protective CD8+T-Cell Immunity

Anraku, I., Harvey, T. J., Linedale, R., Gardner, J., Harrich, D., Suhrbier, A., & Khromykh, A. A. (2002) · Journal of Virology · doi:10.1128/jvi.76.8.3791-3799.2002 · Cited by 76

Reversion of Cold-Adapted Live Attenuated Influenza Vaccine into a Pathogenic Virus

Zhou, B., Meliopoulos, V., Wang, W., Lin, X., Stucker, K. M., Halpin, R., Stockwell, T. B., Schultz‐Cherry, S., & Wentworth, D. E. (2016) · Journal of Virology · doi:10.1128/jvi.00163-16 · Cited by 75

Quality of life outcomes in APOLLO, the phase 3 trial of the RNAi therapeutic patisiran in patients with hereditary transthyretin-mediated amyloidosis

Obici, L. P., Berk, J. L., González‐Duarte, A., Coelho, T., Gillmore, J. D., Schmidt, H., Schilling, M., Yamashita, T., Labeyrie, C., Brannagan, T. H., et al. (2020) · Amyloid · doi:10.1080/13506129.2020.1730790 · Cited by 75

RNA Interference as A Gene-Specific Approach for Molecular Medicine

Grünweller, A., & Hartmann, R. K. (2005) · Current Medicinal Chemistry · doi:10.2174/092986705774933489 · Cited by 72

Molecular basis of primary hyperoxaluria: clues to innovative treatments

Dindo, M., Conter, C., Oppici, E., Ceccarelli, V., Marinucci, L., & Cellini, B. (2018) · Urolithiasis · doi:10.1007/s00240-018-1089-z · Cited by 72

Leading RNA interference therapeutics part 2: silencing delta-aminolevulinic acid synthase 1, with a focus on givosiran

de Paula Brandão, P. R., Titze-de-Almeida, S. S., & Titze-de-Almeida, R. (2020) · Molecular Diagnosis & Therapy · doi:10.1007/s40291-019-00438-6 · Cited by 72

Multiple shRNAs expressed by an inducible pol II promoter can knock down the expression of multiple target genes

Xia, X., Zhou, H., & Xu, Z. (2006) · BioTechniques · doi:10.2144/000112198 · Cited by 71

Inhibition of hepatitis B virus replication via HBV DNA cleavage by Cas9 from Staphylococcus aureus

Liu, Y., Zhao, M., Gong, M., Xu, Y., Xie, C., Deng, H., Li, X., Wu, H., & Wang, Z. (2018) · Antiviral Research · doi:10.1016/j.antiviral.2018.02.011 · Cited by 71

Pseudomonas aeruginosa infection augments inflammation through miR-301b repression of c-Myb-mediated immune activation and infiltration

Li, X., He, S., Li, R., Zhou, X., Zhang, S., Yu, M., Ye, Y., Wang, Y., Huang, C., & Wu, M. (2016) · Nature Microbiology · doi:10.1038/nmicrobiol.2016.132 · Cited by 70

First-in-human phase I study of ISTH0036, an antisense oligonucleotide selectively targeting transforming growth factor beta 2 (TGF-β2), in subjects with open-angle glaucoma undergoing glaucoma filtration surgery

Pfeiffer, N., Voykov, B., Renieri, G., Bell, K. C., Richter, P., Weigel, M., Thieme, H., Wilhelm, B., Lorenz, K., Feindor, M., et al. (2017) · PLoS ONE · doi:10.1371/journal.pone.0188899 · Cited by 70

Immunogenicity and Protection Efficacy of a Naked Self-Replicating mRNA-Based Zika Virus Vaccine

Zhong, Z., Catani, J. P. P., Cafferty, S. M., Couck, L., Broeck, W. V. D., Gorlé, N., Vandenbroucke, R. E., Devriendt, B., Ulbert, S., Cnops, L., et al. (2019) · Vaccines · doi:10.3390/vaccines7030096 · Cited by 70

Antisense Oligonucleotides Promote Exon Inclusion and Correct the Common c.-32-13T>G GAA Splicing Variant in Pompe Disease

van der Wal, E., Bergsma, A. J., Pijnenburg, J. M., van der Ploeg, A. T., & Pijnappel, W. W. M. P. (2017) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2017.03.001 · Cited by 69

Viral Vectors Applied for RNAi-Based Antiviral Therapy

Lundstrom, K. H. (2020) · Viruses · doi:10.3390/v12090924 · Cited by 69

Polymer–Lipid Pharmaceutical Nanocarriers: Innovations by New Formulations and Production Technologies

Bochicchio, S., Lamberti, G., & Barba, A. A. (2021) · Pharmaceutics · doi:10.3390/pharmaceutics13020198 · Cited by 68

Antisense oligonucleotide eluforsen is safe and improves respiratory symptoms in F508DEL cystic fibrosis

Dřevı́nek, P., Pressler, T., Cipolli, M., Boeck, K. D., Schwarz, C., Bouisset, F., Boff, M., Henig, N. R., Paquette-Lamontagne, N., Montgomery, S., et al. (2019) · Journal of Cystic Fibrosis · doi:10.1016/j.jcf.2019.05.014 · Cited by 65

Liposomal Grb2 antisense oligodeoxynucleotide (BP1001) in patients with refractory or relapsed haematological malignancies: a single-centre, open-label, dose-escalation, phase 1/1b trial

Ohanian, M. N., Ashizawa, A. T., Garcia‐Manero, G., Pemmaraju, N., Kadia, T. M., Jabbour, E. J., Ravandi, F., Borthakur, G. M., Andreeff, M., Konopleva, M. Y., et al. (2018) · The Lancet Haematology · doi:10.1016/s2352-3026(18)30021-8 · Cited by 63

PLGA-based dual targeted nanoparticles enhance miRNA transfection efficiency in hepatic carcinoma

Cai, C., Xie, Y., Wu, L., Chen, X., Liu, H., Zhou, Y., Zou, H., Liu, D., Zhao, Y., Kong, X., et al. (2017) · Scientific Reports · doi:10.1038/srep46250 · Cited by 62

Receptor-targeted aptamer-siRNA conjugate-directed transcriptional regulation of HIV-1

Zhou, J., Lazar, D. C., Li, H., Xia, X., Satheesan, S., Charlins, P., O’Meally, D., Akkina, R., Saayman, S. M., Weinberg, M. S., et al. (2018) · Theranostics · doi:10.7150/thno.23085 · Cited by 61

Strand Invasion of Mixed-Sequence, Double-Helical B-DNA by γ-Peptide Nucleic Acids Containing G-Clamp Nucleobases under Physiological Conditions

Rapireddy, S., Bahal, R., & Ly, D. H. (2011) · Biochemistry · doi:10.1021/bi2002554 · Cited by 60

Inhibition of CCR5-Dependent HIV-1 Infection by Hairpin Ribozyme Gene Therapy against CC-Chemokine Receptor 5

Feng, Y., Leavitt, M. C., Tritz, R., Duarte, E. A., Kang, D. E., Mamounas, M. J., Gilles, P., Wong‐Staal, F., Kennedy, S. P., Merson, J. R., et al. (2000) · Virology · doi:10.1006/viro.2000.0536 · Cited by 60

A Simple Cytosine to G‐Clamp Nucleobase Substitution Enables Chiral γ‐PNAs to Invade Mixed‐Sequence Double‐Helical B‐form DNA

Chenna, V., Rapireddy, S., Sahu, B., Ausín, C., Pedroso, E., & Ly, D. H. (2008) · ChemBioChem · doi:10.1002/cbic.200800441 · Cited by 59

Quantum Dots-siRNA Nanoplexes for Gene Silencing in Central Nervous System Tumor Cells

Lin, G., Chen, T., Zou, J., Wang, Y., Wang, X., Li, J., Huang, Q., Fu, Z., Zhao, Y., Lin, M. C. M., et al. (2017) · Frontiers in Pharmacology · doi:10.3389/fphar.2017.00182 · Cited by 59

DNA vaccines

Coban, C., Kobiyama, K., Jounai, N., Tozuka, M., & Ishii, K. J. (2013) · Human Vaccines & Immunotherapeutics · doi:10.4161/hv.25893 · Cited by 58

Gene Silencing Activity of siRNA Molecules Containing Phosphorodithioate Substitutions

Yang, X., Sierant, M., Janicka, M., Pęczek, Ł., Martinez, C., Hassell, T., Li, N., Li, X., Wang, T., & Nawrot, B. (2012) · ACS Chemical Biology · doi:10.1021/cb300078e · Cited by 58

Enhanced Delivery and Potency of Self-Amplifying mRNA Vaccines by Electroporation in Situ

Cu, Y., Broderick, K. E., Banerjee, K., Hickman, J., Otten, G. R., Barnett, S. W., Kichaev, G., Sardesai, N. Y., Ulmer, J. B., & Geall, A. J. (2013) · Vaccines · doi:10.3390/vaccines1030367 · Cited by 58

Inhibition of Dengue Virus Infections in Cell Cultures and in AG129 Mice by a Small Interfering RNA Targeting a Highly Conserved Sequence

Stein, D. A., Perry, S. T., Buck, M. D., OEHMEN, C. S., Fischer, M. A., Poore, E. A., Smith, J. L., Lancaster, A. M., Hirsch, A. J., Slifka, M. K., et al. (2011) · Journal of Virology · doi:10.1128/jvi.05298-11 · Cited by 58

Screening of efficient polymers for siRNA delivery in a library of hydrophobically modified polyethyleneimines

Shen, W., Wang, H., Ling-hu, Y., Lv, J., Chang, H., & Cheng, Y. (2016) · Journal of Materials Chemistry B · doi:10.1039/c6tb01929c · Cited by 58

Biochemical properties of phosphonoacetate and thiophosphonoacetate oligodeoxyribonucleotides

Sheehan, D. M. (2003) · Nucleic Acids Research · doi:10.1093/nar/gkg439 · Cited by 57

Antisense oligonucleotide eluforsen improves CFTR function in F508del cystic fibrosis

Sermet‐Gaudelus, I., Clancy, J. P., Nichols, D. P., Nick, J. A., Boeck, K. D., Solomon, G. M., Mall, M. A., Bolognese, J. A., Bouisset, F., den Hollander, W., et al. (2018) · Journal of Cystic Fibrosis · doi:10.1016/j.jcf.2018.10.015 · Cited by 57

Investigating the Impact of Delivery System Design on the Efficacy of Self-Amplifying RNA Vaccines

Anderluzzi, G., Lou, G., Gallorini, S., Brazzoli, M., Johnson, R., O’Hagan, D. T., Baudner, B. C., & Perrie, Y. (2020) · Vaccines · doi:10.3390/vaccines8020212 · Cited by 57

Induction of Protective Immunity against Toxoplasma gondii in Mice by Nucleoside Triphosphate Hydrolase-II (NTPase-II) Self-amplifying RNA Vaccine Encapsulated in Lipid Nanoparticle (LNP)

Luo, F., Zheng, L., Hu, Y., Liu, S., Wang, Y., Xiong, Z., Hu, X., & Tan, F. (2017) · Frontiers in Microbiology · doi:10.3389/fmicb.2017.00605 · Cited by 56

Enhancing the therapeutic delivery of oligonucleotides by chemical modification and nanoparticle encapsulation

Sun, Y., Zhao, Y., Zhao, X., Lee, R. J., Teng, L., & Zhou, C. (2017) · Molecules · doi:10.3390/molecules22101724 · Cited by 56

Advancements in Nucleic Acid Based Therapeutics against Respiratory Viral Infections

Asha, K., Kumar, P., Sanicas, M., Meseko, C. A., Khanna, M., & Kumar, B. (2018) · Journal of Clinical Medicine · doi:10.3390/jcm8010006 · Cited by 56

Design of in vitro Transcribed mRNA Vectors for Research and Therapy

Tusup, M., French, L. E., Matos, M. D., Gatfield, D., Kündig, T. M., & Pascolo, S. (2019) · CHIMIA International Journal for Chemistry · doi:10.2533/chimia.2019.391 · Cited by 55

Self-Replicating RNA

Tews, B. A., & Meyers, G. (2016) · Methods in molecular biology · doi:10.1007/978-1-4939-6481-9_2 · Cited by 54

Protective Efficacy of Nucleic Acid Vaccines Against Transmission of Zika Virus During Pregnancy in Mice

Jagger, B. W., Dowd, K. A., Chen, R. E., Desai, P., Foreman, B., Burgomaster, K. E., Himansu, S., Kong, W.-P., Graham, B. S., Pierson, T. C., et al. (2019) · The Journal of Infectious Diseases · doi:10.1093/infdis/jiz338 · Cited by 53

Gene editing and RNAi approaches for COVID-19 diagnostics and therapeutics

Berber, B., Aydın, C., Kocabaş, F., Güney-Esken, G., Yılancıoğlu, K., Alpaslan, M. K., Çalıseki, M., Yüce, M., Demir, S., & Taştan, C. (2020) · Gene Therapy · doi:10.1038/s41434-020-00209-7 · Cited by 52

iHIVARNA phase IIa, a randomized, placebo-controlled, double-blinded trial to evaluate the safety and immunogenicity of iHIVARNA-01 in chronically HIV-infected patients under stable combined antiretroviral therapy

de Jong, W., Aerts, J. L., Allard, S. D., Brander, C., Buyze, J., Florence, É., Gorp, E. C. M. V., Vanham, G. L., Leal, L., Mothe, B., et al. (2019) · Trials · doi:10.1186/s13063-019-3409-1 · Cited by 51

Antisense Inhibition of Glucagon Receptor by IONIS-GCGRRx Improves Type 2 Diabetes Without Increase in Hepatic Glycogen Content in Patients With Type 2 Diabetes on Stable Metformin Therapy

Morgan, E. S., Tai, L.-J., Pham, N. C., Overman, J. K., Watts, L. M., Smith, A. V., Jung, S. W., Gajdošík, M., Krššák, M., Krebs, M., et al. (2019) · Diabetes Care · doi:10.2337/dc18-1343 · Cited by 50

Former orphan riboswitches reveal unexplored areas of bacterial metabolism, signaling, and gene control processes

Sherlock, M. E., & Breaker, R. R. (2020) · RNA · doi:10.1261/rna.074997.120 · Cited by 50

RNA-based adjuvant CV8102 enhances the immunogenicity of a licensed rabies vaccine in a first-in-human trial

Doener, F., Hong, H. S., Meyer, I., Tadjalli-Mehr, K., Daehling, A., Heidenreich, R., Koch, S. D., Fotin-Mleczek, M., & Gnad-Vogt, U. (2019) · Vaccine · doi:10.1016/j.vaccine.2019.02.024 · Cited by 48

Hairpin ribozymes in combination with siRNAs against highly conserved hepatitis C virus sequence inhibit RNA replication and protein translation from hepatitis C virus subgenomic replicons

Jarczak, D., Korf, M., Beger, C., Manns, M. P., & Krüger, M. (2005) · FEBS Journal · doi:10.1111/j.1742-4658.2005.04986.x · Cited by 47

Electroporation Knows No Boundaries: The Use of Electrostimulation for siRNA Delivery in Cells and Tissues

Luft, C., & Ketteler, R. (2015) · SLAS DISCOVERY · doi:10.1177/1087057115579638 · Cited by 47

The role of follicular helper T cells and the germinal center in HIV-1 gp120 DNA prime and gp120 protein boost vaccination

Hollister, K., Chen, Y., Wang, S., Wu, H., Mondal, A., Clegg, N., Lu, S., & Dent, A. L. (2014) · Human Vaccines & Immunotherapeutics · doi:10.4161/hv.28659 · Cited by 44

Use of Single-Chain Antibody Derivatives for Targeted Drug Delivery

Safdari, Y., Ahmadzadeh, V., Khalili, M., Jaliani, H. Z., Zarei, V., & Moghadam, V. E. (2016) · Molecular Medicine · doi:10.2119/molmed.2016.00043 · Cited by 44

Ribozymes in the Age of Molecular Therapeutics

Bagheri, S., & Kashani–Sabet, M. (2004) · Current Molecular Medicine · doi:10.2174/1566524043360410 · Cited by 43

The Role of mRNA Stability in the Control of Globin Gene Expression

Russell, J., Morales, J., & Liebhaber, S. A. (1997) · Progress in nucleic acid research and molecular biology · doi:10.1016/s0079-6603(08)60283-4 · Cited by 42

Familial amyloidotic polyneuropathy: current and emerging treatment options for transthyretin-mediated amyloidosis

Hund, E. (2012) · The Application of Clinical Genetics · doi:10.2147/tacg.s19903 · Cited by 41

Development of RNA aptamer that inhibits methyltransferase activity of dengue virus

Jung, J. I., Han, S. R., & Lee, S. (2017) · Biotechnology Letters · doi:10.1007/s10529-017-2462-7 · Cited by 41

A randomized controlled clinical trial of intravitreous fomivirsen for treatment of newly diagnosed peripheral cytomegalovirus retinitis in patients with AIDS

Belfort, R., Da, G., Johnson, D. W., Mansour, S., Muccioli, C., Sheppard, J., Mora-Durate, J., Chan, C., Palestine, A., Perez, J. E. A., et al. (2002) · UCL Discovery (University College London) · Cited by 41

Evaluation of a locked nucleic acid form of antisense oligo targeting HIF-1α in advanced hepatocellular carcinoma

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