From bench side to clinic: Potential and challenges of RNA vaccines and therapeutics in infectious diseases
Abstract
The functional and structural versatility of Ribonucleic acids (RNAs) makes them ideal candidates for overcoming the limitations imposed by small molecule-based drugs. Hence, RNA-based biopharmaceuticals such as messenger RNA (mRNA) vaccines, antisense oligonucleotides (ASOs), small interfering RNAs (siRNAs), microRNA mimics, anti-miRNA oligonucleotides (AMOs), aptamers, riboswitches, and CRISPR-Cas9 are emerging as vital tools for the treatment and prophylaxis of many infectious diseases. Some of the major challenges to overcome in the area of RNA-based therapeutics have been the instability of single-stranded RNAs, delivery to the diseased cell, and immunogenicity. However, recent advancements in the delivery systems of in vitro transcribed mRNA and chemical modifications for protection against nucleases and reducing the toxicity of RNA have facilitated the entry of several exogenous RNAs into clinical trials. In this review, we provide an overview of RNA-based vaccines and therapeutics, their production, delivery, current advancements, and future translational potential in treating infectious diseases.
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Unmodified mRNAs can trigger inflammation and immune reactions when introduced into the body as foreign invaders, thus compromising efficacy and safety.
“Unmodified mRNAs can trigger inflammation and immune reactions when introduced into the body as foreign invaders, thus compromising efficacy and safety [52].”
From Exosome for mRNA delivery: Strategies and therapeutic applications (2024) · cites this paper as [52]
Cites
Bartel (2004) · doi:10.1016/s0092-8674(04)00045-5 · Cited by 34,714 · see citing works
A Programmable Dual-RNA–Guided DNA Endonuclease in Adaptive Bacterial Immunity
Jinek, M., Chylinski, K., Fonfara, I., Hauer, M., Doudna, J. A., & Charpentier, E. (2012) · Science · doi:10.1126/science.1225829 · Cited by 17,116 · see citing works
Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans
Fire, A., Xu, S., Montgomery, M. K., Kostas, S. A., Driver, S. E., & Mello, C. C. (1998) · Nature · doi:10.1038/35888 · Cited by 15,429 · see citing works
Safety and Efficacy of the BNT162b2 mRNA Covid-19 Vaccine
Polack, F. P., Thomas, S. J., Kitchin, N., Absalon, J., Gurtman, A., Lockhart, S., Perez, J. L., Pérez Marc, G., Moreira, E. D., Zerbini, C., et al. (2020) · New England Journal of Medicine · doi:10.1056/nejmoa2034577 · Cited by 14,592 · see citing works
The C. elegans heterochronic gene lin-4 encodes small RNAs with antisense complementarity to lin-14
Lee, R. C., Feinbaum, R. L., & Ambros, V. (1993) · Cell · doi:10.1016/0092-8674(93)90529-y · Cited by 12,948 · see citing works
In vitro selection of RNA molecules that bind specific ligands
Ellington, A. D., & Szostak, J. W. (1990) · Nature · doi:10.1038/346818a0 · Cited by 9,845 · see citing works
Duplexes of 21-nucleotide RNAs mediate RNA interference in cultured mammalian cells
Elbashir, S. M., Harborth, J., Lendeckel, W., Yalcin, A., Weber, K., & Tuschl, T. (2001) · Nature · doi:10.1038/35078107 · Cited by 9,456 · see citing works
Most mammalian mRNAs are conserved targets of microRNAs
Friedman, R. C., Farh, K. K.-H., Burge, C. B., & Bartel, D. P. (2009) · Genome Research · doi:10.1101/gr.082701.108 · Cited by 8,462 · see citing works
mRNA vaccines — a new era in vaccinology
Pardi, N., Hogan, M. J., Porter, F. W., & Weissman, D. (2018) · Nature Reviews Drug Discovery · doi:10.1038/nrd.2017.243 · Cited by 4,667 · see citing works
Wightman, B., Ha, I., & Ruvkun, G. (1993) · Cell · doi:10.1016/0092-8674(93)90530-4 · Cited by 4,292 · see citing works
Direct Gene Transfer into Mouse Muscle in Vivo
Wolff, J. A., Malone, R. W., Williams, P., Chong, W., Acsadi, G., Jani, A., & Felgner, P. L. (1990) · Science · doi:10.1126/science.1690918 · Cited by 4,173 · see citing works
Silencing of microRNAs in vivo with ‘antagomirs’
Krützfeldt, J., Rajewsky, N., Braich, R., Rajeev, K. G., Tuschl, T., Manoharan, M., & Stoffel, M. (2005) · Nature · doi:10.1038/nature04303 · Cited by 3,949 · see citing works
PLGA-based nanoparticles: An overview of biomedical applications
Danhier, F., Ansorena, E., Silva, J. M., Coco, R., Breton, A. L., & Véronique Préat, A. (2012) · Journal of Controlled Release · doi:10.1016/j.jconrel.2012.01.043 · Cited by 3,884 · see citing works
CRISPR-Mediated Modular RNA-Guided Regulation of Transcription in Eukaryotes
Gilbert, L. A., Larson, M. H., Morsut, L., Liu, Z., Brar, G. A., Torres, S. E., Stern-Ginossar, N., Brandman, O., Whitehead, E. H., Doudna, J. A., et al. (2013) · Cell · doi:10.1016/j.cell.2013.06.044 · Cited by 3,696 · see citing works
Hopkins, A. L., & Groom, C. R. (2002) · Nature Reviews Drug Discovery · doi:10.1038/nrd892 · Cited by 3,305 · see citing works
An mRNA Vaccine against SARS-CoV-2 — Preliminary Report
Jackson, L. A., Anderson, E. J., Rouphael, N. G., Roberts, P. C., Makhene, M., Coler, R. N., McCullough, M. P., Chappell, J. D., Denison, M. R., Stevens, L. J., et al. (2020) · New England Journal of Medicine · doi:10.1056/nejmoa2022483 · Cited by 3,237 · see citing works
Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis
Adams, D., Gonzalez-Duarte, A., O’Riordan, W. D., Yang, C.-C., Ueda, M., Kristen, A. V., Tournev, I., Schmidt, H. H., Coelho, T., Berk, J. L., et al. (2018) · New England Journal of Medicine · doi:10.1056/nejmoa1716153 · Cited by 3,047 · see citing works
The RNA moiety of ribonuclease P is the catalytic subunit of the enzyme
Guerrier-Takada, C., Gardiner, K., Marsh, T., Pace, N., & Altman, S. (1983) · Cell · doi:10.1016/0092-8674(83)90117-4 · Cited by 2,684 · see citing works
Zamore, P. D., Tuschl, T., Sharp, P. A., & Bartel, D. P. (2000) · Cell · doi:10.1016/s0092-8674(00)80620-0 · Cited by 2,667 · see citing works
CRISPR RNA Maturation by Trans-Encoded Small RNA and Host Factor RNase III
Deltcheva, E., Chylinski, K., Sharma, C. M., Gonzales, K., Chao, Y., Pirzada, Z. A., Eckert, M. R., Vogel, J., & Charpentier, E. (2011) · Nature · doi:10.1038/nature09886 · Cited by 2,563 · see citing works
mRNA-based therapeutics--developing a new class of drugs
Sahin, U., Karikó, K., & Türeci, Ö. (2014) · Nature Reviews Drug Discovery · doi:10.1038/nrd4278 · Cited by 2,474 · see citing works
Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy
Mendell, J. R., Al-Zaidy, S., Shell, R., Arnold, W. D., Rodino-Klapac, L. R., Prior, T. W., Lowes, L., Alfano, L., Berry, K., Church, K., et al. (2017) · New England Journal of Medicine · doi:10.1056/nejmoa1706198 · Cited by 2,381 · see citing works
Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy
Finkel, R. S., Mercuri, E., Darras, B. T., Connolly, A. M., Kuntz, N. L., Kirschner, J., Chiriboga, C. A., Saito, K., Servais, L., Tizzano, E., et al. (2017) · New England Journal of Medicine · doi:10.1056/nejmoa1702752 · Cited by 2,337 · see citing works
Kruger, K., Grabowski, P. J., Zaug, A. J., Sands, J., Gottschling, D. E., & Cech, T. R. (1982) · Cell · doi:10.1016/0092-8674(82)90414-7 · Cited by 2,331 · see citing works
Asymmetry in the Assembly of the RNAi Enzyme Complex
Schwarz, D. S., Hutvágner, G., Du, T., Xu, Z., Aronin, N., & Zamore, P. D. (2003) · Cell · doi:10.1016/s0092-8674(03)00759-1 · Cited by 2,323 · see citing works
The Noncoding RNA Revolution—Trashing Old Rules to Forge New Ones
Cech, T. R., & Steitz, J. A. (2014) · Cell · doi:10.1016/j.cell.2014.03.008 · Cited by 2,270 · see citing works
Treatment of HCV Infection by Targeting MicroRNA
Janssen, H. L. A., Reesink, H. W., Lawitz, E., Zeuzem, S., Rodriguez‐Torres, M., Patel, K., van der Meer, A. J., Patick, A. K., Chen, A. P., Zhou, Y., et al. (2013) · New England Journal of Medicine · doi:10.1056/nejmoa1209026 · Cited by 2,206 · see citing works
Therapeutic silencing of an endogenous gene by systemic administration of modified siRNAs
Soutschek, J., Akinc, A., Bramlage, B., Charissé, K., Constien, R., Donoghue, M., Elbashir, S. M., Geick, A., Hadwiger, P., Harborth, J., et al. (2004) · Nature · doi:10.1038/nature03121 · Cited by 2,189 · see citing works
Rational siRNA design for RNA interference
Reynolds, A., Leake, D., Boese, Q., Scaringe, S., Marshall, W. S., & Khvorova, A. (2004) · Nature Biotechnology · doi:10.1038/nbt936 · Cited by 2,094 · see citing works
miR-122 regulation of lipid metabolism revealed by in vivo antisense targeting
Esau, C., Davis, S., Murray, S. F., Yu, X. X., Pandey, S. K., Pear, M., Watts, L., Booten, S. L., Graham, M., McKay, R., et al. (2006) · Cell Metabolism · doi:10.1016/j.cmet.2006.01.005 · Cited by 2,082 · see citing works
CRISPR–Cas9 Structures and Mechanisms
Jiang, F., & Doudna, J. A. (2017) · Annual Review of Biophysics · doi:10.1146/annurev-biophys-062215-010822 · Cited by 2,029 · see citing works
Karikó, K., Muramatsu, H., Welsh, F. A., Ludwig, J., Kato, H., Akira, S., & Weissman, D. (2008) · Molecular Therapy · doi:10.1038/mt.2008.200 · Cited by 1,930 · see citing works
An overview of the antimicrobial resistance mechanisms of bacteria
Reygaert, W. C. (2018) · AIMS Microbiology · doi:10.3934/microbiol.2018.3.482 · Cited by 1,914 · see citing works
Hilton, I. B., D’Ippolito, A. M., Vockley, C. M., Thakore, P. I., Crawford, G. E., Reddy, T. E., & Gersbach, C. A. (2015) · Nature Biotechnology · doi:10.1038/nbt.3199 · Cited by 1,879 · see citing works
Zamecnik, P. C., & Stephenson, M. L. (1978) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.75.1.280 · Cited by 1,792 · see citing works
Li, H., Yang, Y., Hong, W., Huang, M., Wu, M., & Zhao, X. (2020) · Signal Transduction and Targeted Therapy · doi:10.1038/s41392-019-0089-y · Cited by 1,771 · see citing works
Two Phase 3 Trials of Inclisiran in Patients with Elevated LDL Cholesterol
Ray, K. K., Wright, R. S., Kallend, D., Koenig, W., Leiter, L. A., Raal, F. J., Bisch, J. A., Richardson, T., Jaros, M., Wijngaard, P. L. J., et al. (2020) · New England Journal of Medicine · doi:10.1056/nejmoa1912387 · Cited by 1,665 · see citing works
Therapeutic Silencing of MicroRNA-122 in Primates with Chronic Hepatitis C Virus Infection
Lanford, R. E., Hildebrandt-Eriksen, E. S., Petri, A., Persson, R., Lindow, M., Munk, M. E., Kauppinen, S., & Ørum, H. (2009) · Science · doi:10.1126/science.1178178 · Cited by 1,654 · see citing works
The Onpattro story and the clinical translation of nanomedicines containing nucleic acid-based drugs
Akinc, A., Maier, M. A., Manoharan, M., Fitzgerald, K., Jayaraman, M., Barros, S., Ansell, S., Du, X., Hope, M. J., Madden, T. D., et al. (2019) · Nature Nanotechnology · doi:10.1038/s41565-019-0591-y · Cited by 1,636 · see citing works
A guide to vaccinology: from basic principles to new developments
Pollard, A. J., & Bijker, E. M. (2021) · Nature Reviews Immunology · doi:10.1038/s41577-020-00479-7 · Cited by 1,593 · see citing works
Inotersen Treatment for Patients with Hereditary Transthyretin Amyloidosis
Benson, M. D., Waddington‐Cruz, M., Berk, J. L., Polydefkis, M. J., Dyck, P. J. B., Wang, A. K., Planté‐Bordeneuve, V., Barroso, F. A., Merlini, G., Obici, L. P., et al. (2018) · New England Journal of Medicine · doi:10.1056/nejmoa1716793 · Cited by 1,452 · see citing works
Viral vectors for gene therapy: the art of turning infectious agents into vehicles of therapeutics
Kay, M., Glorioso, J., & Naldini, L. (2001) · Nature Medicine · doi:10.1038/83324 · Cited by 1,444 · see citing works
The current state and future directions of RNAi-based therapeutics
Setten, R. L., Rossi, J. J., & Han, S.-P. (2019) · Nature Reviews Drug Discovery · doi:10.1038/s41573-019-0017-4 · Cited by 1,443 · see citing works
Bennett, C. F., & Swayze, E. E. (2010) · The Annual Review of Pharmacology and Toxicology · doi:10.1146/annurev.pharmtox.010909.105654 · Cited by 1,381 · see citing works
Antisense Oligonucleotides as Therapeutic Agents—Is the Bullet Really Magical?
Stein, C., & Cheng, Y. C. (1993) · Science · doi:10.1126/science.8351515 · Cited by 1,303 · see citing works
An Unstable Intermediate Carrying Information from Genes to Ribosomes for Protein Synthesis
Brenner, S., Jacob, F., & Meselson, M. S. (1961) · Nature · doi:10.1038/190576a0 · Cited by 1,235 · see citing works
Editing DNA Methylation in the Mammalian Genome
Liu, X. S., Wu, H., Ji, X., Stelzer, Y., Wu, X., Czauderna, S., Shu, J., Dadon, D., Young, R. A., & Jaenisch, R. (2016) · Cell · doi:10.1016/j.cell.2016.08.056 · Cited by 1,224 · see citing works
Phage Response to CRISPR-Encoded Resistance in Streptococcus thermophilus
Deveau, H., Barrangou, R., Garneau, J. E., Labonté, J., Fremaux, C., Boyaval, P., Romero, D. A., Horvath, P., & Moineau, S. (2007) · Journal of Bacteriology · doi:10.1128/jb.01412-07 · Cited by 1,196 · see citing works
Potent and persistent in vivo anti-HBV activity of chemically modified siRNAs
Morrissey, D. V., Lockridge, J. A., Shaw, L., Blanchard, K. S., Jensen, K., Breen, W., Hartsough, K. C., Machemer, L., Radka, S. F., Jadhav, V., et al. (2005) · Nature Biotechnology · doi:10.1038/nbt1122 · Cited by 1,168 · see citing works
Overcoming cellular barriers for RNA therapeutics
Dowdy, S. F. (2017) · Nature Biotechnology · doi:10.1038/nbt.3802 · Cited by 1,139 · see citing works
Bikard, D., Jiang, W., Samai, P., Hochschild, A., Zhang, F., & Marraffini, L. A. (2013) · Nucleic Acids Research · doi:10.1093/nar/gkt520 · Cited by 1,111 · see citing works
Lipoprotein(a) Reduction in Persons with Cardiovascular Disease
Tsimikas, S., Karwatowska-Prokopczuk, E., Gouni-Berthold, I., Tardif, J.-C., Baum, S. J., Steinhagen-Thiessen, E., Shapiro, M. D., Stroes, E. S., Moriarty, P. M., Nordestgaard, B. G., et al. (2020) · New England Journal of Medicine · doi:10.1056/nejmoa1905239 · Cited by 1,094 · see citing works
RNAi therapeutics: a potential new class of pharmaceutical drugs
Bumcrot, D. A., Manoharan, M., Koteliansky, V., & Sah, D. W. Y. (2006) · Nature Chemical Biology · doi:10.1038/nchembio839 · Cited by 1,086 · see citing works
mRNA is an endogenous ligand for toll-like receptor 3
Karikó, K., Ni, H., Capodici, J., Lamphier, M., & Weissman, D. (2004) · Journal of Biological Chemistry · doi:10.1074/jbc.m310175200 · Cited by 1,062 · see citing works
Non-coding RNAs as drug targets
Matsui, M., & Corey, D. R. (2016) · Nature Reviews Drug Discovery · doi:10.1038/nrd.2016.117 · Cited by 1,036 · see citing works
Finkel, R. S., Chiriboga, C. A., Vajsar, J., Day, J., Montes, J., Vivo, D. C. D., Yamashita, M., Rigo, F., Hung, G., Schneider, E., et al. (2016) · The Lancet · doi:10.1016/s0140-6736(16)31408-8 · Cited by 1,019 · see citing works
Feldkötter, M., Schwarzer, V., Wirth, R., Wienker, T. F., & Wirth, B. (2002) · The American Journal of Human Genetics · doi:10.1086/338627 · Cited by 1,005 · see citing works
Applications of CRISPR technologies in research and beyond
Barrangou, R., & Doudna, J. A. (2016) · Nature Biotechnology · doi:10.1038/nbt.3659 · Cited by 1,005 · see citing works
Safety and efficacy of RNAi therapy for transthyretin amyloidosis
Coelho, T., Adams, D., Silva, A., Lozeron, P., Hawkins, P. N., Mant, T., Perez, J., Chiesa, J., Warrington, S., Tranter, E., et al. (2013) · New England Journal of Medicine · doi:10.1056/nejmoa1208760 · Cited by 971 · see citing works
Passenger-Strand Cleavage Facilitates Assembly of siRNA into Ago2-Containing RNAi Enzyme Complexes
Matranga, C., Tomari, Y., Shin, C., Bartel, D. P., & Zamore, P. D. (2005) · Cell · doi:10.1016/j.cell.2005.08.044 · Cited by 969 · see citing works
Monani, U. R. (1999) · Human Molecular Genetics · doi:10.1093/hmg/8.7.1177 · Cited by 969 · see citing works
Koenig, M., Beggs, A. H., Moyer, M., Scherpf, S., Heindrich, K., Bettecken, T., Meng, G., Müller, C. R., Lindlöf, M., Kääriäinen, H. A., et al. (1989) · Europe PMC (PubMed Central) · Cited by 958 · see citing works
RNA-Based Therapeutics: Current Progress and Future Prospects
Burnett, J., & Rossi, J. J. (2012) · Chemistry & Biology · doi:10.1016/j.chembiol.2011.12.008 · Cited by 910 · see citing works
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.2017.02.017 · Cited by 909 · see citing works
Schiffelers, R. M. (2004) · Nucleic Acids Research · doi:10.1093/nar/gnh140 · Cited by 900 · see citing works
Synthetic dsRNA Dicer substrates enhance RNAi potency and efficacy
Kim, D.-H., Behlke, M. A., Rose, S. D., Chang, M.-S., Choi, S., & Rossi, J. J. (2004) · Nature Biotechnology · doi:10.1038/nbt1051 · Cited by 884 · see citing works
The cap and poly(A) tail function synergistically to regulate mRNA translational efficiency.
Gallie, D. R. (1991) · Genes & Development · doi:10.1101/gad.5.11.2108 · Cited by 868 · see citing works
Patient-Customized Oligonucleotide Therapy for a Rare Genetic Disease
Kim, J., Hu, C., Achkar, C. M. E., Black, L. E., Douville, J., Larson, A. A., Pendergast, M. K., Goldkind, S. F., Lee, E. A., Kuniholm, A., et al. (2019) · New England Journal of Medicine · doi:10.1056/nejmoa1813279 · Cited by 835 · see citing works
Inclisiran for the Treatment of Heterozygous Familial Hypercholesterolemia
Raal, F. J., Kallend, D., Ray, K. K., Turner, T., Koenig, W., Wright, R. S., Wijngaard, P. L. J., Curcio, D., Jaros, M. J., Leiter, L. A., et al. (2020) · New England Journal of Medicine · doi:10.1056/nejmoa1913805 · Cited by 818 · see citing works
CIRCLE-Seq: A highly sensitive in vitro screen for genome-wide CRISPR-cas9 nuclease off-targets
Tsai, S. Q., Nguyen, N. T., Malagon-Lopez, J., Topkar, V. V., Aryee, M. J., & Joung, J. K. (2017) · Nature Methods · doi:10.1038/nmeth.4278 · Cited by 814 · see citing works
Position-specific chemical modification of siRNAs reduces “off-target” transcript silencing
Jackson, A. L., Burchard, J., Leake, D., Reynolds, A., Schelter, J. M., Guo, J., Johnson, J. M., Lim, L. P., Karpilow, J. M., Nichols, K. E., et al. (2006) · RNA · doi:10.1261/rna.30706 · Cited by 790 · see citing works
Repurposing the CRISPR-Cas9 system for targeted DNA methylation
Vojta, A., Dobrinić, P., Tadić, V., Bočkor, L., Korać, P., Jülg, B. D., Klasić, M., & Zoldoš, V. (2016) · Nucleic Acids Research · doi:10.1093/nar/gkw159 · Cited by 772 · see citing works
Hua, Y., Sahashi, K., Rigo, F., Hung, G., Horev, G., Bennett, C. F., & Krainer, A. R. (2011) · Nature · doi:10.1038/nature10485 · Cited by 744 · see citing works
Therapeutic miRNA and siRNA: Moving from Bench to Clinic as Next Generation Medicine
Chakraborty, C., Sharma, A. R., Sharma, G., Doss, C. G. P., & Lee, S. (2017) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2017.06.005 · Cited by 731 · see citing works
GalNAc-siRNA conjugates: leading the way for delivery of RNAi therapeutics
Springer, A. D., & Dowdy, S. F. (2018) · Nucleic Acid Therapeutics · doi:10.1089/nat.2018.0736 · Cited by 711 · see citing works
Development of a gene-editing approach to restore vision loss in Leber congenital amaurosis type 10
Maeder, M. L., Stefanidakis, M., Wilson, C. J., Baral, R., Barrera, L. A., Bounoutas, G. S., Bumcrot, D., Chao, H., Ciulla, D. M., DaSilva, J. A., et al. (2019) · Nature Medicine · doi:10.1038/s41591-018-0327-9 · Cited by 707 · see citing works
Freier, S. (1997) · Nucleic Acids Research · doi:10.1093/nar/25.22.4429 · Cited by 692 · see citing works
Microfluidic Synthesis of Highly Potent Limit-size Lipid Nanoparticles for In Vivo Delivery of siRNA
Belliveau, N. M., Huft, J., Lin, P. J., Chen, S., Leung, A. K., Leaver, T. J., Wild, A. W., Lee, J. B., Taylor, R. J., Tam, Y. K., et al. (2012) · Molecular Therapy - Nucleic Acids · doi:10.1038/mtna.2012.28 · Cited by 685 · see citing works
Sensing Small Molecules by Nascent RNA
Mironov, A. S., Gusarov, I., Rafikov, R., Errais, L. L., Shatalin, K. Y., Kreneva, R. A., Perumov, D. A., & Nudler, E. (2002) · Cell · doi:10.1016/s0092-8674(02)01134-0 · Cited by 682 · see citing works
Advances in mRNA Vaccines for Infectious Diseases
Zhang, C., Maruggi, G., Shan, H., & Li, J. (2019) · Frontiers in Immunology · doi:10.3389/fimmu.2019.00594 · Cited by 680 · see citing works
Nonviral delivery of self-amplifying RNA vaccines
Geall, A. J., Verma, A., Otten, G. R., Shaw, C. A., Hekele, A., Banerjee, K., Cu, Y., Beard, C. W., Brito, L. A., Krucker, T., et al. (2012) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1209367109 · Cited by 675 · see citing works
Volanesorsen and Triglyceride Levels in Familial Chylomicronemia Syndrome
Witztum, J. L., Gaudet, D., Freedman, S. D., Alexander, V. J., Digenio, A., Williams, K. R., Yang, Q., Hughes, S. G., Geary, R. S., Arca, M., et al. (2019) · New England Journal of Medicine · doi:10.1056/nejmoa1715944 · Cited by 669 · see citing works
Functional annotation of native enhancers with a Cas9–histone demethylase fusion
Kearns, N. A., Pham, H., Tabak, B., Genga, R. M., Silverstein, N. J., Garber, M., & Maehr, R. (2015) · Nature Methods · doi:10.1038/nmeth.3325 · Cited by 656 · see citing works
Advances in the delivery of RNA therapeutics: from concept to clinical reality
Kaczmarek, J. C., Kowalski, P. S., & Anderson, D. G. (2017) · Genome Medicine · doi:10.1186/s13073-017-0450-0 · Cited by 656 · see citing works
Host Immune System Gene Targeting by a Viral miRNA
Stern‐Ginossar, N., Elefant, N., Zimmermann, A., Wolf, D. G., Saleh, N., Biton, M., Horwitz, E. P., Prokocimer, Z., Prichard, M. N., Hahn, G., et al. (2007) · Science · doi:10.1126/science.1140956 · Cited by 646 · see citing works
Chemistry, mechanism and clinical status of antisense oligonucleotides and duplex RNAs
Shen, X., & Corey, D. R. (2017) · Nucleic Acids Research · doi:10.1093/nar/gkx1239 · Cited by 639 · see citing works
Silencing of microRNA families by seed-targeting tiny LNAs
Obad, S., dos Santos, C. O., Petri, A., Heidenblad, M., Broom, O. J., Ruse, C. I., Fu, C., Lindow, M., Stenvang, J., Straarup, E. M., et al. (2011) · Nature Genetics · doi:10.1038/ng.786 · Cited by 627 · see citing works
Structural variations and stabilising modifications of synthetic siRNAs in mammalian cells
Czauderna, F. (2003) · Nucleic Acids Research · doi:10.1093/nar/gkg393 · Cited by 625 · see citing works
Familial Hypercholesterolemia: Screening, diagnosis and management of pediatric and adult patients
Goldberg, A. C., Hopkins, P. N., Toth, P. P., Ballantyne, C. M., Rader, D. J., Robinson, J. G., Daniels, S. R., Gidding, S. S., de Ferranti, S. D., Ito, M. K., et al. (2011) · Journal of clinical lipidology · doi:10.1016/j.jacl.2011.03.001 · Cited by 622 · see citing works
A Mouse Model of SARS-CoV-2 Infection and Pathogenesis
Sun, S.-H., Chen, Q., Gu, H.-J., Yang, G., Wang, Y.-X., Huang, X.-Y., Liu, S.-S., Zhang, N.-N., Li, X.-F., Xiong, R., et al. (2020) · Cell Host & Microbe · doi:10.1016/j.chom.2020.05.020 · Cited by 617 · see citing works
Prakash, T. P., Graham, M. J., Yu, J., Carty, R., Low, A., Chappell, A. E., Schmidt, K., Zhao, C., Aghajan, M., Murray, H. F., et al. (2014) · Nucleic Acids Research · doi:10.1093/nar/gku531 · Cited by 616 · see citing works
Nimjee, S. M., White, R. R., Becker, R. C., & Sullenger, B. A. (2017) · The Annual Review of Pharmacology and Toxicology · doi:10.1146/annurev-pharmtox-010716-104558 · Cited by 616 · see citing works
Patisiran: First Global Approval
Hoy, S. M. (2018) · Drugs · doi:10.1007/s40265-018-0983-6 · Cited by 616 · see citing works
Inhibition of hepatitis B virus in mice by RNA interference
McCaffrey, A. P., Nakai, H., Pandey, K., Huang, Z., Salazar, F. H., Xu, H., Wieland, S. F., Marion, P. L., & Kay, M. A. (2003) · Nature Biotechnology · doi:10.1038/nbt824 · Cited by 609 · see citing works
Phase 3 Trial of RNAi Therapeutic Givosiran for Acute Intermittent Porphyria
Balwani, M., Sardh, E., Ventura, P., Peiró, P. A., Rees, D. C., Stölzel, U., Bissell, D. M., Bonkovsky, H. L., Windyga, J., Anderson, K. E., et al. (2020) · New England Journal of Medicine · doi:10.1056/nejmoa1913147 · Cited by 608 · see citing works
Locked nucleic acid (LNA): fine-tuning the recognition of DNA and RNA
Braasch, D. A., & Corey, D. R. (2001) · Chemistry & Biology · doi:10.1016/s1074-5521(00)00058-2 · Cited by 604 · see citing works
Vollmer, J., Weeratna, R. D., Payette, P., Jurk, M., Schetter, C., Laucht, M., Wader, T., Tluk, S., Liu, M., Davis, H. L., et al. (2003) · European Journal of Immunology · doi:10.1002/eji.200324032 · Cited by 592 · see citing works
Dassie, J. P., Liu, X., Thomas, G. S., Whitaker, R. M., Thiel, K. W., Stockdale, K. R., Meyerholz, D. K., McCaffrey, A. P., McNamara, J. O., & Giangrande, P. H. (2009) · Nature Biotechnology · doi:10.1038/nbt.1560 · Cited by 591 · see citing works
Sequence-specific inhibition of small RNA function
Hutvágner, G., Simard, M. J., Mello, C. C., & Zamore, P. D. (2004) · PLoS Biology · doi:10.1371/journal.pbio.0020098 · Cited by 584 · see citing works
AAV-Mediated Gene Therapy for Research and Therapeutic Purposes
Samulski, R. J., & Muzyczka, N. (2014) · Annual Review of Virology · doi:10.1146/annurev-virology-031413-085355 · Cited by 582 · see citing works
New Vaccine Technologies to Combat Outbreak Situations
Rauch, S., Jasny, E., Schmidt, K. E., & Petsch, B. (2018) · Frontiers in Immunology · doi:10.3389/fimmu.2018.01963 · Cited by 579 · see citing works
Design of antisense oligonucleotides stabilized by locked nucleic acids
Kurreck, J. (2002) · Nucleic Acids Research · doi:10.1093/nar/30.9.1911 · Cited by 576 · see citing works
Hua, Y., Vickers, T. A., Okunola, H. L., Bennett, C. F., & Krainer, A. R. (2008) · The American Journal of Human Genetics · doi:10.1016/j.ajhg.2008.01.014 · Cited by 576 · see citing works
Harnessing the CRISPR/Cas9 system to disrupt latent HIV-1 provirus
Ebina, H., Misawa, N., Kanemura, Y., & Koyanagi, Y. (2013) · Scientific Reports · doi:10.1038/srep02510 · Cited by 565 · see citing works
Phase 1–2 Trial of Antisense Oligonucleotide Tofersen for <i>SOD1</i> ALS
Miller, T., Cudkowicz, M., Shaw, P. J., Andersen, P. M., Atassi, N., Bucelli, R. C., Genge, A., Glass, J., Ladha, S., Ludolph, A. L., et al. (2020) · New England Journal of Medicine · doi:10.1056/nejmoa2003715 · Cited by 565 · see citing works
Locked nucleic acid (LNA) mediated improvements in siRNA stability and functionality
Elmén, J. (2005) · Nucleic Acids Research · doi:10.1093/nar/gki193 · Cited by 563 · see citing works
Genome-wide CRISPR Screens Reveal Host Factors Critical for SARS-CoV-2 Infection
Wei, J., Alfajaro, M. M., DeWeirdt, P. C., Hanna, R. E., Lu-Culligan, W. J., Cai, W. L., Strine, M. S., Zhang, S.-M., Graziano, V. R., Schmitz, C. O., et al. (2020) · Cell · doi:10.1016/j.cell.2020.10.028 · Cited by 559 · see citing works
The Structural and Functional Diversity of Metabolite-Binding Riboswitches
Roth, A., & Breaker, R. R. (2009) · Annual Review of Biochemistry · doi:10.1146/annurev.biochem.78.070507.135656 · Cited by 555 · see citing works
Antisense Oligonucleotides: An Emerging Area in Drug Discovery and Development
Dhuri, K., Bechtold, C., Quijano, E., Pham, H. T. T., Gupta, A., Vikram, A., & Bahal, R. (2020) · Journal of Clinical Medicine · doi:10.3390/jcm9062004 · Cited by 554 · see citing works
Passini, M. A., Bu, J., Richards, A. M., Kinnecom, C., Sardi, S. P., Stanek, L. M., Hua, Y., Rigo, F., Matson, J. E., Hung, G., et al. (2011) · Science Translational Medicine · doi:10.1126/scitranslmed.3001777 · Cited by 547 · see citing works
A Glycine-Dependent Riboswitch That Uses Cooperative Binding to Control Gene Expression
Mandal, M., Lee, M. N., Barrick, J. E., Zasha Weinberg, A., Emilsson, G. M., Ruzzo, W. L., & Breaker, R. R. (2004) · Science · doi:10.1126/science.1100829 · Cited by 545 · see citing works
Familial Hypercholesterolemia: Screening, diagnosis and management of pediatric and adult patients
Goldberg, A. C., Hopkins, P. N., Toth, P. P., Ballantyne, C. M., Rader, D. J., Robinson, J. G., Daniels, S. R., Gidding, S. S., de Ferranti, S. D., Ito, M. K., et al. (2011) · Journal of Clinical Lipidology · doi:10.1016/j.jacl.2011.04.003 · Cited by 545 · see citing works
Lumasiran, an RNAi Therapeutic for Primary Hyperoxaluria Type 1
Garrelfs, S. F., Frishberg, Y., Hulton, S. A., Koren, M. J., O’Riordan, W. D., Cochat, P., Deschênes, G., Shasha-Lavsky, H., Saland, J. M., van’t Hoff, W. G., et al. (2021) · New England Journal of Medicine · doi:10.1056/nejmoa2021712 · Cited by 531 · see citing works
Alberer, M., Gnad-Vogt, U., Hong, H. S., Mehr, K. T., Backert, L., Finak, G., Gottardo, R., Bica, M. A., Garofano, A., Koch, S. D., et al. (2017) · The Lancet · doi:10.1016/s0140-6736(17)31665-3 · Cited by 530 · see citing works
RNA-directed gene editing specifically eradicates latent and prevents new HIV-1 infection
Hu, W., Kaminski, R., Yang, F., Zhang, Y., Cosentino, L., Li, F., Luo, B., Alvarez-Carbonell, D., Garcia-Mesa, Y., Karn, J., et al. (2014) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1405186111 · Cited by 527 · see citing works
CRISPR Interference Efficiently Induces Specific and Reversible Gene Silencing in Human iPSCs
Mandegar, M. A., Huebsch, N., Frolov, E. B., Shin, E., Truong, A., Olvera, M. P., Chan, A. H., Miyaoka, Y., Holmes, K., Spencer, C. I., et al. (2016) · Cell Stem Cell · doi:10.1016/j.stem.2016.01.022 · Cited by 525 · see citing works
Gold Nanoparticles for Nucleic Acid Delivery
Ding, Y., Jiang, Z., Saha, K., Kim, C. S., Kim, S. T., Landis, R. F., & Rotello, V. M. (2014) · Molecular Therapy · doi:10.1038/mt.2014.30 · Cited by 521 · see citing works
Mendell, J. R., Goemans, N., Lowes, L. P., Alfano, L. N., Berry, K., Shao, J., Kaye, E. M., & Mercuri, E. M. (2015) · Annals of Neurology · doi:10.1002/ana.24555 · Cited by 519 · see citing works
mRNA as a Transformative Technology for Vaccine Development to Control Infectious Diseases
Maruggi, G., Zhang, C., Li, J., Ulmer, J. B., & Yu, D. (2019) · Molecular Therapy · doi:10.1016/j.ymthe.2019.01.020 · Cited by 507 · see citing works
Vogel, A. B., Lambert, L., Kinnear, E., Busse, D., Erbar, S., Reuter, K. C., Wicke, L., Perkovic, M., Beissert, T., Haas, H., et al. (2018) · Molecular Therapy · doi:10.1016/j.ymthe.2017.11.017 · Cited by 501 · see citing works
Development of CRISPR as an Antiviral Strategy to Combat SARS-CoV-2 and Influenza
Abbott, T. R., Dhamdhere, G. R., Liu, Y., Lin, X., Goudy, L., Zeng, L., Chemparathy, A. G., Chmura, S. A., Heaton, N. S., Debs, R. J., et al. (2020) · Cell · doi:10.1016/j.cell.2020.04.020 · Cited by 500 · see citing works
The growth of siRNA-based therapeutics: updated clinical studies
Zhang, M. M., Bahal, R., Rasmussen, T. P., Manautou, J. E., & Zhong, X.-B. (2021) · Biochemical Pharmacology · doi:10.1016/j.bcp.2021.114432 · Cited by 497 · see citing works
Eteplirsen in the treatment of Duchenne muscular dystrophy
Lim, K. R. Q., Maruyama, R., & Yokota, T. (2017) · Drug Design Development and Therapy · doi:10.2147/dddt.s97635 · Cited by 496 · see citing works
Systemic Leukocyte-Directed siRNA Delivery Revealing Cyclin D1 as an Anti-Inflammatory Target
Peer, D., Park, E. J., Morishita, Y., Carman, C. V., & Shimaoka, M. (2008) · Science · doi:10.1126/science.1149859 · Cited by 491 · see citing works
Self-amplifying RNA vaccines for infectious diseases
Bloom, K., van den Berg, F., & Arbuthnot, P. (2021) · Gene Therapy · doi:10.1038/s41434-020-00204-y · Cited by 487 · see citing works
The distributions, mechanisms, and structures of metabolite-binding riboswitches
Barrick, J. E., & Breaker, R. R. (2007) · Genome biology · doi:10.1186/gb-2007-8-11-r239 · Cited by 482 · see citing works
McKay, P. F., Hu, K., Blakney, A. K., Samnuan, K., Brown, J. C., Penn, R., Zhou, J., Bouton, C. R., Rogers, P., Polra, K., et al. (2020) · Nature Communications · doi:10.1038/s41467-020-17409-9 · Cited by 462 · see citing works
Kloosterman, W. P., Lagendijk, A. K., Ketting, R. F., Moulton, J. D., & Plasterk, R. H. A. (2007) · PLoS Biology · doi:10.1371/journal.pbio.0050203 · Cited by 461 · see citing works
DNA Vaccines—How Far From Clinical Use?
Hobernik, D., & Bros, M. (2018) · International Journal of Molecular Sciences · doi:10.3390/ijms19113605 · Cited by 457 · see citing works
Specificity, duplex degradation and subcellular localization of antagomirs
Krützfeldt, J., Kuwajima, S., Braich, R. S., Rajeev, K. G., Pena, J., Tuschl, T., Manoharan, M., & Stoffel, M. (2007) · Nucleic Acids Research · doi:10.1093/nar/gkm024 · Cited by 456 · see citing works
Feldman, R. A., Fuhr, R., Smolenov, I., (Mick) Ribeiro, A., Panther, L., Watson, M., Senn, J. J., Smith, M., Almarsson, Ӧ., Pujar, H. S., et al. (2019) · Vaccine · doi:10.1016/j.vaccine.2019.04.074 · Cited by 454 · see citing works
Preclinical and clinical development of siRNA-based therapeutics
Özcan, G., Özpolat, B., Coleman, R. L., Sood, A. K., & Lopez‐Berestein, G. (2015) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2015.01.007 · Cited by 451 · see citing works
Wang, Y., Kang, H., Liu, X., & Tong, Z. (2020) · Journal of Medical Virology · doi:10.1002/jmv.25721 · Cited by 451 · see citing works
Mapping the genomic landscape of CRISPR-Cas9 cleavage
Cameron, P., Fuller, C. K., Donohoue, P. D., Jones, B. N., Thompson, M. S., Carter, M. M., Gradia, S., Vidal, B., Garner, E., Slorach, E. M., et al. (2017) · Nature Methods · doi:10.1038/nmeth.4284 · Cited by 443 · see citing works
Using siRNA in prophylactic and therapeutic regimens against SARS coronavirus in Rhesus macaque
Li, B.-J., Tang, Q., Cheng, D., Qin, C., Xie, F. Y., Wei, Q., Xu, J., Liu, Y., Zheng, B.-J., Woodle, M. C., et al. (2005) · Nature Medicine · doi:10.1038/nm1280 · Cited by 433 · see citing works
Chemical modification and design of anti-miRNA oligonucleotides
Lennox, K. A., & Behlke, M. A. (2011) · Gene Therapy · doi:10.1038/gt.2011.100 · Cited by 432 · see citing works
siRNA Delivery Strategies: A Comprehensive Review of Recent Developments
Tatiparti, K., Sau, S., Kashaw, S. K., & Iyer, A. K. (2017) · Nanomaterials · doi:10.3390/nano7040077 · Cited by 429 · see citing works
Improved targeting of miRNA with antisense oligonucleotides
Davis, S. (2006) · Nucleic Acids Research · doi:10.1093/nar/gkl183 · Cited by 422 · see citing works
Scott, L. J. (2020) · Drugs · doi:10.1007/s40265-020-01269-0 · Cited by 422 · see citing works
RNA-Based Therapeutics: From Antisense Oligonucleotides to miRNAs
Bajan, S., & Hutvágner, G. (2020) · Cells · doi:10.3390/cells9010137 · Cited by 399 · see citing works
Cech, T. R. (2000) · Science · doi:10.1126/science.289.5481.878 · Cited by 382 · see citing works
Protection against lethal influenza virus challenge by RNA interference in vivo
Tompkins, S. M., Lo, C.-Y., Tumpey, T. M., & Epstein, S. L. (2004) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.0402630101 · Cited by 378 · see citing works
A CRISPR-based approach for targeted DNA demethylation
Xu, X., Tao, Y., Gao, X., Zhang, L., Li, X., Zou, W., Ruan, K., Wang, F., Xu, G., & Hu, R. (2016) · Cell Discovery · doi:10.1038/celldisc.2016.9 · Cited by 377 · see citing works
Tailoring mRNA Vaccine to Balance Innate/Adaptive Immune Response
Linares-Fernández, S., Lacroix, C., Exposito, J.-Y., & Verrier, B. (2020) · Trends in Molecular Medicine · doi:10.1016/j.molmed.2019.10.002 · Cited by 376 · see citing works
Riboswitches: Structures and MechanismsPreprint
Garst, A. D., Edwards, A. L., & Batey, R. (2010) · Cold Spring Harbor Perspectives in Biology · doi:10.1101/cshperspect.a003533 · Cited by 373 · see citing works
Phosphorothioate modified oligonucleotide–protein interactions
Crooke, S. T., Vickers, T. A., & Liang, X.-H. (2020) · Nucleic Acids Research · doi:10.1093/nar/gkaa299 · Cited by 372 · see citing works
Targeting of Antithrombin in Hemophilia A or B with RNAi Therapy
Pasi, J., Rangarajan, S., Georgiev, P., Mant, T., Creagh, M. D., Lissitchkov, T. J., Bevan, D. H., Austin, S. K., Hay, C. R. M., Hegemann, I., et al. (2017) · New England Journal of Medicine · doi:10.1056/nejmoa1616569 · Cited by 369 · see citing works
Orlandini von Niessen, A. G., Poleganov, M. A., Rechner, C., Plaschke, A., Kranz, L. M., Fesser, S., Diken, M., Löwer, M., Vallazza, B., Beissert, T., et al. (2019) · Molecular Therapy · doi:10.1016/j.ymthe.2018.12.011 · Cited by 365 · see citing works
FDA Approves Eteplirsen for Duchenne Muscular Dystrophy: The Next Chapter in the Eteplirsen Saga
Aartsma-Rus, A. M., & Krieg, A. Μ. (2016) · Nucleic Acid Therapeutics · doi:10.1089/nat.2016.0657 · Cited by 364 · see citing works
Comparative Structure and Function Analysis of the RIG-I-Like Receptors: RIG-I and MDA5
Brisse, M. E., & Ly, H. (2019) · Frontiers in Immunology · doi:10.3389/fimmu.2019.01586 · Cited by 361 · see citing works
Determination of the Role of the Human RNase H1 in the Pharmacology of DNA-like Antisense Drugs
Wu, H., Lima, W. F., Zhang, H., Fan, A., Sun, H., & Crooke, S. T. (2004) · Journal of Biological Chemistry · doi:10.1074/jbc.m311683200 · Cited by 351 · see citing works
(2001) · PubMed Central · Cited by 342 · see citing works
A Cationic Nanoemulsion for the Delivery of Next-generation RNA Vaccines
Brito, L. A., Chan, M., Shaw, C. A., Hekele, A., Carsillo, T., Schaefer, M., Archer, J., Seubert, A., Otten, G. R., Beard, C. W., et al. (2014) · Molecular Therapy · doi:10.1038/mt.2014.133 · Cited by 338 · see citing works
Use of the CRISPR/Cas9 system as an intracellular defense against HIV-1 infection in human cells
Liao, H., Gu, Y., Diaz, A., Marlett, J. M., Takahashi, Y., Li, M., Suzuki, K., Xu, R., Hishida, T., Chang, C.-J., et al. (2015) · Nature Communications · doi:10.1038/ncomms7413 · Cited by 338 · see citing works
Sekijima, Y. (2015) · Journal of Neurology Neurosurgery & Psychiatry · doi:10.1136/jnnp-2014-308724 · Cited by 337 · see citing works
Seto, A. G., Beatty, X., Lynch, J. M., Hermreck, M., Tetzlaff, M. T., Duvic, M. A., & Jackson, A. L. (2018) · British Journal of Haematology · doi:10.1111/bjh.15547 · Cited by 333 · see citing works
Increased dystrophin production with golodirsen in patients with Duchenne muscular dystrophy
Frank, D. E., Schnell, F. J., Akana, C., El-Husayni, S. H., Desjardins, C. A., Morgan, J. E., Charleston, J. S., Sardone, V., Domingos, J., Dickson, G., et al. (2020) · Neurology · doi:10.1212/wnl.0000000000009233 · Cited by 332 · see citing works
A Pilot Study of the Telomerase Inhibitor Imetelstat for Myelofibrosis
Tefferi, A., Lasho, T. L., Begna, K. H., Patnaik, M. M., Zblewski, D., Finke, C. M., Laborde, R. R., Wassie, E. A., Schimek, L., Hanson, C. A., et al. (2015) · New England Journal of Medicine · doi:10.1056/nejmoa1310523 · Cited by 323 · see citing works
Allergen-Induced Asthmatic Responses Modified by a GATA3-Specific DNAzyme
Krug, N., Hohlfeld, J. M., Kirsten, A., Kornmann, O., Beeh, K. M., Kappeler, D., Korn, S., Ignatenko, S., Timmer, W., Rogon, C., et al. (2015) · New England Journal of Medicine · doi:10.1056/nejmoa1411776 · Cited by 321 · see citing works
Induction of the interferon response by siRNA is cell type– and duplex length–dependent
Reynolds, A., Anderson, E. M., Vermeulen, A., Federov, Y., Robinson, K., Leake, D., Karpilow, J. M., Marshall, W. S., & Khvorova, A. (2006) · RNA · doi:10.1261/rna.2340906 · Cited by 317 · see citing works
Heo, Y. (2020) · Drugs · doi:10.1007/s40265-020-01267-2 · Cited by 317 · see citing works
Schulte, L. N., Eulalio, A., Mollenkopf, H., Reinhardt, R., & Vogel, J. (2011) · EMBO Journal · doi:10.1038/emboj.2011.94 · Cited by 316 · see citing works
The Emerging Role of RNA as a Therapeutic Target for Small Molecules
Connelly, C. M., Moon, M. H., & Schneekloth, J. S. (2016) · Cell chemical biology · doi:10.1016/j.chembiol.2016.05.021 · Cited by 315 · see citing works
Cell specific delivery of modified mRNA expressing therapeutic proteins to leukocytes
Veiga, N., Goldsmith, M., Granot, Y., Rosenblum, D., Dammes, N., Kedmi, R., Ramishetti, S., & Peer, D. (2018) · Nature Communications · doi:10.1038/s41467-018-06936-1 · Cited by 308 · see citing works
On the art of identifying effective and specific siRNAs
Pei, Y., & Tuschl, T. (2006) · Nature Methods · doi:10.1038/nmeth911 · Cited by 302 · see citing works
Kennedy, E. M., Kornepati, A. V. R., Goldstein, M., Bogerd, H. P., Poling, B. C., Whisnant, A. W., Kastan, M. B., & Cullen, B. R. (2014) · Journal of Virology · doi:10.1128/jvi.01879-14 · Cited by 296 · see citing works
MicroRNA-223 controls susceptibility to tuberculosis by regulating lung neutrophil recruitment
Dorhoi, A., Iannaccone, M., Farinacci, M., Fae, K. C., Schreiber, J., Moura‐Alves, P., Nouailles, G., Mollenkopf, H., Oberbeck‐Müller, D., Jörg, S., et al. (2013) · Journal of Clinical Investigation · doi:10.1172/jci67604 · Cited by 296 · see citing works
In Vivo Excision of HIV-1 Provirus by saCas9 and Multiplex Single-Guide RNAs in Animal Models
Yin, C., Zhang, T., Qu, X., Zhang, Y., Putatunda, R., Xiao, X., Li, F., Xiao, W., Zhao, H., Dai, S., et al. (2017) · Molecular Therapy · doi:10.1016/j.ymthe.2017.03.012 · Cited by 296 · see citing works
Altered gene expression in plants due totrans interactions between homologous genes
Jorgensen, R. A. (1990) · Trends in biotechnology · doi:10.1016/0167-7799(90)90220-r · Cited by 289 · see citing works
McKinlay, C. J., Vargas, J. R., Blake, T. R., Hardy, J. W., Kanada, M., Contag, C. H., Wender, P. A., & Waymouth, R. M. (2017) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1614193114 · Cited by 288 · see citing works
Bitko, V., & Barik, S. (2001) · BMC Microbiology · doi:10.1186/1471-2180-1-34 · Cited by 287 · see citing works
CRISPR/Cas9-Mediated Genome Editing of Herpesviruses Limits Productive and Latent Infections
van Diemen, F. R., Kruse, E. M., Hooykaas, M. J. G., Bruggeling, C. E., Schürch, A. C., van Ham, P. M., Imhof, S. M., Nijhuis, M., Wiertz, E. J. H. J., & Lebbink, R. J. (2016) · PLoS Pathogens · doi:10.1371/journal.ppat.1005701 · Cited by 285 · see citing works
Zhou, J.-H., Swiderski, P., Li, H.-T., Zhang, J., Neff, C., Akkina, R., & Rossi, J. (2009) · Nucleic Acids Research · doi:10.1093/nar/gkp185 · Cited by 284 · see citing works
Gallant‐Behm, C. L., Piper, J., Lynch, J. M., Seto, A. G., Hong, S. J., Mustoe, T. A., Maari, C., Pestano, L. A., Dalby, C. M., Jackson, A. L., et al. (2018) · Journal of Investigative Dermatology · doi:10.1016/j.jid.2018.11.007 · Cited by 283 · see citing works
Pardi, N., Parkhouse, K., Kirkpatrick, E., McMahon, M., Zost, S. J., Mui, B. L., Tam, Y. K., Karikó, K., Barbosa, C. J., Madden, T. D., et al. (2018) · Nature Communications · doi:10.1038/s41467-018-05482-0 · Cited by 281 · see citing works
Gaudet, D., Karwatowska-Prokopczuk, E., Baum, S. J., Hurh, E., Kingsbury, J., Bartlett, V. J., Figueroa, A. L., Piscitelli, P., Singleton, W., Witztum, J. L., et al. (2020) · European Heart Journal · doi:10.1093/eurheartj/ehaa689 · Cited by 279 · see citing works
Xiao, B., Liu, Z., Li, B., Tang, B., Li, W., Guo, G., Shi, Y., Wang, F., Wu, Y., Tong, W., et al. (2009) · The Journal of Infectious Diseases · doi:10.1086/605443 · Cited by 278 · see citing works
Anti-miRNA oligonucleotides: A comprehensive guide for design
Lima, J., Cerqueira, L., Figueiredo, C., Oliveíra, C., & Azevedo, N. F. (2018) · RNA Biology · doi:10.1080/15476286.2018.1445959 · Cited by 278 · see citing works
Wang, F., Zuroske, T., & Watts, J. K. (2020) · Nature Reviews Drug Discovery · doi:10.1038/d41573-020-00078-0 · Cited by 275 · see citing works
van der Ree, M. H., de Vree, J. M., Stelma, F., Willemse, S. B., van der Valk, M., Rietdijk, S. T., Molenkamp, R., Schinkel, J., van Nuenen, A. C., Beuers, U. H., et al. (2017) · The Lancet · doi:10.1016/s0140-6736(16)31715-9 · Cited by 271 · see citing works
Rader, D. J., & Kastelein, J. J. P. (2014) · Circulation · doi:10.1161/circulationaha.113.001292 · Cited by 264 · see citing works
Phase 2 gene therapy trial of an anti-HIV ribozyme in autologous CD34+ cells
Mitsuyasu, R. T., Merigan, T. C., Carr, A. D., Zack, J. A., Winters, M. A., Workman, C., Bloch, M. T., Lalezari, J. P., Becker, S., Thornton, L., et al. (2009) · Nature Medicine · doi:10.1038/nm.1932 · Cited by 263 · see citing works
Phase 1 Trial of an RNA Interference Therapy for Acute Intermittent Porphyria
Sardh, E., Harper, P., Balwani, M., Stein, P. E., Rees, D. C., Bissell, D. M., Desnick, R. J., Parker, C., Phillips, J. D., Bonkovsky, H. L., et al. (2019) · New England Journal of Medicine · doi:10.1056/nejmoa1807838 · Cited by 259 · see citing works
Genetic determinants of plasma triglycerides
Johansen, C. T., Kathiresan, S., & Hegele, R. A. (2010) · Journal of Lipid Research · doi:10.1194/jlr.r009720 · Cited by 258 · see citing works
Fabani, M. M., & Gait, M. J. (2007) · RNA · doi:10.1261/rna.844108 · Cited by 253 · see citing works
Delivery of therapeutic oligonucleotides with cell penetrating peptides
Boisguérin, P., Deshayes, S., Gait, M. J., O’Donovan, L., Godfrey, C., Betts, C., Wood, M. J. A., & Lebleu, B. (2015) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2015.02.008 · Cited by 245 · see citing works
Evolving landscape in the management of transthyretin amyloidosis
Hawkins, P. N., Ando, Y., Dispenzeri, A., González‐Duarte, A., Adams, D., & Suhr, O. B. (2015) · Annals of Medicine · doi:10.3109/07853890.2015.1068949 · Cited by 243 · see citing works
Review of CRISPR/Cas9 sgRNA Design Tools
Cui, Y., Xu, J., Cheng, M., Liao, X., & Peng, S. (2018) · Interdisciplinary Sciences Computational Life Sciences · doi:10.1007/s12539-018-0298-z · Cited by 243 · see citing works
Nusinersen, an antisense oligonucleotide drug for spinal muscular atrophy
Corey, D. R. (2017) · Nature Neuroscience · doi:10.1038/nn.4508 · Cited by 242 · see citing works
Passive immunotherapy of viral infections: ‘super-antibodies’ enter the fray
Walker, L., & Burton, D. (2018) · Nature reviews. Immunology · doi:10.1038/nri.2017.148 · Cited by 242 · see citing works
Erasmus, J. H., Khandhar, A. P., O’Connor, M. A., Walls, A. C., Hemann, E. A., Murapa, P., Archer, J., Leventhal, S., Fuller, J. T., Lewis, T. B., et al. (2020) · Science Translational Medicine · doi:10.1126/scitranslmed.abc9396 · Cited by 241 · see citing works
Locked nucleic acid: modality, diversity, and drug discovery
Hagedorn, P., Persson, R., Funder, E. D., Albæk, N., Diemer, S. L., Hansen, D. J., Møller, M. R., Papargyri, N., Christiansen, H., Hansen, B. R., et al. (2017) · Drug Discovery Today · doi:10.1016/j.drudis.2017.09.018 · Cited by 237 · see citing works
Bioconjugated Oligonucleotides: Recent Developments and Therapeutic Applications
Benizri, S., Gissot, A., Martin, A., Vialet, B., Grinstaff, M. W., & Barthélémy, P. (2019) · Bioconjugate Chemistry · doi:10.1021/acs.bioconjchem.8b00761 · Cited by 233 · see citing works
Gouni-Berthold, I., Alexander, V. J., Yang, Q., Hurh, E., Steinhagen-Thiessen, E., Moriarty, P. M., Hughes, S. G., Gaudet, D., Hegele, R. A., O'Dea, L. S. L., et al. (2021) · The Lancet Diabetes & Endocrinology · doi:10.1016/s2213-8587(21)00046-2 · Cited by 232 · see citing works
Alexander, V. J., Xia, S., Hurh, E., Hughes, S. G., O’Dea, L., Geary, R. S., Witztum, J. L., & Tsimikas, S. (2019) · European Heart Journal · doi:10.1093/eurheartj/ehz209 · Cited by 231 · see citing works
In Vivo Targeting of Clostridioides difficile Using Phage-Delivered CRISPR-Cas3 Antimicrobials
Selle, K., Fletcher, J. R., Tuson, H. H., Schmitt, D. S., McMillan, L. J., Vridhambal, G. S., Rivera, A. J., Montgomery, S. A., Fortier, L., Barrangou, R., et al. (2020) · mBio · doi:10.1128/mbio.00019-20 · Cited by 228 · see citing works
The peptide nucleic acids (PNAs), powerful tools for molecular genetics and cytogenetics
Pellestor, F., & Paulasova, P. (2004) · European Journal of Human Genetics · doi:10.1038/sj.ejhg.5201226 · Cited by 224 · see citing works
Crooke, R. M., Graham, M. J., Lemonidis, K. M., Whipple, C. P., Koo, S., & Perera, R. J. (2005) · Journal of Lipid Research · doi:10.1194/jlr.m400492-jlr200 · Cited by 224 · see citing works
Development of antibody-siRNA conjugate targeted to cardiac and skeletal muscles
Sugo, T., Terada, M., Oikawa, T., Miyata, K., Nishimura, S., Kenjo, E., Ogasawara-Shimizu, M., Makita, Y., Imaichi, S., Murata, S., et al. (2016) · Journal of Controlled Release · doi:10.1016/j.jconrel.2016.06.036 · Cited by 224 · see citing works
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 · see citing works
Flagged first, then by citations
9 of the 435 references couldn’t be matched to a known paper.
Cited by
Sorted by citations
Exosome for mRNA delivery: Strategies and therapeutic applications
Iqbal, Z., Rehman, K., Mahmood, A., Shabbir, M., Liang, Y., Duan, L., & Zeng, H. (2024) · Journal of Nanobiotechnology · doi:10.1186/s12951-024-02634-x · Cited by 144 · see citing works
Emerging Delivery Systems for Enabling Precision Nucleic Acid Therapeutics
Bian, X., Zhou, L., Luo, Z., Liu, G., Hang, Z., Li, H., Li, F., & Wen, Y. (2025) · ACS Nano · doi:10.1021/acsnano.4c11858 · Cited by 97 · see citing works
Using aptamers for targeted delivery of RNA therapies
Driscoll, J., Gondaliya, P., Zinn, D. A., Jain, R., Yan, I. K., Dong, H., & Patel, T. C. (2025) · Molecular Therapy · doi:10.1016/j.ymthe.2025.02.047 · Cited by 58 · see citing works
mRNA vaccines: a new era in vaccine development
Chandra, S., Wilson, J. C., Good, D. M., & Wei, M. Q. (2024) · Oncology Research Featuring Preclinical and Clinical Cancer Therapeutics · doi:10.32604/or.2024.043987 · Cited by 58 · see citing works
Dai, B., Zhu, Y., Li, X., Liang, Z., Xu, S., Zhang, S., Zhang, Z., Bai, S., Tong, W., Cao, M., et al. (2023) · Advanced Science · doi:10.1002/advs.202300897 · Cited by 45 · see citing works
MicroRNA-based therapeutics for inflammatory disorders of the microbiota-gut-brain axis
Datta, N., Johnson, C., Kao, D. H., Gurnani, P., Alexander, C., Polytarchou, C., & Monaghan, T. M. (2023) · Pharmacological Research · doi:10.1016/j.phrs.2023.106870 · Cited by 39 · see citing works
Călina, D. C., Hernández, A. F., Härtung, T., Egorov, A. M., Izotov, B. N., Νikolouzakis, Τ. Κ., Tsatsakis, A. M., Vlachoyiannopoulos, P. G., & Docea, A. O. (2021) · Life · doi:10.3390/life11090907 · Cited by 31 · see citing works
The Novelty of mRNA Viral Vaccines and Potential Harms: A Scoping Review
Halma, M. T. J., Rose, J., Jenks, A., & Lawrie, T. A. (2023) · J — Multidisciplinary Scientific Journal · doi:10.3390/j6020017 · Cited by 25 · see citing works
MicroRNAs modulate neuroinflammation after intracerebral hemorrhage: Prospects for new therapy
Xia, S., Zheng, Y., Yan, F., & Chen, G. (2022) · Frontiers in Immunology · doi:10.3389/fimmu.2022.945860 · Cited by 24 · see citing works
Babalola, B., Akinsuyi, O. S., Folajimi, E. O., Olujimi, F., Otunba, A. A., Chikere, B., Adewumagun, I. A., & Adetobi, T. E. (2023) · Biomedicine & pharmacotherapy = Biomedecine & pharmacotherapie · doi:10.1016/j.biopha.2023.115099 · Cited by 15 · see citing works
Development of microRNA-based therapeutics for central nervous system diseases
Xia, S., Xu, C., Liu, F., & Chen, G. (2023) · European Journal of Pharmacology · doi:10.1016/j.ejphar.2023.175956 · Cited by 18 · see citing works
Sofroniou, A., & Ridley, A. (2025) · British Journal of Biomedical Science · doi:10.3389/bjbs.2025.14557 · Cited by 13 · see citing works
RNA research for drug discovery: Recent advances and critical insight
Aja, P. M., Agu, P. C., Ogbu, C. O., Alum, E. U., Fasogbon, I. V., Musyoka, A. M., Ngwueche, W., Egwu, C. O., Tusubira, D., & Ross, K. (2025) · Gene · doi:10.1016/j.gene.2025.149342 · Cited by 16 · see citing works
Sartorius, K., Antwi, S. O., Chuturgoon, A. A., Roberts, L. R., & Kramvis, A. (2022) · Frontiers in Oncology · doi:10.3389/fonc.2022.891812 · Cited by 12 · see citing works
Cui, W., Lin, Q., Wu, Y., Wang, X., Zhang, Y., Lin, X., Zhang, L., Liu, X., Han, L., & Zhou, Z. (2023) · ACS Synthetic Biology · doi:10.1021/acssynbio.3c00519 · Cited by 6 · see citing works
RNA-Based Vaccines and Therapeutics Against Intracellular Pathogens
Kola, N. S., Patel, D., & Thakur, A. (2024) · Methods in molecular biology · doi:10.1007/978-1-0716-3890-3_21 · Cited by 6 · see citing works
Tang, Y., Zou, J., Li, Q., Liu, Y., Guo, X. D., Bai, H., He, X. D., Gan, C., Zhao, X., Wu, W., et al. (2025) · International Journal of Pharmaceutics · doi:10.1016/j.ijpharm.2025.126189 · Cited by 5 · see citing works
Transitional Insight into the RNA-Based Oligonucleotides in Cancer Treatment
Tabasi, H., Mollazadeh, S., Fazeli, E., Abnus, K., Taghdisi, S. M., Ramezani, M., & Alibolandi, M. (2023) · Applied Biochemistry and Biotechnology · doi:10.1007/s12010-023-04597-5 · Cited by 5 · see citing works
Qureshi, A., & Connolly, J. B. (2023) · Malaria Journal · doi:10.1186/s12936-023-04665-5 · Cited by 4 · see citing works
Aptamer-Based Targeted Drug Delivery Systems
Tiwari, R., Gulbake, A., Kumar, P., Gulbake, A. S., Gupta, R., Bisht, D., & Sethiya, N. K. (2022) · Nanotechnology in the life sciences · doi:10.1007/978-3-031-12658-1_4 · Cited by 3 · see citing works
Nedaeinia, R., Ranjbar, M., Goli, M., Etebari, M., Safabakhsh, S., Bayram, H., Ferns, G. A., Tehrani, H. M., & Salehi, R. (2024) · Current Medicinal Chemistry · doi:10.2174/0109298673300236240529195835 · Cited by 1 · see citing works
An Introduction to RNA-Based Therapeutics
Dakal, T. C., & Gupta, B. (2025) · Springer eBooks · doi:10.1007/978-981-96-3456-9_1 · Cited by 1 · see citing works
mRNA nanotherapeutics for sepsis: immune modulation strategies and translational challenges
Liu, Y., Wang, K., Gao, F., Bai, X., Yang, J., Li, Z., & Wang, Y.-C. (2026) · Journal of Nanobiotechnology · doi:10.1186/s12951-026-04366-6 · Cited by 1 · see citing works
- Authors
- Pandey, M., Ojha, D., Bansal, S., Rode, A. B., & Chawla, G.
- Affiliations
- Regional Centre for Biotechnology (IN)
- Corresponding author
- Rode, Ambadas B.
- Chawla, Geetanjali
- Venue
- Molecular Aspects of Medicine
- Publisher
- Elsevier BV
- Published
- 2021-10
- Volume
- 81
- Pages
- 101003
- ISSN
- 0098-2997 (ISSN-L)
- OpenAlex
- W3185845179
- Cited by
- 23
- References
- 435
- Citations by year
- 2026: 1 · 2025: 6 · 2024: 4 · 2023: 8 · 2022: 3 · 2021: 1
- FWCI
- 1.26
- Type
- journal article
- Language
- en
- Open access
- closed
- Licence
- Locations
- Molecular Aspects of Medicine (publishedVersion)
- PubMed (publishedVersion)
- Funders
- Department of Science and Technology, Ministry of Science and Technology, India (IA/I(S)/17/1/503085)
- The Wellcome Trust DBT India Alliance (IA/I(S)/17/1/503085)
- Department of Biotechnology, Ministry of Science and Technology, India (IA/I(S)/17/1/503085)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA and protein synthesis mechanisms
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Research and Splicing
- Keywords
- RNA therapeutics, RNA vaccines, infectious diseases, mRNA vaccines, antisense oligonucleotides, siRNA, miRNA mimics, antagomir, aptamers, riboswitches, CRISPR/Cas9, RNA delivery systems, RNA modification, immunogenicity, translational medicine, clinical trials
- MeSH terms
- mRNA Vaccines, Communicable Diseases, Humans, Oligonucleotides, Vaccines, Synthetic, Oligonucleotides, Antisense, RNA, Small Interfering
About these checks
Registry, retraction, Cites and statistics results are computed, not judged, with one exception: a printed reference that no index lists is read once by a model before it counts as a citation error. Claims are extracted by a model and kept only when their quote appears verbatim in the text; each cited source is then checked, with a model’s help, for whether it says what the claim relies on. Those verdicts can be wrong — read the claim’s quote and the cited source before relying on one.
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