Improving siRNA Delivery In Vivo Through Lipid Conjugation

Therapeutic silencing of an endogenous gene by systemic administration of modified siRNAs

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The chemical evolution of oligonucleotide therapies of clinical utility

Khvorova, A., & Watts, J. K. (2017) · Nature Biotechnology · doi:10.1038/nbt.3765 · Cited by 1,216 · see citing works

Multivalent <i>N</i> -Acetylgalactosamine-Conjugated siRNA Localizes in Hepatocytes and Elicits Robust RNAi-Mediated Gene Silencing

Nair, J. K., Willoughby, J. L. S., Chan, A., Charisse, K., Alam, M. R., Wang, Q., Hoekstra, M., Kandasamy, P., Kel’in, A. V., Milstein, S., et al. (2014) · Journal of the American Chemical Society · doi:10.1021/ja505986a · Cited by 1,141 · 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

Mechanisms and optimization of in vivo delivery of lipophilic siRNAs

Wolfrum, C., Shi, S., Jayaprakash, K. N., Jayaraman, M., Wang, G., Pandey, R. K., Rajeev, K. G., Nakayama, T., Charrise, K., Ndungo, E. M., et al. (2007) · Nature Biotechnology · doi:10.1038/nbt1339 · Cited by 974 · see citing works

Pharmacokinetics, biodistribution and cell uptake of antisense oligonucleotides

Geary, R. S., Norris, D. A., Yu, R. Z., & Bennett, C. F. (2015) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2015.01.008 · Cited by 846 · see citing works

A highly durable RNAi therapeutic inhibitor of PCSK9

Fitzgerald, K., White, S., Borodovsky, A., Bettencourt, B. R., Strahs, A., Clausen, V., Wijngaard, P., Horton, J. D., Taubel, J., Brooks, A., et al. (2017) · New England Journal of Medicine · doi:10.1056/nejmoa1609243 · Cited by 768 · see citing works

Lipid map of the mammalian cell

Gerrit van, M. E. E. R., & de Kroon, A. I. P. M. (2010) · Journal of Cell Science · doi:10.1242/jcs.071233 · Cited by 688 · see citing works

Phosphorothioates, Essential Components of Therapeutic Oligonucleotides

Eckstein, F. (2014) · Nucleic Acid Therapeutics · doi:10.1089/nat.2014.0506 · Cited by 684 · see citing works

Lipid‐based nanotherapeutics for siRNA delivery

Schroeder, A., Levins, C. G., Cortez, C., Langer, R., & Anderson, D. G. (2009) · Journal of Internal Medicine · doi:10.1111/j.1365-2796.2009.02189.x · Cited by 450 · see citing works

Fully 2‘-Modified Oligonucleotide Duplexes with Improved in Vitro Potency and Stability Compared to Unmodified Small Interfering RNA

Allerson, C. R., Sioufi, N. B., Jarres, R., Prakash, T. P., Naik, N., Berdeja, A., Wanders, L., Griffey, R. H., Swayze, E. E., & Bhat, B. (2005) · Journal of Medicinal Chemistry · doi:10.1021/jm049167j · Cited by 448 · see citing works

Therapeutic silencing of mutant huntingtin with siRNA attenuates striatal and cortical neuropathology and behavioral deficits

DiFiglia, M., Sena‐Esteves, M., Chase, K. O., Sapp, E., Pfister, E., Sass, M., Yoder, J., Reeves, P., Pandey, R. K., Rajeev, K. G., et al. (2007) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.0708285104 · Cited by 417 · see citing works

Efficient In Vivo Delivery of siRNA to the Liver by Conjugation of α-Tocopherol

Nishina, K., Unno, T., Uno, Y., Kubodera, T., Kanouchi, T., Mizusawa, H., & Yokota, T. (2008) · Molecular Therapy · doi:10.1038/mt.2008.14 · Cited by 312 · see citing works

Preclinical and Clinical Advances of GalNAc-Decorated Nucleic Acid Therapeutics

Huang, Y. (2016) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2016.12.003 · Cited by 295 · see citing works

Single-Stranded siRNAs Activate RNAi in Animals

Lima, W. F., Prakash, T. P., Murray, H. M., Kinberger, G. A., Li, W., Chappell, A. E., Li, C., Murray, S., Gaus, H. J., Seth, P. P., et al. (2012) · Cell · doi:10.1016/j.cell.2012.08.014 · Cited by 256 · see citing works

Biodistribution of phosphodiester and phosphorothioate siRNA

Braasch, D. A., Paroo, Z., Constantinescu, A., Ren, G., Öz, O. K., Mason, R. P., & Corey, D. R. (2004) · Bioorganic & Medicinal Chemistry Letters · doi:10.1016/j.bmcl.2003.12.074 · Cited by 251 · see citing works

Cell-penetrating peptide-conjugated antisense oligonucleotides restore systemic muscle and cardiac dystrophin expression and function

Yin, H., Moulton, H. M., Seow, Y., Boyd, C., Boutilier, J. K., Iverson, P. L., & Wood, M. J. A. (2008) · Human Molecular Genetics · doi:10.1093/hmg/ddn293 · Cited by 225 · see citing works

Oligonucleotide Therapeutics — A New Class of Cholesterol-Lowering Drugs

Khvorova, A. (2017) · New England Journal of Medicine · doi:10.1056/nejmp1614154 · Cited by 184 · see citing works

Comparison of partially and fully chemically-modified siRNA in conjugate-mediated delivery in vivo

Hassler, M. R., Turanov, A. A., Alterman, J. F., Haraszti, R. A., Coles, A. H., Osborn, M. F., Echeverria, D., Nikan, M., Salomon, W. E., Roux, L., et al. (2018) · Nucleic Acids Research · doi:10.1093/nar/gky037 · Cited by 181 · see citing works

Phosphorothioates in molecular biology

Eckstein, F., & Gish, G. D. (1989) · Trends in Biochemical Sciences · doi:10.1016/0968-0004(89)90130-8 · Cited by 175 · see citing works

Lipid nanoparticles for targeted siRNA delivery - going from bench to bedside

Zatsepin (2016) · International Journal of Nanomedicine · doi:10.2147/ijn.s106625 · Cited by 162 · see citing works

Asialoglycoprotein receptor 1 mediates productive uptake of N-acetylgalactosamine-conjugated and unconjugated phosphorothioate antisense oligonucleotides into liver hepatocytes

Tanowitz, M., Hettrick, L. A., Revenko, A. S., Kinberger, G. A., Prakash, T. P., & Seth, P. P. (2017) · Nucleic Acids Research · doi:10.1093/nar/gkx960 · Cited by 161 · see citing works

Hydrophobically Modified siRNAs Silence Huntingtin mRNA in Primary Neurons and Mouse Brain

Alterman, J. F., Hall, L. M., Coles, A. H., Hassler, M. R., Didiot, M.-C., Chase, K., Abraham, J., Sottosanti, E., Johnson, E., Sapp, E., et al. (2015) · Molecular Therapy - Nucleic Acids · doi:10.1038/mtna.2015.38 · Cited by 158 · see citing works

5′‐( E )‐Vinylphosphonate: A Stable Phosphate Mimic Can Improve the RNAi Activity of siRNA–GalNAc Conjugates

Parmar, R. G., Willoughby, J. L. S., Liu, J., Foster, D. J., Brigham, B., Theile, C. S., Charissé, K., Akinc, A., Guidry, E. N., Pei, Y., et al. (2016) · ChemBioChem · doi:10.1002/cbic.201600130 · Cited by 143 · see citing works

Visualization of self-delivering hydrophobically modified siRNA cellular internalization

Ly, S., Navaroli, D. M., Didiot, M.-C., Cardia, J., Pandarinathan, L., Alterman, J. F., Fogarty, K., Standley, C., Lifshitz, L. M., Bellve, K. D., et al. (2017) · Nucleic Acids Research · doi:10.1093/nar/gkw1005 · Cited by 132 · see citing works

5΄-Vinylphosphonate improves tissue accumulation and efficacy of conjugated siRNAs in vivo

Haraszti, R. Á., Roux, L., Coles, A. H., Turanov, A. A., Alterman, J. F., Echeverria, D., Godinho, B. M. D. C., D, N. A. M., & Khvorova, A. (2017) · Nucleic Acids Research · doi:10.1093/nar/gkx507 · Cited by 128 · see citing works

Lipophilic siRNA targets albumin in situ and promotes bioavailability, tumor penetration, and carrier-free gene silencing

Sarett, S. M., Werfel, T. A., Lee, L., Jackson, M. A., Kilchrist, K. V., Brantley‐Sieders, D. M., & Duvall, C. L. (2017) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1621240114 · Cited by 126 · see citing works

Gene silencing following siRNA delivery to skin via coated steel microneedles: In vitro and in vivo proof-of-concept

Chong, R. H. E., González-González, E., Lara, M. F., Speaker, T. J., Contag, C. H., Kaspar, R. L., Coulman, S. A., Hargest, R., & Birchall, J. C. (2013) · Journal of Controlled Release · doi:10.1016/j.jconrel.2012.12.030 · Cited by 122 · see citing works

Peptide-conjugated antisense oligonucleotides for targeted inhibition of a transcriptional regulator in vivo

Henke, E., Perk, J., Vider, J., de Candia, P., Chin, Y., Solit, D. B., Ponomarev, V., Cartegni, L., Manova, K., Rosen, N. X., et al. (2008) · Nature Biotechnology · doi:10.1038/nbt1366 · Cited by 119 · see citing works

Co-Injection of a Targeted, Reversibly Masked Endosomolytic Polymer Dramatically Improves the Efficacy of Cholesterol-Conjugated Small Interfering RNAs In Vivo

Wong, S. C., Klein, J. J., Hamilton, H., Chu, Q., Frey, C. L., Trubetskoy, V. S., Hegge, J., Wakefield, D. H., Rozema, D. B., & Lewis, D. L. (2012) · Nucleic Acid Therapeutics · doi:10.1089/nat.2012.0389 · Cited by 101 · see citing works

Peptide-mediated Cell and In Vivo Delivery of Antisense Oligonucleotides and siRNA

Järver, P., Coursindel, T., Andaloussi, S. E., Godfrey, C., Wood, M. J. A., & Gait, M. J. (2012) · Molecular Therapy — Nucleic Acids · doi:10.1038/mtna.2012.18 · Cited by 98 · see citing works

Stereochemical bias introduced during RNA synthesis modulates the activity of phosphorothioate siRNAs

Jahns, H., Roos, M. M., Imig, J., Baumann, F., Wang, Y., Gilmour, R., & Hall, J. (2015) · Nature Communications · doi:10.1038/ncomms7317 · Cited by 95 · see citing works

Characterizing the effect of GalNAc and phosphorothioate backbone on binding of antisense oligonucleotides to the asialoglycoprotein receptor

Schmidt, K., Prakash, T. P., Donner, A. J., Kinberger, G. A., Gaus, H. J., Low, A., Østergaard, M. E., Bell, M., Swayze, E. E., & Seth, P. P. (2017) · Nucleic Acids Research · doi:10.1093/nar/gkx060 · Cited by 93 · see citing works

Transdermal Delivery of siRNA through Microneedle Array

Deng, Y. P., Chen, J., Zhao, Y., Yan, X., Zhang, L., Choy, K. W., Hu, J., Sant, H. J., Gale, B. K., & Tang, T. (2016) · Scientific Reports · doi:10.1038/srep21422 · Cited by 92 · see citing works

Silencing Myostatin Using Cholesterol-conjugated siRNAs Induces Muscle Growth

Khan, T., Weber, H., DiMuzio, J. M., Matter, A. M., Dogdas, B., Shah, T., Thankappan, A., Disa, J., Jadhav, V., Lubbers, L. S., et al. (2016) · Molecular Therapy — Nucleic Acids · doi:10.1038/mtna.2016.55 · Cited by 90 · see citing works

siRNA carrying an (E)-vinylphosphonate moiety at the 5΄ end of the guide strand augments gene silencing by enhanced binding to human Argonaute-2

Elkayam, E., Parmar, R. G., Brown, C. R., Willoughby, J. L. S., Theile, C. S., Manoharan, M., & Joshua‐Tor, L. (2016) · Nucleic Acids Research · doi:10.1093/nar/gkw1171 · Cited by 90 · see citing works

Docosahexaenoic Acid Conjugation Enhances Distribution and Safety of siRNA upon Local Administration in Mouse Brain

Nikan, M., Osborn, M. F., Coles, A. H., Godinho, B. M., Hall, L. M., Haraszti, R. A., Hassler, M. R., Echeverria, D., Aronin, N., & Khvorova, A. (2016) · Molecular Therapy - Nucleic Acids · doi:10.1038/mtna.2016.50 · Cited by 85 · see citing works

Antisense Oligonucleotides Used to Target the DUX4 mRNA as Therapeutic Approaches in FaciosScapuloHumeral Muscular Dystrophy (FSHD)

Ansseau, E., Vanderplanck, C., Wauters, A., Harper, S. Q., Coppée, F., & Belayew, A. (2017) · Genes · doi:10.3390/genes8030093 · Cited by 80 · see citing works

Structural Analysis of Human Argonaute-2 Bound to a Modified siRNA Guide

Schirle, N. T., Kinberger, G. A., Murray, H. F., Lima, W. F., Prakash, T. P., & MacRae, I. J. (2016) · Journal of the American Chemical Society · doi:10.1021/jacs.6b04454 · Cited by 79 · see citing works

Novel Hydrophobically Modified Asymmetric RNAi Compounds (sd-rxRNA) Demonstrate Robust Efficacy in the Eye

Byrne, M., Tzekov, R. T., Wang, Y., Rodgers, A., Cardia, J. P., Ford, G., Holton, K., Pandarinathan, L., Lapierre, J., Stanney, W., et al. (2013) · Journal of Ocular Pharmacology and Therapeutics · doi:10.1089/jop.2013.0148 · Cited by 78 · see citing works

Endothelial Cell–Specific FGD5 Involvement in Vascular Pruning Defines Neovessel Fate in Mice

Cheng, C., Haasdijk, R. A., Tempel, D., van de Kamp, E. H. M., Herpers, R., Bos, F. L., den Dekker, W. K., Blonden, L. A. J., de Jong, R., Bürgisser, P. E., et al. (2012) · Circulation · doi:10.1161/circulationaha.111.064030 · Cited by 71 · see citing works

Chimeric Antisense Oligonucleotide Conjugated to α-Tocopherol

Nishina, T., Numata, J., Nishina, K., Yoshida‐Tanaka, K., Nitta, K., Piao, W., Iwata, R., Ito, S., Kuwahara, H., Wada, T., et al. (2015) · Molecular Therapy — Nucleic Acids · doi:10.1038/mtna.2014.72 · Cited by 70 · see citing works

Lipophilic siRNAs mediate efficient gene silencing in oligodendrocytes with direct CNS delivery

Chen, Q., Butler, D., Querbes, W., Pandey, R. K., Ge, P., Maier, M. A., Zhang, L., Rajeev, K. G., Nechev, L. V., Koteliansky, V., et al. (2010) · Journal of Controlled Release · doi:10.1016/j.jconrel.2010.02.011 · Cited by 67 · see citing works

Facile Synthesis, Geometry, and 2′-Substituent-Dependent in Vivo Activity of 5′-(E)- and 5′-(Z)-Vinylphosphonate-Modified siRNA Conjugates

Parmar, R. G., Brown, C. R., Matsuda, S., Willoughby, J. L. S., Theile, C. S., Charissé, K., Foster, D. J., Zlatev, I., Jadhav, V., Maier, M. A., et al. (2018) · Journal of Medicinal Chemistry · doi:10.1021/acs.jmedchem.7b01147 · Cited by 57 · see citing works

Cholesterol incorporation into bacterial membranes

Razin, S. (1975) · Journal of Bacteriology · doi:10.1128/jb.124.1.570-572.1975 · Cited by 55 · see citing works

Pharmacokinetic Profiling of Conjugated Therapeutic Oligonucleotides: A High-Throughput Method Based Upon Serial Blood Microsampling Coupled to Peptide Nucleic Acid Hybridization Assay

Godinho, B. M. D. C., Gilbert, J. W., Haraszti, R. A., Coles, A. H., Biscans, A., Roux, L., Nikan, M., Echeverria, D., Hassler, M., & Khvorova, A. (2017) · Nucleic Acid Therapeutics · doi:10.1089/nat.2017.0690 · Cited by 52 · see citing works

Synergistic effect of phosphorothioate, 5′-vinylphosphonate and GalNAc modifications for enhancing activity of synthetic siRNA

Prakash, T. P., Kinberger, G. A., Murray, H. M., Chappell, A. E., Riney, S., Graham, M. J., Lima, W. F., Swayze, E. E., & Seth, P. P. (2016) · Bioorganic & Medicinal Chemistry Letters · doi:10.1016/j.bmcl.2016.04.063 · Cited by 51 · see citing works

Synthesis and Evaluation of Parenchymal Retention and Efficacy of a Metabolically Stable <i>O</i>-Phosphocholine-<i>N</i>-docosahexaenoyl-<scp>l</scp>-serine siRNA Conjugate in Mouse Brain

Nikan, M., Osborn, M. F., Coles, A. H., Biscans, A., Godinho, B. M. D. C., Haraszti, R. A., Sapp, E., Echeverria, D., DiFiglia, M., Aronin, N., et al. (2017) · Bioconjugate Chemistry · doi:10.1021/acs.bioconjchem.7b00226 · Cited by 35 · see citing works

Gene Silencing in Skin After Deposition of Self-Delivery siRNA With a Motorized Microneedle Array Device

Hickerson, R. P., Wey, W. C., Rimm, D. L., Speaker, T. J., Suh, S., Flores, M. A., González-González, E., Leake, D., Contag, C. H., & Kaspar, R. L. (2013) · Molecular Therapy — Nucleic Acids · doi:10.1038/mtna.2013.56 · Cited by 29 · see citing works

Amino-Modified and Lipid-Conjugated Dicer-Substrate siRNA Enhances RNAi Efficacy

Kubo, T., Takei, Y., Mihara, K., Yanagihara, K., & Seyama, T. (2012) · Bioconjugate Chemistry · doi:10.1021/bc200333w · Cited by 26 · see citing works

Lipid-Conjugated 27-Nucleotide Double-Stranded RNAs with Dicer-Substrate Potency Enhance RNAi-Mediated Gene Silencing

Kubo, T., Yanagihara, K., Takei, Y., Mihara, K., Sato, Y., & Seyama, T. (2012) · Molecular Pharmaceutics · doi:10.1021/mp2006278 · Cited by 23 · see citing works

Specific Cellular Incorporation of a Pyrene-Labelled Cholesterol: Lipoprotein-Mediated Delivery toward Ordered Intracellular Membranes

Gaibelet, G., Allart, S., Tercé, F., Azalbert, V., Bertrand‐Michel, J., Hamdi, S. M., Collet, X., & Orlowski, S. (2015) · PLoS ONE · doi:10.1371/journal.pone.0121563 · Cited by 15 · see citing works

Therapeutic siRNA: state of the art

Hu, B., Zhong, L., Weng, Y., Peng, L., Huang, Y., Zhao, Y., & Liang, X.-J. (2020) · Signal Transduction and Targeted Therapy · doi:10.1038/s41392-020-0207-x · Cited by 1,586 · see citing works

Emerging concepts of miRNA therapeutics: from cells to clinic

Diener, C., Keller, A., & Meese, E. U. (2022) · Trends in Genetics · doi:10.1016/j.tig.2022.02.006 · Cited by 981 · 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

Expanding RNAi therapeutics to extrahepatic tissues with lipophilic conjugates

Brown, K. M., Nair, J. K., Janas, M. M., Anglero-Rodriguez, Y. I., Dang, L. T. H., Peng, H., Theile, C. S., Castellanos-Rizaldos, E., Brown, C., Foster, D., et al. (2022) · Nature Biotechnology · doi:10.1038/s41587-022-01334-x · Cited by 258 · see citing works

A divalent siRNA chemical scaffold for potent and sustained modulation of gene expression throughout the central nervous system

Alterman, J. F., Godinho, B. M. D. C., Hassler, M. R., Ferguson, C. M., Echeverria, D., Sapp, E., Haraszti, R. A., Coles, A. H., Conroy, F., Miller, R., et al. (2019) · Nature Biotechnology · doi:10.1038/s41587-019-0205-0 · Cited by 227 · see citing works

Diverse lipid conjugates for functional extra-hepatic siRNA delivery in vivo

Biscans, A., Coles, A. H., Haraszti, R. Á., Echeverria, D., Hassler, M. R., Osborn, M. F., & Khvorova, A. (2018) · Nucleic Acids Research · doi:10.1093/nar/gky1239 · Cited by 206 · see citing works

Derivatization with fatty acids in peptide and protein drug discovery

Kurtzhals, P., Østergaard, S., Nishimura, E., & Kjeldsen, T. (2022) · Nature Reviews Drug Discovery · doi:10.1038/s41573-022-00529-w · Cited by 137 · see citing works

A perspective on oligonucleotide therapy: Approaches to patient customization

Thakur, S., Sinhari, A., Jain, P., & Jadhav, H. R. (2022) · Frontiers in Pharmacology · doi:10.3389/fphar.2022.1006304 · Cited by 114 · see citing works

Partial inhibition of the overactivated Ku80-dependent DNA repair pathway rescues neurodegeneration in C9ORF72 -ALS/FTD

Lopez-Gonzalez, R., Yang, D., Pribadi, M., Kim, T. S., Krishnan, G., Choi, S. Y., Lee, S., Coppola, G., & Gao, F. (2019) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1901313116 · Cited by 86 · see citing works

Docosanoic acid conjugation to siRNA enables functional and safe delivery to skeletal and cardiac muscles

Biscans, A., Caiazzi, J., McHugh, N., Hariharan, V. N., Muhuri, M., & Khvorova, A. (2020) · Molecular Therapy · doi:10.1016/j.ymthe.2020.12.023 · Cited by 75 · see citing works

The chemical structure and phosphorothioate content of hydrophobically modified siRNAs impact extrahepatic distribution and efficacy

Biscans, A., Caiazzi, J., Davis, S. M., McHugh, N., Sousa, J., & Khvorova, A. (2020) · Nucleic Acids Research · doi:10.1093/nar/gkaa595 · Cited by 68 · see citing works

Advances in the Design of (Nano)Formulations for Delivery of Antisense Oligonucleotides and Small Interfering RNA: Focus on the Central Nervous System

Mendonça, M. C. P., Kont, A., Aburto, M. R., Cryan, J. F., & O’Driscoll, C. M. (2021) · Molecular Pharmaceutics · doi:10.1021/acs.molpharmaceut.0c01238 · Cited by 73 · see citing works

Recent Advances and Prospects in RNA Drug Development

Tani, H. (2024) · International Journal of Molecular Sciences · doi:10.3390/ijms252212284 · Cited by 73 · see citing works

A critical analysis of methods used to investigate the cellular uptake and subcellular localization of RNA therapeutics

Deprey, K. L., Batistatou, N., & Kritzer, J. A. (2020) · Nucleic Acids Research · doi:10.1093/nar/gkaa576 · Cited by 68 · see citing works

Topical siRNA delivery to the cornea and anterior eye by hybrid silicon-lipid nanoparticles

Rachwalska, P., Torabi‐Pour, N., Sutera, F. M., Ahmed, M. H., Thomas, K. G., Nesbit, M. A., Welsh, M., Moore, T., & Saffie-Siebert, S. R. (2020) · Journal of Controlled Release · doi:10.1016/j.jconrel.2020.07.004 · Cited by 64 · see citing works

The valency of fatty acid conjugates impacts siRNA pharmacokinetics, distribution, and efficacy in vivo

Biscans, A., Coles, A. H., Echeverria, D., & Khvorova, A. (2019) · Journal of Controlled Release · doi:10.1016/j.jconrel.2019.03.028 · Cited by 64 · see citing works

Di-valent siRNA-mediated silencing of MSH3 blocks somatic repeat expansion in mouse models of Huntington’s disease

O’Reilly, D., Belgrad, J., Ferguson, C. M., Summers, A., Sapp, E., McHugh, C. A., Mathews, E. W., Boudi, A., Buchwald, J. E., Ly, S., et al. (2023) · Molecular Therapy · doi:10.1016/j.ymthe.2023.05.006 · Cited by 60 · see citing works

Targeted delivery of RNAi to cancer cells using RNA-ligand displaying exosome

Uddin, N., Binzel, D. W., Shu, D., Fu, T., & Guo, P. (2022) · Acta Pharmaceutica Sinica B · doi:10.1016/j.apsb.2022.11.019 · Cited by 61 · see citing works

Therapeutic RNA-silencing oligonucleotides in metabolic diseases

Goga, A., & Stoffel, M. (2022) · Nature Reviews Drug Discovery · doi:10.1038/s41573-022-00407-5 · Cited by 58 · see citing works

Molecularly engineered siRNA conjugates for tumor-targeted RNAi therapy

Lee, J. W., Choi, J., Choi, Y., Kim, K., Yang, Y., Kim, S. H., Yoon, H. Y., & Kwon, I. C. (2022) · Journal of Controlled Release · doi:10.1016/j.jconrel.2022.09.040 · Cited by 55 · see citing works

Innovative developments and emerging technologies in RNA therapeutics

Halloy, F., Biscans, A., Bujold, K. E., Debacker, A. J., Hill, A. C., Lacroix, A., Luige, O., Strömberg, R., Sundström, L., Vogel, J., et al. (2022) · RNA Biology · doi:10.1080/15476286.2022.2027150 · Cited by 54 · see citing works

Structural optimization of siRNA conjugates for albumin binding achieves effective MCL1-directed cancer therapy

Hoogenboezem, E. N., Patel, S. S., Lo, J. H., Cavnar, A. B., Babb, L. M., Francini, N., Gbur, E. F., Patil, P., Colazo, J. M., Michell, D. L., et al. (2024) · Nature Communications · doi:10.1038/s41467-024-45609-0 · Cited by 52 · see citing works

Considerations and recommendations for assessment of plasma protein binding and drug–drug interactions for siRNA therapeutics

Humphreys, S. C., Davis, J. A., Iqbal, S., Kamel, A. M., Kulmatycki, K. M., Lao, Y., Liu, X., Rodgers, J., Snoeys, J., Vigil, A., et al. (2022) · Nucleic Acids Research · doi:10.1093/nar/gkac456 · Cited by 50 · see citing works

Engineered ionizable lipid siRNA conjugates enhance endosomal escape but induce toxicity in vivo

Biscans, A., Ly, S., McHugh, N., Cooper, D., & Khvorova, A. (2022) · Journal of Controlled Release · doi:10.1016/j.jconrel.2022.07.041 · Cited by 50 · see citing works

Nanotechnology, equity and global health

Salamanca‐Buentello, F., & Daar, A. S. (2021) · Nature Nanotechnology · doi:10.1038/s41565-021-00899-z · Cited by 50 · see citing works

Engineered Polymer–siRNA Polyplexes Provide Effective Treatment of Lung Inflammation

Jeon, T., Luther, D. C., Goswami, R., Bell, C. J., Nagaraj, H., Çiçek, Y. A., Huang, R., Mas‐Rosario, J. A., Elia, J. L., Im, J., et al. (2023) · ACS Nano · doi:10.1021/acsnano.2c08690 · Cited by 54 · see citing works

Targeting RNA with synthetic oligonucleotides: Clinical success invites new challenges

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Divalent siRNAs are bioavailable in the lung and efficiently block SARS-CoV-2 infection

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