siRNA versus miRNA as therapeutics for gene silencing

MicroRNAs: Target Recognition and Regulatory Functions

Bartel, D. P. (2009) · Cell · doi:10.1016/j.cell.2009.01.002 · Cited by 20,068 · 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

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

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

Oncomirs — microRNAs with a role in cancer

Esquela‐Kerscher, A., & Slack, F. J. (2006) · Nature reviews. Cancer · doi:10.1038/nrc1840 · Cited by 7,038 · see citing works

Regulation of microRNA biogenesis

Ha, M., & Kim, V. N. (2014) · Nature Reviews Molecular Cell Biology · doi:10.1038/nrm3838 · Cited by 5,462 · see citing works

Microarray analysis shows that some microRNAs downregulate large numbers of target mRNAs

Lim, L. P., Lau, N. C., Garrett-Engele, P., Grimson, A., Schelter, J. M., Castle, J., Bartel, D. P., Linsley, P. S., & Johnson, J. M. (2005) · Nature · doi:10.1038/nature03315 · Cited by 4,755 · see citing works

Species-specific recognition of single-stranded RNA via toll-like receptor 7 and 8

Heil, F., Hemmi, H., Hochrein, H., Ampenberger, F., Kirschning, C., Akira, S., Lipford, G., Wagner, H., & Bauer, S. (2004) · Science · doi:10.1126/science.1093620 · Cited by 3,947 · see citing works

RNA interference is mediated by 21- and 22-nucleotide RNAsPreprint

Elbashir, S. M., Lendeckel, W., & Tuschl, T. (2001) · Genes & Development · doi:10.1101/gad.862301 · Cited by 3,310 · see citing works

Biogenesis of small RNAs in animals

Kim, V. N., Han, J., & Siomi, M. C. (2009) · Nature Reviews Molecular Cell Biology · doi:10.1038/nrm2632 · Cited by 3,181 · see citing works

Non-viral vectors for gene-based therapy

Yin, H., Kanasty, R. L., Eltoukhy, A. A., Vegas, A. J., Dorkin, J. R., & Anderson, D. G. (2014) · Nature Reviews Genetics · doi:10.1038/nrg3763 · Cited by 3,123 · see citing works

miRBase: integrating microRNA annotation and deep-sequencing data

Kozomara, A., & Griffiths-Jones, S. (2011) · Nucleic Acids Research · doi:10.1093/nar/gkq1027 · Cited by 3,042 · see citing works

Role of the Vascular Endothelial Growth Factor Pathway in Tumor Growth and Angiogenesis

Hicklin, D. J., & Ellis, L. M. (2004) · Journal of Clinical Oncology · doi:10.1200/jco.2005.06.081 · Cited by 3,010 · see citing works

Functional siRNAs and miRNAs exhibit strand bias

Khvorova, A., Reynolds, A., & Jayasena, S. D. (2003) · Cell · doi:10.1016/s0092-8674(03)00801-8 · Cited by 2,645 · see citing works

Evidence of RNAi in humans from systemically administered siRNA via targeted nanoparticles

Davis, M. E., Zuckerman, J. E., Choi, C. H. J., Seligson, D. B., Tolcher, A. W., Alabi, C. A., Yen, Y., Heidel, J. D., & Ribas, A. (2010) · Nature · doi:10.1038/nature08956 · Cited by 2,508 · see citing works

Non-coding RNA

Mattick, J. S., & Makunin, I. V. (2006) · Human Molecular Genetics · doi:10.1093/hmg/ddl046 · Cited by 2,471 · see citing works

Gene silencing by microRNAs: contributions of translational repression and mRNA decay

Huntzinger, E., & Izaurralde, E. (2011) · Nature Reviews Genetics · doi:10.1038/nrg2936 · Cited by 2,339 · 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

Toxicity of cationic lipids and cationic polymers in gene delivery

Lv, H., Zhang, S., Wang, B., Cui, S., & Yan, J. (2006) · Journal of Controlled Release · doi:10.1016/j.jconrel.2006.04.014 · Cited by 2,286 · 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

MicroRNAs in cancer: biomarkers, functions and therapy

Hayes, J., Peruzzi, P. P., & Lawler, S. (2014) · Trends in Molecular Medicine · doi:10.1016/j.molmed.2014.06.005 · Cited by 2,049 · see citing works

Cyclodextrin-based pharmaceutics: past, present and future

Davis, M. E., & Brewster, M. E. (2004) · Nature Reviews Drug Discovery · doi:10.1038/nrd1576 · Cited by 2,002 · see citing works

Control of translation and mRNA degradation by miRNAs and siRNAs: Table 1.

Valencia-Sanchez, M. A., Liu, J., Hannon, G. J., & Parker, R. (2006) · Genes & Development · doi:10.1101/gad.1399806 · Cited by 1,751 · see citing works

MicroRNA dysregulation in cancer: diagnostics, monitoring and therapeutics. A comprehensive review

Iorio, M. V., & Croce, C. M. (2012) · EMBO Molecular Medicine · doi:10.1002/emmm.201100209 · Cited by 1,738 · see citing works

Therapeutic microRNA Delivery Suppresses Tumorigenesis in a Murine Liver Cancer Model

Kota, J., Chivukula, R. R., O’Donnell, K. A., Wentzel, E. A., Montgomery, C. L., Hwang, H., Chang, T.-C., Vivekanandan, P., Torbenson, M. S., Clark, K. R., et al. (2009) · Cell · doi:10.1016/j.cell.2009.04.021 · Cited by 1,711 · see citing works

Fatality in mice due to oversaturation of cellular microRNA/short hairpin RNA pathways

Grimm, D., Streetz, K. L., Jopling, C. L., Storm, T. A., Pandey, K., Davis, C. R., Marion, P., Salazar, F., & Kay, M. A. (2006) · Nature · doi:10.1038/nature04791 · Cited by 1,677 · see citing works

Activation of the interferon system by short-interfering RNAs

Sledz, C. A., Holko, M., de Veer, M. J., Silverman, R. H., & Williams, B. R. G. (2003) · Nature Cell Biology · doi:10.1038/ncb1038 · Cited by 1,401 · see citing works

RNA Interference: Biology, Mechanism, and Applications

Agrawal, N., Dasaradhi, P. V. N., Mohmmed, A., Malhotra, P., Bhatnagar, R. K., & Mukherjee, S. K. (2003) · Microbiology and Molecular Biology Reviews · doi:10.1128/mmbr.67.4.657-685.2003 · Cited by 1,312 · see citing works

siRNAs can function as miRNAsPreprint

Doench, J. G., Petersen, C. P., & Sharp, P. A. (2003) · Genes & Development · doi:10.1101/gad.1064703 · Cited by 1,262 · see citing works

Sequence-dependent stimulation of the mammalian innate immune response by synthetic siRNA

Judge, A. D., Sood, V., Shaw, J. R., Fang, D., McClintock, K., & MacLachlan, I. (2005) · Nature Biotechnology · doi:10.1038/nbt1081 · Cited by 1,250 · see citing works

Sequence-specific potent induction of IFN-α by short interfering RNA in plasmacytoid dendritic cells through TLR7

Hornung, V., Guenthner‐Biller, M., Bourquin, C., Ablasser, A., Schlee, M., Uematsu, S., Noronha, A. M., Manoharan, M., Akira, S., de Fougerolles, A., et al. (2005) · Nature Medicine · doi:10.1038/nm1191 · Cited by 1,233 · see citing works

Polo-like kinases and the orchestration of cell division

Barr, F. A., Silljé, H. H. W., & Nigg, E. A. (2004) · Nature Reviews Molecular Cell Biology · doi:10.1038/nrm1401 · Cited by 1,092 · see citing works

Recognizing and avoiding siRNA off-target effects for target identification and therapeutic application

Jackson, A. L., & Linsley, P. S. (2009) · Nature Reviews Drug Discovery · doi:10.1038/nrd3010 · Cited by 1,092 · 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

Strategies for silencing human disease using RNA interference

Kim, D. H., & Rossi, J. J. (2007) · Nature Reviews Genetics · doi:10.1038/nrg2006 · Cited by 1,078 · see citing works

Argonaute proteins: functional insights and emerging roles

Meister, G. (2013) · Nature Reviews Genetics · doi:10.1038/nrg3462 · Cited by 1,008 · see citing works

Dendrimers in gene delivery

Dufès, C., Uchegbu, I. F., & Schätzlein, A. G. (2005) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2005.09.017 · Cited by 984 · 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

Induction of an interferon response by RNAi vectors in mammalian cells

Bridge, A. J., Kaeser-Pebernard, S., Ducraux, A., Nicoulaz, A.-L., & Iggo, R. D. (2003) · Nature Genetics · doi:10.1038/ng1173 · Cited by 962 · see citing works

Widespread siRNA “off-target” transcript silencing mediated by seed region sequence complementarity

Jackson, A. L., Burchard, J., Schelter, J. M., Chau, B. N., Cleary, M. A., Lim, L. P., & Linsley, P. S. (2006) · RNA · doi:10.1261/rna.25706 · Cited by 953 · see citing works

Sequence- and target-independent angiogenesis suppression by siRNA via TLR3

Kleinman, M. E., Yamada, K., Takeda, A., Chandrasekaran, V., Nozaki, M., Baffi, J., Albuquerque, R., Yamasaki, S., Itaya, M., Pan, Y., et al. (2008) · Nature · doi:10.1038/nature06765 · Cited by 931 · 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

siRNA function in RNAi: A chemical modification analysis

Chiu, Y., & Rana, T. M. (2003) · RNA · doi:10.1261/rna.5103703 · Cited by 863 · see citing works

Suppression of non-small cell lung tumor development by the let-7 microRNA family

Kumar, M. S., Erkeland, S. J., Pester, R. E., Chen, C. Y., Ebert, M. S., Sharp, P. A., & Jacks, T. (2008) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.0712321105 · Cited by 844 · see citing works

RNA interference in the clinic: challenges and future directions

Pecot, C. V., Calin, G. A., Coleman, R. L., Lopez‐Berestein, G., & Sood, A. K. (2010) · Nature reviews. Cancer · doi:10.1038/nrc2966 · Cited by 822 · see citing works

Current prospects for RNA interference-based therapies

Davidson, B. L., & McCray, P. B. (2011) · Nature Reviews Genetics · doi:10.1038/nrg2968 · Cited by 760 · see citing works

miR-34 – a microRNA replacement therapy is headed to the clinic

Bader, A. (2012) · Front. Gene. · doi:10.3389/fgene.2012.00120 · Cited by 728 · see citing works

RNAi-mediated gene-targeting through systemic application of polyethylenimine (PEI)-complexed siRNA in vivo

Urban-Klein, B., Werth, S., Abuharbeid, S., Czubayko, F., & Aigner, A. M. (2004) · Gene Therapy · doi:10.1038/sj.gt.3302425 · Cited by 714 · see citing works

First-in-Humans Trial of an RNA Interference Therapeutic Targeting VEGF and KSP in Cancer Patients with Liver Involvement

Tabernero, J. M., Shapiro, G. I., LoRusso, P. M., Cervantes, A. M. R., Schwartz, G. K., Weiss, G. J., Paz-Ares, L. G., Cho, D. C., Infante, J. R., Alsina, M., et al. (2013) · Cancer Discovery · doi:10.1158/2159-8290.cd-12-0429 · Cited by 700 · 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

Progress towards in Vivo Use of siRNAs

Behlke, M. A. (2006) · Molecular Therapy · doi:10.1016/j.ymthe.2006.01.001 · Cited by 677 · see citing works

Systemic Delivery of Tumor Suppressor microRNA Mimics Using a Neutral Lipid Emulsion Inhibits Lung Tumors in Mice

Trang, P., Wiggins, J. F., Daige, C. L., Cho, C., Omotola, M., Brown, D., Weidhaas, J. B., Bader, A. G., & Slack, F. J. (2011) · Molecular Therapy · doi:10.1038/mt.2011.48 · Cited by 669 · see citing works

siRNA vs. shRNA: Similarities and differences

Rao, D. D., Vorhies, J. S., Senzer, N. N., & Nemunaitis, J. J. (2009) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2009.04.004 · Cited by 658 · see citing works

miR-15a and miR-16-1 in cancer: discovery, function and future perspectives

Aqeilan, R. I., Calin, G. A., & Croce, C. M. (2009) · Cell Death and Differentiation · doi:10.1038/cdd.2009.69 · Cited by 650 · see citing works

Development of microRNA therapeutics is coming of age

van Rooij, E., & Kauppinen, S. (2014) · EMBO Molecular Medicine · doi:10.15252/emmm.201100899 · Cited by 625 · see citing works

The Promise of MicroRNA Replacement Therapy

Bader, A. G., Brown, D., & Winkler, M. (2010) · Cancer Research · doi:10.1158/0008-5472.can-10-2010 · Cited by 618 · see citing works

Short interfering RNAs can induce unexpected and divergent changes in the levels of untargeted proteins in mammalian cells

Scacheri, P. C., Rozenblatt–Rosen, O., Caplen, N. J., Wolfsberg, T. G., Umayam, L. A., Lee, J. C., Hughes, C. M., Shanmugam, K. S., Bhattacharjee, A., Meyerson, M. M., et al. (2004) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.0308698100 · Cited by 613 · see citing works

Expanding the MicroRNA Targeting Code: Functional Sites with Centered Pairing

Shin, C., Nam, J.-W., Farh, K. K.-H., Chiang, H. R., Shkumatava, A., & Bartel, D. P. (2010) · Molecular Cell · doi:10.1016/j.molcel.2010.06.005 · Cited by 611 · see citing works

Progress in microRNA delivery

Zhang, Y., Wang, Z. J., & Gemeinhart, R. A. (2013) · Journal of Controlled Release · doi:10.1016/j.jconrel.2013.09.015 · Cited by 602 · see citing works

Vitamin E TPGS as a molecular biomaterial for drug delivery

Zhang, Z., Tan, S., & Feng, S. (2012) · Biomaterials · doi:10.1016/j.biomaterials.2012.03.046 · Cited by 586 · see citing works

In vivo activity of nuclease-resistant siRNAs

Layzer, J. M., McCaffrey, A. P., Tanner, A. K., Huang, Z., Kay, M. A., & Sullenger, B. A. (2004) · RNA · doi:10.1261/rna.5239604 · Cited by 581 · see citing works

Effect of an RNA interference drug on the synthesis of proprotein convertase subtilisin/kexin type 9 (PCSK9) and the concentration of serum LDL cholesterol in healthy volunteers: a randomised, single-blind, placebo-controlled, phase 1 trial

Fitzgerald, K. J., Frank-Kamenetsky, M. D., Shulga‐Morskaya, S., Liebow, A., Bettencourt, B. R., Sutherland, J. E., Hutabarat, R. M., Clausen, V. A., Karsten, V., Cehelsky, J. E., et al. (2013) · The Lancet · doi:10.1016/s0140-6736(13)61914-5 · Cited by 574 · see citing works

Design of Noninflammatory Synthetic siRNA Mediating Potent Gene Silencing in Vivo

Judge, A. D., Bola, G., Lee, A. C. H., & MacLachlan, I. (2005) · Molecular Therapy · doi:10.1016/j.ymthe.2005.11.002 · Cited by 571 · see citing works

Sequence-Specific Knockdown of EWS-FLI1 by Targeted, Nonviral Delivery of Small Interfering RNA Inhibits Tumor Growth in a Murine Model of Metastatic Ewing's Sarcoma

Hu‐Lieskovan, S., Heidel, J. D., Bartlett, D. W., Davis, M. E., & Triche, T. J. (2005) · Cancer Research · doi:10.1158/0008-5472.can-05-0565 · Cited by 568 · 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

Tolerance for mutations and chemical modifications in a siRNA

Amarzguioui, M. (2003) · Nucleic Acids Research · doi:10.1093/nar/gkg147 · Cited by 534 · see citing works

RNA Interference in Mammalian Cells by Chemically-Modified RNA

Braasch, D. A., Jensen, S., Liu, Y., Kaur, K. J., Arar, K., White, M. A., & Corey, D. R. (2003) · Biochemistry · doi:10.1021/bi0343774 · Cited by 531 · see citing works

Structure of the guide-strand-containing argonaute silencing complex

Wang, Y., Sheng, G., Juranek, S., Tuschl, T., & Patel, D. J. (2008) · Nature · doi:10.1038/nature07315 · Cited by 531 · see citing works

Nanoparticles Modified With Tumor-targeting scFv Deliver siRNA and miRNA for Cancer Therapy

Chen, Y., Zhu, X., Zhang, X., Liu, B., & Huang, L. (2010) · Molecular Therapy · doi:10.1038/mt.2010.136 · Cited by 516 · see citing works

Hyaluronic acid-chitosan nanoparticles for co-delivery of MiR-34a and doxorubicin in therapy against triple negative breast cancer

Deng, X., Cao, M., Zhang, J., Hu, K., Yin, Z., Zhou, Z., Xiao, X., Yang, Y., Sheng, W., Wu, Y. F., et al. (2014) · Biomaterials · doi:10.1016/j.biomaterials.2014.02.006 · Cited by 515 · see citing works

Developing MicroRNA Therapeutics

van Rooij, E., Purcell, A. L., & Levin, A. A. (2012) · Circulation Research · doi:10.1161/circresaha.111.247916 · Cited by 498 · see citing works

Off-target effects by siRNA can induce toxic phenotype

Federov, Y., Anderson, E. M., Birmingham, A., Reynolds, A., Karpilow, J. M., Robinson, K., Leake, D., Marshall, W. S., & Khvorova, A. (2006) · RNA · doi:10.1261/rna.28106 · Cited by 493 · see citing works

Polo-like kinase (Plk)1 depletion induces apoptosis in cancer cells

Liu, X., & Erikson, R. L. (2003) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1031523100 · Cited by 474 · see citing works

First microRNA mimic enters clinic

Bouchie, A. J. (2013) · Nature Biotechnology · doi:10.1038/nbt0713-577 · Cited by 471 · see citing works

siRNA and Innate Immunity

Robbins, M., Judge, A. D., & MacLachlan, I. (2009) · Oligonucleotides · doi:10.1089/oli.2009.0180 · Cited by 453 · 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

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

Synthetic shRNAs as potent RNAi triggers

Siolas, D., Lerner, C. G., Burchard, J., Ge, W., Linsley, P. S., Paddison, P. J., Hannon, G. J., & Cleary, M. A. (2004) · Nature Biotechnology · doi:10.1038/nbt1052 · Cited by 445 · see citing works

Liposomal siRNA nanocarriers for cancer therapy

Özpolat, B., Sood, A. K., & Lopez‐Berestein, G. (2014) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2013.12.008 · Cited by 445 · see citing works

A randomized, double-blind, placebo-controlled study of an RNAi-based therapy directed against respiratory syncytial virus

DeVincenzo, J. P., Lambkin‐Williams, R., Wilkinson, T. M. A., Cehelsky, J. E., Nochur, S., Walsh, E. E., Meyers, R. E., Gollob, J., & Vaishnaw, A. K. (2010) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.0912186107 · Cited by 440 · see citing works

Correlating animal and human phase Ia/Ib clinical data with CALAA-01, a targeted, polymer-based nanoparticle containing siRNA

Zuckerman, J. E., Gritli, I., Tolcher, A. W., Heidel, J. D., Lim, D. W., Morgan, R. J., Chmielowski, B., Ribas, A., Davis, M. E., & Yen, Y. (2014) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1411393111 · Cited by 436 · see citing works

Small RNAs with Imperfect Match to Endogenous mRNA Repress Translation

Saxena, S., Jónsson, Z. O., & Dutta, A. (2003) · Journal of Biological Chemistry · doi:10.1074/jbc.m307089200 · Cited by 436 · see citing works

Chemically modified siRNA: tools and applications

Watts, J. K., Deleavey, G. F., & Damha, M. J. (2008) · Drug Discovery Today · doi:10.1016/j.drudis.2008.05.007 · Cited by 431 · see citing works

Systemic Delivery of Synthetic MicroRNA-16 Inhibits the Growth of Metastatic Prostate Tumors via Downregulation of Multiple Cell-cycle Genes

Takeshita, F., Patrawala, L., Osaki, M., Takahashi, R., Yamamoto, Y., Kosaka, N., Kawamata, M., Kelnar, K., Bader, A. G., Brown, D., et al. (2009) · Molecular Therapy · doi:10.1038/mt.2009.207 · Cited by 420 · see citing works

MicroRNA replacement therapy for miR-145 and miR-33a is efficacious in a model of colon carcinoma.

Ibrahim, A., Weirauch, U., Thomas, M., Grünweller, A., Hartmann, R. K., & Aigner, A. (2011) · Cancer Research · doi:10.1158/0008-5472.can-10-4645 · Cited by 398 · see citing works

MicroRNA therapeutics

Broderick, J. A., & Zamore, P. D. (2011) · Gene Therapy · doi:10.1038/gt.2011.50 · Cited by 387 · see citing works

Anti-miRNA oligonucleotides (AMOs): ammunition to target miRNAs implicated in human disease?

Weiler, J., Hunziker, J., & Hall, J. (2005) · Gene Therapy · doi:10.1038/sj.gt.3302654 · Cited by 371 · see citing works

siRNA-mediated off-target gene silencing triggered by a 7 nt complementation

Lin, X. (2005) · Nucleic Acids Research · doi:10.1093/nar/gki762 · Cited by 368 · see citing works

Mutagenesis and Oncogenesis by Chromosomal Insertion of Gene Transfer Vectors

Baum, C., Kustikova, O. S., Modlich, U., Li, Z., & Fehse, B. (2006) · Human Gene Therapy · doi:10.1089/hum.2006.17.253 · Cited by 360 · see citing works

Chemical modification of siRNAs to improve serum stability without loss of efficacy

Choung, S., Kim, Y., Kim, S., Park, H.-O., & Choi, Y. (2006) · Biochemical and Biophysical Research Communications · doi:10.1016/j.bbrc.2006.02.049 · Cited by 348 · see citing works

Biological gene delivery vehicles: beyond viral vectors.

Seow, Y., & Wood, M. (2009) · Molecular Therapy · doi:10.1038/mt.2009.41 · Cited by 339 · see citing works

Developing therapeutic microRNAs for cancer

Bader, A. G., Brown, D., Stoudemire, J., & Lammers, P. (2011) · Gene Therapy · doi:10.1038/gt.2011.79 · Cited by 335 · see citing works

A large-scale chemical modification screen identifies design rules to generate siRNAs with high activity, high stability and low toxicity

Bramsen, J. B., Laursen, M. B., Nielsen, A. F., Hansen, T. B., Bus, C., Langkjær, N., Babu, B. R., Højland, T., Abramov, M., Van Aerschot, A., et al. (2009) · Nucleic Acids Research · doi:10.1093/nar/gkp106 · Cited by 332 · see citing works

An alternative mode of microRNA target recognition

Chi, S. W., Hannon, G. J., & Darnell, R. B. (2012) · Nature Structural & Molecular Biology · doi:10.1038/nsmb.2230 · Cited by 328 · see citing works

The impact of target site accessibility on the design of effective siRNAs

Tafer, H., Ameres, S. L., Obernosterer, G., et al. (2008) · Nature Biotechnology · doi:10.1038/nbt1404 · Cited by 309 · see citing works

An RNAi therapeutic targeting antithrombin to rebalance the coagulation system and promote hemostasis in hemophilia

Sehgal, A., Barros, S. A., Ivanciu, L., Cooley, B. C., Qin, J., Racie, T., Hettinger, J. L., Carioto, M., Jiang, Y., Brodsky, J., et al. (2015) · Nature Medicine · doi:10.1038/nm.3847 · Cited by 307 · see citing works

Therapeutic potentials of gene silencing by RNA interference: Principles, challenges, and new strategies

Deng, Y. P., Wang, C. C., Choy, K. W., Du, Q., Chen, J., Wang, Q., Lu, L., Chung, T. K. H., & Tang, T. (2014) · Gene · doi:10.1016/j.gene.2013.12.019 · Cited by 302 · see citing works

Atu027, a Liposomal Small Interfering RNA Formulation Targeting Protein Kinase N3, Inhibits Cancer Progression

Aleku, M., Schulz, P. M., Keil, O., Santel, A., Schaeper, U., Dieckhoff, B., Janke, O., Endruschat, J., Durieux, B., Röder, N., et al. (2008) · Cancer Research · doi:10.1158/0008-5472.can-08-2428 · Cited by 293 · see citing works

Delivering the promise of miRNA cancer therapeutics

Pereira, D. M., Rodrigues, P. M., Borralho, P. M., & Rodrigues, C. M. P. (2012) · Drug Discovery Today · doi:10.1016/j.drudis.2012.10.002 · Cited by 288 · see citing works

Silica-based nanoparticles for biomedical applications

Bitar, A., Ahmad, N. M., Fessi, H., & Elaı̈ssari, A. (2012) · Drug Discovery Today · doi:10.1016/j.drudis.2012.06.014 · Cited by 287 · see citing works

RNA interference using boranophosphate siRNAs: structure-activity relationships

Hall, A. (2004) · Nucleic Acids Research · doi:10.1093/nar/gkh936 · Cited by 286 · see citing works

Positional Effect of Chemical Modifications on Short Interference RNA Activity in Mammalian Cells

Prakash, T. P., Allerson, C. R., Dande, P., Vickers, T. A., Sioufi, N. B., Jarres, R., Baker, B. F., Swayze, E. E., Griffey, R. H., & Bhat, B. (2005) · Journal of Medicinal Chemistry · doi:10.1021/jm050044o · Cited by 285 · see citing works

Ribonucleotide Reductase Inhibitors and Future Drug Design

Shao, J., Zhou, B., Chu, B. W., & Yen, Y. (2006) · Current Cancer Drug Targets · doi:10.2174/156800906777723949 · Cited by 284 · see citing works

Induction of apoptosis by an inhibitor of the mitotic kinesin KSP requires both activation of the spindle assembly checkpoint and mitotic slippage

Tao, W., South, V. J., Zhang, Y. L., Davide, J. P., Farrell, L., Kohl, N. E., Sepp‐Lorenzino, L., & Lobell, R. B. (2005) · Cancer Cell · doi:10.1016/j.ccr.2005.06.003 · Cited by 275 · see citing works

Comparison of different antisense strategies in mammalian cells using locked nucleic acids, 2'-O-methyl RNA, phosphorothioates and small interfering RNA

Grünweller, A. (2003) · Nucleic Acids Research · doi:10.1093/nar/gkg409 · Cited by 269 · see citing works

The RNA-Induced Silencing Complex Is a Mg2+-Dependent Endonuclease

Schwarz, D. S., Tomari, Y., & Zamore, P. D. (2004) · Current Biology · doi:10.1016/j.cub.2004.03.008 · Cited by 268 · see citing works

Tumor-targeting dual peptides-modified cationic liposomes for delivery of siRNA and docetaxel to gliomas

Yang, Z., Li, J., Wang, Z.-Z., Dong, D., & Qi, X. (2014) · Biomaterials · doi:10.1016/j.biomaterials.2014.03.017 · Cited by 267 · see citing works

The response of mammalian cells to double-stranded RNA

Gantier, M. P., & Williams, B. R. G. (2007) · Cytokine & Growth Factor Reviews · doi:10.1016/j.cytogfr.2007.06.016 · Cited by 266 · see citing works

First-in-human Mutation-targeted siRNA Phase Ib Trial of an Inherited Skin Disorder

Leachman, S. A., Hickerson, R. P., Schwartz, M. E., Bullough, E. E., Hutcherson, S. L., Boucher, K. M., Hansen, C. D., Eliason, M. J., Srivatsa, G. S., Kornbrust, D. J., et al. (2009) · Molecular Therapy · doi:10.1038/mt.2009.273 · Cited by 263 · see citing works

Role of anti-oncomirs miR-143 and -145 in human colorectal tumors

Akao, Y., Nakagawa, Y., Hirata, I., et al. (2010) · Cancer Gene Therapy · doi:10.1038/cgt.2009.88 · Cited by 262 · see citing works

First-in-Human Phase I Study of the Liposomal RNA Interference Therapeutic Atu027 in Patients With Advanced Solid Tumors

Schultheis, B. S., Strumberg, D., Santel, A., Vank, C., Gebhardt, F., Keil, O., Lange, C. M., Giese, K., Kaufmann, J., Khan, M., et al. (2014) · Journal of Clinical Oncology · doi:10.1200/jco.2013.55.0376 · Cited by 262 · see citing works

Postexposure Protection of Guinea Pigs against a Lethal Ebola Virus Challenge Is Conferred by RNA Interference

Geisbert, T. W., Hensley, L. E., Kagan, E., Yu, E. Z., Geisbert, J. B., Daddario-DiCaprio, K. M., Fritz, E. A., Jahrling, P. B., McClintock, K., Phelps, J. R., et al. (2006) · The Journal of Infectious Diseases · doi:10.1086/504267 · Cited by 254 · 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

Evaluation of the safety, tolerability and pharmacokinetics of ALN-RSV01, a novel RNAi antiviral therapeutic directed against respiratory syncytial virus (RSV)

DeVincenzo, J. P., Cehelsky, J., Alvarez, R., Elbashir, S. M., Harborth, J., Toudjarska, I., Nechev, L. V., Murugaiah, V., VLIET, A., & Vaishnaw, A. K. (2008) · Antiviral Research · doi:10.1016/j.antiviral.2007.11.009 · Cited by 247 · see citing works

Locked Nucleic Acids: Promising Nucleic Acid Analogs for Therapeutic Applications

Veedu, R. N., & Wengel, J. (2010) · Chemistry & Biodiversity · doi:10.1002/cbdv.200900343 · Cited by 241 · see citing works

Inhibition of cancer stem cell-like properties and reduced chemoradioresistance of glioblastoma using microRNA145 with cationic polyurethane-short branch PEI

Yang, Y.-P., Chien, Y., Chiou, G., Cherng, J., Wang, M., Lo, W., Chang, Y.-L., Huang, P., Chen, Y., Shih, Y., et al. (2011) · Biomaterials · doi:10.1016/j.biomaterials.2011.10.071 · Cited by 238 · see citing works

An efficient and low immunostimulatory nanoparticle formulation for systemic siRNA delivery to the tumor

Chono, S., Li, S., Conwell, C. C., & Huang, L. (2008) · Journal of Controlled Release · doi:10.1016/j.jconrel.2008.07.006 · Cited by 237 · see citing works

Inhibition of Neuroblastoma Tumor Growth by Targeted Delivery of MicroRNA-34a Using Anti-Disialoganglioside GD2 Coated Nanoparticles

Tivnan, A., Orr, W. S., Gubala, V., Nooney, R. I., Williams, D. E., McDonagh, C. M., Prenter, S., Harvey, H. M., Domingo-Fernandéz, R., Bray, I. M., et al. (2012) · PLoS ONE · doi:10.1371/journal.pone.0038129 · Cited by 233 · see citing works

Oligodeoxynucleoside phosphorothioate stability in subcellular extracts, culture media, sera and cerebrospinal fluid

Campbell, J. M., Bacon, T. A., & Wickstrom, E. (1990) · Journal of Biochemical and Biophysical Methods · doi:10.1016/0165-022x(90)90084-p · Cited by 230 · see citing works

Therapeutic Delivery of MicroRNA-29b by Cationic Lipoplexes for Lung Cancer

Wu, Y., Crawford, M., Mao, Y., Lee, R. J., Davis, I. C., Elton, T. S., Lee, L. J., & Nana-Sinkam, S. P. (2013) · Molecular Therapy — Nucleic Acids · doi:10.1038/mtna.2013.14 · Cited by 223 · see citing works

MicroRNAs in neurodegenerative diseases and their therapeutic potential

Junn, E., & Mouradian, M. M. (2011) · Pharmacology & Therapeutics · doi:10.1016/j.pharmthera.2011.10.002 · Cited by 222 · see citing works

A protocol for designing siRNAs with high functionality and specificity

Birmingham, A., Anderson, E. M., Sullivan, K., Reynolds, A., Boese, Q., Leake, D., Karpilow, J. M., & Khvorova, A. (2007) · Nature Protocols · doi:10.1038/nprot.2007.278 · Cited by 222 · see citing works

Pulmonary delivery of therapeutic siRNA

Lam, J., Liang, W., & Chan, H. (2011) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2011.02.006 · Cited by 214 · see citing works

Innate Immunity to Adenovirus

Hendrickx, R., Stichling, N., Koelen, J. A., Kuryk, Ł., Lipiec, A., & Greber, U. F. (2014) · Human Gene Therapy · doi:10.1089/hum.2014.001 · Cited by 212 · see citing works

RNA Interference Therapy in Lung Transplant Patients Infected with Respiratory Syncytial Virus

Zamora, M. R., Budev, M. M., Rolfe, M. W., et al. (2010) · American Journal of Respiratory and Critical Care Medicine · doi:10.1164/rccm.201003-0422oc · Cited by 210 · see citing works

Liposome based systems for systemic siRNA delivery: Stability in blood sets the requirements for optimal carrier design

Buyens, K., Smedt, S. C. D., Braeckmans, K., Demeester, J., Peeters, L., van Grunsven, L. A., du Jeu, X. D. M., Sawant, R. R., Torchilin, V. P., Farkasova, K., et al. (2011) · Journal of Controlled Release · doi:10.1016/j.jconrel.2011.10.009 · Cited by 207 · see citing works

Thermodynamic stability and Watson–Crick base pairing in the seed duplex are major determinants of the efficiency of the siRNA-based off-target effect

Ui‐Tei, K., Naito, Y., Nishi, K., et al. (2008) · Nucleic Acids Research · doi:10.1093/nar/gkn902 · Cited by 207 · see citing works

Polyethylenimines for siRNA and miRNA delivery in vivo

Höbel, S., & Aigner, A. M. (2013) · Wiley Interdisciplinary Reviews Nanomedicine and Nanobiotechnology · doi:10.1002/wnan.1228 · Cited by 201 · see citing works

Liposome–polyethylenimine complexes for enhanced DNA and siRNA delivery

Schäfer, J., Höbel, S., Bakowsky, U., & Aigner, A. M. (2010) · Biomaterials · doi:10.1016/j.biomaterials.2010.05.043 · Cited by 196 · see citing works

Evaluation of locked nucleic acid–modified small interfering RNAin vitroandin vivo

Mook, O. R. F., Baas, F., de Wissel, M. B., & Fluiter, K. (2007) · Molecular Cancer Therapeutics · doi:10.1158/1535-7163.mct-06-0195 · Cited by 182 · see citing works

Genome-wide resources of endoribonuclease-prepared short interfering RNAs for specific loss-of-function studies

Kittler, R., Surendranath, V., Heninger, A., Slabicki, M. M., Theis, M., Putz, G., Franke, K., Caldarelli, A., Grabner, H., Kozak, K., et al. (2007) · Nature Methods · doi:10.1038/nmeth1025 · Cited by 182 · see citing works

Non-covalently functionalized single-walled carbon nanotube for topical siRNA delivery into melanoma

Siu, K. S., Chen, D., Zheng, X., Zhang, X., Johnston, N., Liu, Y., Yuan, K., Koropatnick, J. D., Gillies, E. R., & Min, W.-P. (2014) · Biomaterials · doi:10.1016/j.biomaterials.2013.12.079 · Cited by 175 · see citing works

Targeted Nanomedicine for Suppression of CD44 and Simultaneous Cell Death Induction in Ovarian Cancer: An Optimal Delivery of siRNA and Anticancer Drug

Shah, V., Taratula, O., Garbuzenko, O. B., Taratula, O. R., Rodríguez-Rodríguez, L., & Minko, T. (2013) · Clinical Cancer Research · doi:10.1158/1078-0432.ccr-13-1536 · Cited by 173 · see citing works

RNAi-based nanomedicines for targeted personalized therapy

Daka, A., & Peer, D. (2012) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2012.08.014 · Cited by 168 · see citing works

Impact of tumor‐specific targeting and dosing schedule on tumor growth inhibition after intravenous administration of siRNA‐containing nanoparticles

Bartlett, D. W., & Davis, M. E. (2007) · Biotechnology and Bioengineering · doi:10.1002/bit.21668 · Cited by 164 · see citing works

Recent Developments in Nucleic Acid Delivery with Polyethylenimines

Neuberg, P., & Kichler, A. (2014) · Advances in genetics · doi:10.1016/b978-0-12-800148-6.00009-2 · Cited by 163 · see citing works

Silencing Mutant Huntingtin by Adeno-Associated Virus-Mediated RNA Interference Ameliorates Disease Manifestations in the YAC128 Mouse Model of Huntington's Disease

Stanek, L. M., Sardi, S. P., Mastis, B., Richards, A. R., Treleaven, C. M., Taksir, T. V., Misra, K., Cheng, S. H., & Shihabuddin, L. S. (2014) · Human Gene Therapy · doi:10.1089/hum.2013.200 · Cited by 162 · see citing works

Potent siRNA Inhibitors of Ribonucleotide Reductase Subunit RRM2 Reduce Cell Proliferation In vitro and In vivo

Heidel, J. D., Liu, J. Y.-C., Yen, Y., et al. (2007) · Clinical Cancer Research · doi:10.1158/1078-0432.ccr-06-2218 · Cited by 160 · see citing works

Dual drugs (microRNA-34a and paclitaxel)-loaded functional solid lipid nanoparticles for synergistic cancer cell suppression

Shi, S., Han, L., Deng, L., Zhang, Y., Shen, H., Gong, T., Zhang, Z., & Sun, X. (2014) · Journal of Controlled Release · doi:10.1016/j.jconrel.2014.09.005 · Cited by 159 · see citing works

Lipidic Systems for In Vivo siRNA Delivery

Wu, S. Y., & McMillan, N. A. J. (2009) · The AAPS Journal · doi:10.1208/s12248-009-9140-1 · Cited by 156 · see citing works

Recent Advances in Nanoparticle-Mediated siRNA Delivery

Williford, J., Wu, J., Ren, Y., Archang, M. M., Leong, K. W., & Mao, H. (2014) · Annual Review of Biomedical Engineering · doi:10.1146/annurev-bioeng-071813-105119 · Cited by 156 · see citing works

Many commonly used siRNAs risk off-target activity

Snøve, O., & Holen, T. (2004) · Biochemical and Biophysical Research Communications · doi:10.1016/j.bbrc.2004.04.175 · Cited by 149 · see citing works

MicroRNA gets down to business

Mack, G. S. (2007) · Nature Biotechnology · doi:10.1038/nbt0607-631 · Cited by 146 · see citing works

Current Pharmaceutical Design

(2019) · Current Pharmaceutical Design · doi:10.2174/cpd-13816128-606 · Cited by 146 · see citing works

Cationic polyurethanes-short branch PEI-mediated delivery of Mir145 inhibited epithelial–mesenchymal transdifferentiation and cancer stem-like properties and in lung adenocarcinoma

Chiou, G., Cherng, J., Hsu, H., Wang, M., Tsai, C., Lu, K., Chien, Y., Hung, S., Chen, Y., Wong, C.-I., et al. (2012) · Journal of Controlled Release · doi:10.1016/j.jconrel.2012.01.014 · Cited by 144 · see citing works

A new role for microRNAs, as ligands of Toll-like receptors

Fabbri, M., Paone, A., Calore, F., Galli, R., & Croce, C. M. (2013) · RNA Biology · doi:10.4161/rna.23144 · Cited by 143 · see citing works

Recombinant AAV as a Platform for Translating the Therapeutic Potential of RNA Interference

Borel, F., Kay, M. A., & Mueller, C. (2013) · Molecular Therapy · doi:10.1038/mt.2013.285 · Cited by 140 · see citing works

Cancer-targeted MDR-1 siRNA delivery using self-cross-linked glycol chitosan nanoparticles to overcome drug resistance

Yhee, J. Y., Song, S.-Y., Lee, S. J., Park, S.-G., Kim, K., Kim, M. G., Son, S., Koo, H., Kwon, I. C., Jeong, S. Y., et al. (2014) · Journal of Controlled Release · doi:10.1016/j.jconrel.2014.11.019 · Cited by 140 · see citing works

In Vivo Sustained Release of siRNA from Solid Lipid Nanoparticles

Lobovkina, T., Jacobson, G. B., González-González, E., Hickerson, R. P., Leake, D., Kaspar, R. L., Contag, C. H., & Zare, R. N. (2011) · ACS Nano · doi:10.1021/nn203745n · Cited by 139 · see citing works

Molecular Regulation of Endocytosis

Ceresa, B. P. (2012) · InTech eBooks · doi:10.5772/2989 · Cited by 134 · see citing works

Viral vector-mediated RNA interference

Couto, L. B., & High, K. A. (2010) · Current Opinion in Pharmacology · doi:10.1016/j.coph.2010.06.007 · Cited by 129 · see citing works

Binding and Cleavage Specificities of Human Argonaute2

Lima, W. F., Wu, H., Nichols, J. G., Sun, H., Murray, H. M., & Crooke, S. T. (2009) · Journal of Biological Chemistry · doi:10.1074/jbc.m109.010835 · Cited by 126 · see citing works

State-of-the-Art Lentiviral Vectors for Research Use: Risk Assessment and Biosafety Recommendations

Pauwels, K., Gijsbers, R., Toelen, J., Schambach, A., Willard-Gallo, K. E., Verheust, C., Debyser, Z., & Herman, P. (2009) · Current Gene Therapy · doi:10.2174/156652309790031120 · Cited by 126 · see citing works

Gene Expression Analysis in Blood Cells in Response to Unmodified and 2′-Modified siRNAs Reveals TLR-dependent and Independent Effects

Cekaite, L., Furset, G., Hovig, E. J., & Sioud, M. (2006) · Journal of Molecular Biology · doi:10.1016/j.jmb.2006.09.034 · Cited by 121 · see citing works

Endosomal Escape Pathways for Non-Viral Nucleic Acid Delivery Systems

Liang, W., & Lam, J. K. W. (2012) · InTech eBooks · doi:10.5772/46006 · Cited by 121 · see citing works

Utilization of unlocked nucleic acid (UNA) to enhance siRNA performance in vitro and in vivo

Laursen, M. B., Pakula, M. M., Gao, S., Fluiter, K., Mook, O. R. F., Baas, F., Langkjær, N., Wengel, S. L., Wengel, J., Kjems, J., et al. (2010) · Molecular BioSystems · doi:10.1039/b918869j · Cited by 119 · see citing works

Small‐interfering RNA s ( siRNA s) as a promising tool for ocular therapy

Guzmán‐Aránguez, A., Loma, P., & Pintor, J. J. (2013) · British Journal of Pharmacology · doi:10.1111/bph.12330 · Cited by 117 · see citing works

Anti-tumor effects of anti-VEGF siRNA encapsulated with PLGA microspheres in mice

Murata, N., Takashima, Y., Toyoshima, K., et al. (2007) · Journal of Controlled Release · doi:10.1016/j.jconrel.2007.11.017 · Cited by 117 · see citing works

Highly effective inhibition of lung cancer growth and metastasis by systemic delivery of siRNA via multimodal mesoporous silica-based nanocarrier

Chen, Y., Gu, H., Zhang, D. S., Fan, L., Liu, T., & Xia, W. (2014) · Biomaterials · doi:10.1016/j.biomaterials.2014.09.003 · Cited by 117 · see citing works

The induction of tumor apoptosis in B16 melanoma following STAT3 siRNA delivery with a lipid-substituted polyethylenimine

Alshamsan, A., Hamdy, S. I., Samuel, J., El‐Kadi, A. O. S., Lavasanifar, A., & Uludağ, H. (2009) · Biomaterials · doi:10.1016/j.biomaterials.2009.11.003 · Cited by 114 · see citing works

Arginine-Terminated Generation 4 PAMAM Dendrimer as an Effective Nanovector for Functional siRNA Delivery in Vitro and in Vivo

Liu, C., Liu, X., Rocchi, P., Qu, F., Iovanna, J., & Peng, L. (2014) · Bioconjugate Chemistry · doi:10.1021/bc4005156 · Cited by 113 · see citing works

Poly(amidoamine) dendrimer complexes as a platform for gene delivery

Shcharbin, D., Shakhbazau, A., & Bryszewska, M. J. (2013) · Expert Opinion on Drug Delivery · doi:10.1517/17425247.2013.853661 · Cited by 111 · see citing works

microRNA expression in the eyes and their significance in relation to functions

Xu, S. (2008) · Progress in Retinal and Eye Research · doi:10.1016/j.preteyeres.2008.11.003 · Cited by 110 · see citing works

Improved specificity of gene silencing by siRNAs containing unlocked nucleobase analogs

Vaish, N. K., Chen, F., Seth, S., Fosnaugh, K. L., Liu, Y., Adami, R. C., Brown, T., Chen, Y., Harvie, P., Johns, R. E., et al. (2010) · Nucleic Acids Research · doi:10.1093/nar/gkq961 · Cited by 109 · see citing works

PKN3 is required for malignant prostate cell growth downstream of activated PI 3‐kinase

Leenders, F., Möpert, K., Schmiedeknecht, A., Santel, A., Czauderna, F., Aleku, M., Penschuck, S., Dames, S., Sternberger, M., Röhl, T., et al. (2004) · The EMBO Journal · doi:10.1038/sj.emboj.7600345 · Cited by 108 · see citing works

Atu027 Prevents Pulmonary Metastasis in Experimental and Spontaneous Mouse Metastasis Models

Santel, A., Aleku, M., Röder, N., Möpert, K., Durieux, B., Janke, O., Keil, O., Endruschat, J., Dames, S., Lange, C. M., et al. (2010) · Clinical Cancer Research · doi:10.1158/1078-0432.ccr-10-1994 · Cited by 102 · see citing works

Dose-Ranging Evaluation of Intravitreal siRNA PF-04523655 for Diabetic Macular Edema (the DEGAS Study)

Nguyen, Q. D., Schachar, R. A., Nduaka, C. I., Sperling, M., Basile, A. S., Klamerus, K. J., Chi-Burris, K., Yan, E., Paggiarino, D. A., Rosenblatt, I., et al. (2012) · Investigative Ophthalmology & Visual Science · doi:10.1167/iovs.12-9961 · Cited by 101 · see citing works

Intracellular cleavable poly(2-dimethylaminoethyl methacrylate) functionalized mesoporous silica nanoparticles for efficient siRNA delivery in vitro and in vivo

Lin, D., Cheng, Q., Jiang, Q., Huang, Y., Yang, Z., Han, S., Zhao, Y., Guo, S., Liang, Z., & Dong, A. (2013) · Nanoscale · doi:10.1039/c3nr00294b · Cited by 101 · see citing works

Systemic Delivery of microRNA‐34a for Cancer Stem Cell Therapy

Shi, S., Han, L., Gong, T., et al. (2013) · Angewandte Chemie International Edition · doi:10.1002/anie.201208077 · Cited by 100 · see citing works

Vitravene™—Another Piece in the Mosaic

Crooke, S. T. (1998) · Antisense and Nucleic Acid Drug Development · doi:10.1089/oli.1.1998.8.vii · Cited by 96 · 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

Efficient siRNA delivery using water soluble lipopolymer for anti-angiogenic gene therapy

Kim, W. J., Chang, C., Lee, M., & Kim, S. W. (2007) · Journal of Controlled Release · doi:10.1016/j.jconrel.2006.12.026 · Cited by 95 · see citing works

Alternating 2′-O-ribose methylation is a universal approach for generating non-stimulatory siRNA by acting as TLR7 antagonist

Hamm, S., Latz, E., Hangel, D., Müller, T. R., Yu, P., Golenbock, D. T., Sparwasser, T. D., Wagner, H., & Bauer, S. (2009) · Immunobiology · doi:10.1016/j.imbio.2009.09.003 · Cited by 90 · see citing works

Stimuli-responsive liposomes for the delivery of nucleic acid therapeutics

Movahedi, F., Hu, R. G., Becker, D. L., & Xu, C. (2015) · Nanomedicine Nanotechnology Biology and Medicine · doi:10.1016/j.nano.2015.03.006 · Cited by 88 · see citing works

Systemic in vivo lentiviral delivery of miR-15a/16 reduces malignancy in the NZB de novo mouse model of chronic lymphocytic leukemia

Kasar, S. N., Salerno, E., Yuan, Y., Underbayev, C., Vollenweider, D., Laurindo, M. F. L., Fernandes, H., Bonci, D., Addario, A., Mazzella, F. M., et al. (2011) · Genes and Immunity · doi:10.1038/gene.2011.58 · Cited by 87 · see citing works

Evaluation of the siRNA PF-04523655 versus Ranibizumab for the Treatment of Neovascular Age-related Macular Degeneration (MONET Study)

Nguyen, Q. D., Schachar, R. A., Nduaka, C. I., Sperling, M., Klamerus, K. J., Chi-Burris, K., Yan, E., Paggiarino, D. A., Rosenblatt, I., Aitchison, R., et al. (2012) · Ophthalmology · doi:10.1016/j.ophtha.2012.03.043 · Cited by 83 · see citing works

The influence of size, lipid composition and bilayer fluidity of cationic liposomes on the transfection efficiency of nanolipoplexes

Ramezani, M., Khoshhamdam, M., Dehshahri, A., & Malaekeh‐Nikouei, B. (2009) · Colloids and Surfaces B Biointerfaces · doi:10.1016/j.colsurfb.2009.03.018 · Cited by 83 · see citing works

Oncolytic adenovirus co-expressing miRNA-34a and IL-24 induces superior antitumor activity in experimental tumor model

Lou, W., Chen, Q., Ma, L., Liu, J., Yang, Z., Shen, J., Cui, Y., Bian, X., & Qian, C. (2013) · Journal of Molecular Medicine · doi:10.1007/s00109-012-0985-x · Cited by 82 · see citing works

Designing highly active siRNAs for therapeutic applications

Walton, S. P., Wu, M., Gredell, J. A., & Chan, C. (2010) · FEBS Journal · doi:10.1111/j.1742-4658.2010.07903.x · Cited by 76 · see citing works

Highly effective antiangiogenesis via magnetic mesoporous silica-based siRNA vehicle targeting the VEGF gene for orthotopic ovarian cancer therapy

Gu, H., Chen, Y., Wang, X., Liu, T., Zhang, D. S., Wang, Y., & Di, W. J. (2015) · International Journal of Nanomedicine · doi:10.2147/ijn.s78774 · Cited by 74 · see citing works

siRNA Efficiency: Structure or Sequence—That Is the Question

Kurreck, J. (2006) · BioMed Research International · doi:10.1155/jbb/2006/83757 · Cited by 70 · see citing works

Phase 1 dose-escalation study of a siRNA targeting the RTP801 gene in age-related macular degeneration patients

for the PF-04523655 Study Group, Nguyen, Q. D., Schachar, R. A., Nduaka, C. I., Sperling, M., Basile, A. S., Klamerus, K. J., Chi-Burris, K., Yan, E., Paggiarino, D. A., et al. (2012) · Eye · doi:10.1038/eye.2012.106 · Cited by 70 · see citing works

Selection of hyperfunctional siRNAs with improved potency and specificity

Wang, X., Wang, X., Varma, R. K., Beauchamp, L., Magdaleno, S. M., & Sendera, T. J. (2009) · Nucleic Acids Research · doi:10.1093/nar/gkp864 · Cited by 68 · see citing works

Combination of Dendrimer-Nanovector-Mediated Small Interfering RNA Delivery to Target Akt with the Clinical Anticancer Drug Paclitaxel for Effective and Potent Anticancer Activity in Treating Ovarian Cancer

Kala, S., Mak, A. S. C., Liu, X., Posocco, P., Pricl, S., Peng, L., & Wong, A. S. T. (2014) · Journal of Medicinal Chemistry · doi:10.1021/jm401907z · Cited by 67 · see citing works

Sequence determinants of innate immune activation by short interfering RNAs

Goodchild, A., Nopper, N., KING, A. G., Doan, T. B., Tanudji, M., Arndt, G. M., Poidinger, M., Rivory, L. P., & Passioura, T. (2009) · BMC Immunology · doi:10.1186/1471-2172-10-40 · Cited by 66 · see citing works

Titers of lentiviral vectors encoding shRNAs and miRNAs are reduced by different mechanisms that require distinct repair strategies

Liu, Y. P., Vink, M. A., Westerink, J.-T., de Arellano, E. R., Konstantinova, P. S., ter Brake, O., & Berkhout, B. (2010) · RNA · doi:10.1261/rna.1887910 · Cited by 65 · see citing works

Poly(amido amine) is an ideal carrier of miR-7 for enhancing gene silencing effects on the EGFR pathway in U251 glioma cells

Liu, X., Li, G., Su, Z., et al. (2013) · Oncology Reports · doi:10.3892/or.2013.2283 · Cited by 64 · see citing works

Enhanced Intrapulmonary Delivery of Anticancer siRNA for Lung Cancer Therapy Using Cationic Ethylphosphocholine-based Nanolipoplexes

Shim, G., Choi, H., Lee, S. B., Choi, J., Yu, Y. H., Park, D.-E., Choi, Y., Kim, C., & Oh, Y. (2013) · Molecular Therapy · doi:10.1038/mt.2013.10 · Cited by 63 · see citing works

RNAi in Clinical Studies

Kubowicz, P., Żelaszczyk, D., & Pękala, E. (2013) · Current Medicinal Chemistry · doi:10.2174/09298673113209990118 · Cited by 63 · see citing works

Silence of the transcripts: RNA interference in medicine

Barik, S. (2005) · Journal of Molecular Medicine · doi:10.1007/s00109-005-0690-0 · Cited by 61 · see citing works

Adsorption and Release of siRNA from Porous Silica

Steinbacher, J. L., & Landry, C. C. (2013) · Langmuir · doi:10.1021/la402850m · Cited by 60 · see citing works

siRNA Design Principles and Off-Target Effects

Petri, S., & Meister, G. (2013) · Methods in molecular biology · doi:10.1007/978-1-62703-311-4_4 · Cited by 59 · see citing works

Preclinical development of siRNA therapeutics: Towards the match between fundamental science and engineered systems

Videira, M., Arranja, A., Rafael, D. F. D. S., & Gaspar, R. S. (2013) · Nanomedicine Nanotechnology Biology and Medicine · doi:10.1016/j.nano.2013.11.018 · Cited by 58 · see citing works

Synthesis of 2′-O-modified adenosine building blocks and application for RNA interference

Odadzic, D., Bramsen, J. B., Smičius, R., Bus, C., Kjems, J., & Engels, J. W. (2007) · Bioorganic & Medicinal Chemistry · doi:10.1016/j.bmc.2007.09.019 · Cited by 56 · see citing works

Assessment of drug delivery and anticancer potentials of nanoparticles-loaded siRNA targeting STAT3 in lung cancer, in vitro and in vivo

Das, J., Das, S., Paul, A., et al. (2014) · Toxicology Letters · doi:10.1016/j.toxlet.2014.01.009 · Cited by 51 · see citing works

An overview of drugs currently under investigation for the treatment of transthyretin-related hereditary amyloidosis

Obici, L. P., & Merlini, G. (2014) · Expert Opinion on Investigational Drugs · doi:10.1517/13543784.2014.922541 · Cited by 49 · see citing works

Improvement of Doxorubicin Efficacy Using Liposomal Anti-Polo-like Kinase 1 siRNA in Human Renal Cell Carcinomas

Sakurai, Y., Hatakeyama, H., Akita, H., & Harashima, H. (2014) · Molecular Pharmaceutics · doi:10.1021/mp500245z · Cited by 49 · see citing works

Cardiac RNAi therapy using RAGE siRNA/deoxycholic acid-modified polyethylenimine complexes for myocardial infarction

Hong, J.-E., Ku, S. H., Lee, M. S., Jeong, J. H., Mok, H., Choi, D., & Kim, S. H. (2014) · Biomaterials · doi:10.1016/j.biomaterials.2014.05.025 · Cited by 48 · 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

Clustered Regularly Interspaced Short Palindromic Repeats-Based Genome Surgery for the Treatment of Autosomal Dominant Retinitis Pigmentosa

Tsai, Y.-T., Wu, W.-H., Lee, T.-T., Wu, W.-P., Xu, C. L., Park, K. S., Cui, X., Justus, S., Lin, C.-S., Jauregui, R., et al. (2018) · Ophthalmology · doi:10.1016/j.ophtha.2018.04.001 · Cited by 119 · see citing works

Pharmacological intervention in hepatic stellate cell activation and hepatic fibrosis

Schon, H.-T., Bartneck, M., Borkham-Kamphorst, E., Nattermann, J., Lammers, T., Tacke, F., & Weiskirchen, R. (2016) · Frontiers in Pharmacology · doi:10.3389/fphar.2016.00033 · Cited by 97 · see citing works

Exosome-based small RNA delivery: Progress and prospects

Lu, M., Xing, H., Xun, Z., Yang, T., Ding, P., Cai, C., Wang, D., & Zhao, X. (2018) · Asian Journal of Pharmaceutical Sciences · doi:10.1016/j.ajps.2017.07.008 · Cited by 97 · see citing works

Magnetic nanoparticle-induced hyperthermia with appropriate payloads: Paul Ehrlich’s “magic (nano) bullet” for cancer theranostics?

Datta, N. R., Krishnan, S., Speiser, D. E., Neufeld, E., Kuster, N., Bodis, S., & Hofmann, H. (2016) · Cancer Treatment Reviews · doi:10.1016/j.ctrv.2016.09.016 · Cited by 92 · see citing works

RNA delivery biomaterials for the treatment of genetic and rare diseases

Zhao, W., Hou, X., Vick, O. G., & Dong, Y. (2019) · Biomaterials · doi:10.1016/j.biomaterials.2019.119291 · Cited by 82 · see citing works

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 · see citing works

A New Wave of Targeting ‘Undruggable’ Wnt Signaling for Cancer Therapy: Challenges and Opportunities

Park, W.-J., & Kim, M. J. (2023) · Cells · doi:10.3390/cells12081110 · Cited by 57 · see citing works

Genetic engineering approaches to improve posttranslational modification of biopharmaceuticals in different production platforms

Amann, T., Schmieder, V., Faustrup Kildegaard, H., Borth, N., & Andersen, M. R. (2019) · Biotechnology and Bioengineering · doi:10.1002/bit.27101 · Cited by 46 · see citing works

Changing calcium: CRAC channel (STIM and Orai) expression, splicing, and posttranslational modifiers

Niemeyer, B. A. (2016) · American Journal of Physiology-Cell Physiology · doi:10.1152/ajpcell.00034.2016 · Cited by 33 · see citing works

EPAS1 resistance to miRNA-based regulation contributes to prolonged expression of HIF-2 during hypoxia in human endothelial cells

Jaśkiewicz, M., Moszyńska, A., Gebert, M., Collawn, J. F., & Bartoszewski, R. (2023) · Gene · doi:10.1016/j.gene.2023.147376 · Cited by 9 · see citing works

Neurodegenerative disease-associated microRNAs acting as signaling molecules modulate CNS neuron structure and viability

Kumbol, V., Ivanov, A., McGurran, H., Schüler, J., Zhai, Y., Ludwik, K., Hinkelmann, L., Brehm, M., Krüger, C., Küchler, J., et al. (2025) · Cell Communication and Signaling · doi:10.1186/s12964-025-02199-8 · Cited by 8 · see citing works

Double-mismatched siRNAs enhance selective gene silencing of a mutant ALS-causing allele

Changming, Geng, G., HongLiu, et al. (2008) · 中国药理学报：英文版

High potency silencing by single-stranded boranophosphate siRNA

Hall, A., Wan, J., Spesock, A., Sergueeva, Z. A., Shaw, B. R., & Alexander, K. (2006) · Nucleic Acids Research · doi:10.1093/nar/gkl339 · Cited by 110 · see citing works

Viral suppression of RNA silencing in plants

Moissiard, G., & Voinnet, O. (2004) · Molecular Plant Pathology · doi:10.1111/j.1364-3703.2004.00207.x · Cited by 186 · see citing works

Short Interfering RNA (siRNA)—Mediated RNA Interference (RNAi) in Human Cells

Caplen, N. J., & Mousses, S. (2003) · Annals of the New York Academy of Sciences · doi:10.1196/annals.1281.007 · Cited by 83 · see citing works

Role of Silencing Suppressor Proteins

Burgyán, J. (2008) · Methods in molecular biology · doi:10.1007/978-1-59745-102-4_5 · Cited by 40 · see citing works