Development of Novel siRNA Therapeutics: A Review with a Focus on Inclisiran for the Treatment of Hypercholesterolemia

Engineering precision nanoparticles for drug delivery

Mitchell, M. J., Billingsley, M. M., Haley, R. M., Wechsler, M. E., Peppas, N. A., & Langer, R. (2021) · Nature Reviews Drug Discovery · doi:10.1038/s41573-020-0090-8 · Cited by 7,933 · see citing works

Lipid nanoparticles for mRNA delivery

Hou, X., Zaks, T., Langer, R., & Dong, Y. (2021) · Nature Reviews Materials · doi:10.1038/s41578-021-00358-0 · Cited by 4,076 · see citing works

Factors affecting the clearance and biodistribution of polymeric nanoparticles

Alexis, F., Pridgen, E., Molnar, L. K., & Farokhzad, O. C. (2008) · Molecular Pharmaceutics · doi:10.1021/mp800051m · Cited by 3,699 · see citing works

Strategies in the design of nanoparticles for therapeutic applications

Petros, R. A., & DeSimone, J. M. (2010) · Nature Reviews Drug Discovery · doi:10.1038/nrd2591 · Cited by 3,651 · see citing works

Liposome: classification, preparation, and applications

Akbarzadeh, A., Rezaei-Sadabady, R., Davaran, S., Joo, S. W., Zarghami, N., Hanifehpour, Y., Samiei, M., Kouhi, M., & Nejati‐Koshki, K. (2013) · Nanoscale Research Letters · doi:10.1186/1556-276x-8-102 · Cited by 3,648 · see citing works

Poly(ethylene glycol) in Drug Delivery: Pros and Cons as Well as Potential Alternatives

Knop, K., Hoogenboom, R., Fischer, D., & Schubert, U. S. (2010) · Angewandte Chemie International Edition · doi:10.1002/anie.200902672 · Cited by 3,533 · see citing works

How many drug targets are there?

Overington, J. P., Al-Lazikani, B., & Hopkins, A. L. (2006) · Nature Reviews Drug Discovery · doi:10.1038/nrd2199 · Cited by 3,505 · see citing works

The druggable genome

Hopkins, A. L., & Groom, C. R. (2002) · Nature Reviews Drug Discovery · doi:10.1038/nrd892 · Cited by 3,305 · see citing works

Knocking down barriers: advances in siRNA delivery

Whitehead, K. A., Langer, R., & Anderson, D. G. (2009) · Nature Reviews Drug Discovery · doi:10.1038/nrd2742 · Cited by 3,115 · see citing works

Mutations in PCSK9 cause autosomal dominant hypercholesterolemia

Abifadel, M., Varret, M., Rabès, J.-P., Allard, D., Ouguerram, K., Devillers, M., Cruaud, C., Benjannet, S., Wickham, L., Erlich, D., et al. (2003) · Nature Genetics · doi:10.1038/ng1161 · Cited by 2,985 · see citing works

Niacin in Patients with Low HDL Cholesterol Levels Receiving Intensive Statin Therapy

The AIM-HIGH Investigators (2011) · New England Journal of Medicine · doi:10.1056/nejmoa1107579 · Cited by 2,911 · see citing works

Advances and Challenges of Liposome Assisted Drug Delivery

Sercombe, L., Veerati, T., Moheimani, F., Wu, S. Y., Sood, A. K., & Hua, S. (2015) · Frontiers in Pharmacology · doi:10.3389/fphar.2015.00286 · Cited by 2,672 · see citing works

Liposomes as nanomedical devices

Bozzuto, G., & Molinari, A. (2015) · International Journal of Nanomedicine · doi:10.2147/ijn.s68861 · Cited by 2,305 · see citing works

Lipid NanoparticlesFrom Liposomes to mRNA Vaccine Delivery, a Landscape of Research Diversity and Advancement

Tenchov, R., Bird, R., Curtze, A. E., & Zhou, Q. (2021) · ACS Nano · doi:10.1021/acsnano.1c04996 · Cited by 2,221 · see citing works

Carbohydrate-Specific Receptors of the Liver

Ashwell, G., & Harford, J. (1982) · Annual Review of Biochemistry · doi:10.1146/annurev.bi.51.070182.002531 · Cited by 1,914 · see citing works

Rational design of cationic lipids for siRNA delivery

Semple, S. C., Akinc, A., Chen, J., Sandhu, A. P., Mui, B. L., Cho, C. K., Sah, D. W. Y., Stebbing, D., Crosley, E. J., Yaworski, E., et al. (2010) · Nature Biotechnology · doi:10.1038/nbt.1602 · Cited by 1,822 · cited 8× · see citing works

Delivery materials for siRNA therapeutics

Kanasty, R., Dorkin, J. R., Vegas, A., & Anderson, D. (2013) · Nature Materials · doi:10.1038/nmat3765 · Cited by 1,800 · see citing works

Mechanism of Action of Fibrates on Lipid and Lipoprotein Metabolism

Staels, B., Dallongeville, J., Auwerx, J., Schoonjans, K., Leitersdorf, E., & Fruchart, J.-C. (1998) · Circulation · doi:10.1161/01.cir.98.19.2088 · Cited by 1,786 · see citing works

Nanoparticles in the clinic: an update

Anselmo, A. C., & Mitragotri, S. (2019) · Bioengineering & Translational Medicine · doi:10.1002/btm2.10143 · Cited by 1,766 · 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

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

RNA Targeting Therapeutics: Molecular Mechanisms of Antisense Oligonucleotides as a Therapeutic Platform

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

RNAi-mediated gene silencing in non-human primates

Zimmermann, T. S., Lee, A. C. H., Akinc, A., Bramlage, B., Bumcrot, D., Fedoruk, M. N., Harborth, J., Heyes, J. A., Jeffs, L. B., John, M., et al. (2006) · Nature · doi:10.1038/nature04688 · Cited by 1,357 · see citing works

Maximizing the potency of siRNA lipid nanoparticles for hepatic gene silencing in vivo

Jayaraman, M., Ansell, S. M., Mui, B. L., Tam, Y. K., Chen, J., Du, X., Butler, D., Eltepu, L., Matsuda, S., Narayanannair, J. K., et al. (2012) · Angewandte Chemie International Edition · doi:10.1002/anie.201203263 · Cited by 1,267 · see citing works

Lipid Nanoparticle Systems for Enabling Gene Therapies

Cullis, P. R., & Hope, M. J. (2017) · Molecular Therapy · doi:10.1016/j.ymthe.2017.03.013 · Cited by 1,208 · 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

Delivering the Messenger: Advances in Technologies for Therapeutic mRNA Delivery

Kowalski, P. S., Rudra, A., Miao, L., & Anderson, D. G. (2019) · Molecular Therapy · doi:10.1016/j.ymthe.2019.02.012 · Cited by 1,127 · see citing works

The role of sialic acid in determining the survival of glycoproteins in the circulation

Morell, A. G., Gregoriadis, G., Scheinberg, I. H., Hickman, J., & Ashwell, G. (1971) · Journal of Biological Chemistry · doi:10.1016/s0021-9258(19)76994-4 · Cited by 1,072 · see citing works

Solid lipid nanoparticles: A modern formulation approach in drug delivery system

Mukherjee, S., Ray, S., & Thakur, R. S. (2009) · Indian Journal of Pharmaceutical Sciences · doi:10.4103/0250-474x.57282 · Cited by 1,001 · see citing works

Inclisiran in Patients at High Cardiovascular Risk with Elevated LDL Cholesterol

Ray, K. K., Landmesser, U., Leiter, L. A., Kallend, D., Dufour, R., Karakas, M., Hall, T., Troquay, R. P. T., Turner, T., Visseren, F. L. J., et al. (2017) · New England Journal of Medicine · doi:10.1056/nejmoa1615758 · Cited by 992 · see citing works

Solid lipid nanoparticles and nanostructured lipid carriers as novel drug delivery systems: applications, advantages and disadvantages

Mohammadi‐Samani, S., & Ghasemiyeh, P. (2018) · Research in Pharmaceutical Sciences · doi:10.4103/1735-5362.235156 · Cited by 975 · 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

Zika virus protection by a single low-dose nucleoside-modified mRNA vaccination

Pardi, N., Hogan, M. J., Pelc, R. S., Muramatsu, H., Andersen, H., DeMaso, C. R., Dowd, K. A., Sutherland, L. L., Scearce, R. M., Parks, R., et al. (2017) · Nature · doi:10.1038/nature21428 · Cited by 884 · see citing works

Advances in Biomaterials for Drug Delivery

Fenton, O. S., Olafson, K. N., Pillai, P. S., Mitchell, M. J., & Langer, R. (2018) · Advanced Materials · doi:10.1002/adma.201705328 · Cited by 869 · see citing works

Smart Nanoparticles for Drug Delivery Application: Development of Versatile Nanocarrier Platforms in Biotechnology and Nanomedicine

Lombardo, D., Kiselev, M. A., & Caccamo, M. T. (2019) · Journal of Nanomaterials · doi:10.1155/2019/3702518 · Cited by 864 · see citing works

The delivery of therapeutic oligonucleotides

Juliano, R. L. (2016) · Nucleic Acids Research · doi:10.1093/nar/gkw236 · Cited by 863 · see citing works

Receptor-mediated in vitro gene transformation by a soluble DNA carrier system.

Wu, G. Y., & Wu, C. H. (1987) · Journal of Biological Chemistry · doi:10.1016/s0021-9258(18)61209-8 · Cited by 855 · see citing works

RNA-Targeted Therapeutics

Crooke, S. T., Witztum, J. L., Bennett, C. F., & Baker, B. F. (2018) · Cell Metabolism · doi:10.1016/j.cmet.2018.03.004 · Cited by 841 · 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

Delivery of siRNA Therapeutics: Barriers and Carriers

Wang, J., Lu, Z., Wientjes, M. G., & Au, J. L. ‐. (2010) · The AAPS Journal · doi:10.1208/s12248-010-9210-4 · Cited by 804 · see citing works

mRNA-based therapeutics: powerful and versatile tools to combat diseases

Qin, S., Tang, X., Chen, Y., Chen, K., Fan, N., Xiao, W., Zheng, Q., Li, G., Teng, Y., Wu, M., et al. (2022) · Signal Transduction and Targeted Therapy · doi:10.1038/s41392-022-01007-w · Cited by 801 · see citing works

Tools for translation: non-viral materials for therapeutic mRNA delivery

Hajj, K. A., & Whitehead, K. A. (2017) · Nature Reviews Materials · doi:10.1038/natrevmats.2017.56 · Cited by 799 · 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

Application of liposomes in medicine and drug delivery

Daraee, H., Etemadi, A., Kouhi, M., Alimirzalu, S., & Akbarzadeh, A. (2014) · Artificial Cells Nanomedicine and Biotechnology · doi:10.3109/21691401.2014.953633 · Cited by 767 · see citing works

Lipid Nanoparticle Assisted mRNA Delivery for Potent Cancer Immunotherapy

Oberli, M. A., Reichmuth, A. M., Dorkin, J. R., Mitchell, M. J., Fenton, O. S., Jaklenec, A., Anderson, D. G., Langer, R., & Blankschtein, D. (2017) · Nano Letters · doi:10.1021/acs.nanolett.6b03329 · Cited by 719 · 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

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

Therapeutic EphA2 Gene Targeting In vivo Using Neutral Liposomal Small Interfering RNA Delivery

Landen, C. N., Chávez‐Reyes, A., Bucana, C. D., Schmandt, R. E., Deavers, M. T., Lopez‐Berestein, G., & Sood, A. K. (2005) · Cancer Research · doi:10.1158/0008-5472.can-05-0530 · Cited by 674 · 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

siRNA delivery systems for cancer treatment

Oh, Y., & Park, T. G. (2009) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2009.04.018 · Cited by 641 · 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

Therapeutic RNAi targeting PCSK9 acutely lowers plasma cholesterol in rodents and LDL cholesterol in nonhuman primates

Frank-Kamenetsky, M. D., Grefhorst, A., Anderson, N. N., Racie, T., Bramlage, B., Akinc, A., Butler, D., Charissé, K., Dorkin, J. R., Fan, Y., et al. (2008) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.0805434105 · Cited by 635 · see citing works

Stability of Endogenous and Added RNA in Blood Specimens, Serum, and Plasma

Tsui, N. B. Y., Ng, E. K. O., & Lo, Y. D. (2002) · Clinical Chemistry · doi:10.1093/clinchem/48.10.1647 · Cited by 628 · 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

Asialoglycoprotein receptor mediated hepatocyte targeting — Strategies and applications

D’souza, A. A., & Devarajan, P. V. (2015) · Journal of Controlled Release · doi:10.1016/j.jconrel.2015.02.022 · Cited by 624 · see citing works

Targeted delivery of antisense oligonucleotides to hepatocytes using triantennaryN-acetyl galactosamine improves potency 10-fold in mice

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

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

Effect of a monoclonal antibody to PCSK9, REGN727/SAR236553, to reduce low-density lipoprotein cholesterol in patients with heterozygous familial hypercholesterolaemia on stable statin dose with or without ezetimibe therapy: a phase 2 randomised controlled trial

Stein, E. A., Gipe, D., Bergeron, J., Gaudet, D., Weiss, R., Dufour, R., Wu, R., & Pordy, R. C. (2012) · The Lancet · doi:10.1016/s0140-6736(12)60771-5 · Cited by 575 · 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

Cationic lipid saturation influences intracellular delivery of encapsulated nucleic acids

Heyes, J., Palmer, L., Bremner, K., & MacLachlan, I. (2005) · Journal of Controlled Release · doi:10.1016/j.jconrel.2005.06.014 · Cited by 572 · 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

The Limitless Future of RNA Therapeutics

Damase, T. R., Sukhovershin, R. A., Boada, C., Taraballi, F., Pettigrew, R. I., & Cooke, J. P. (2021) · Frontiers in Bioengineering and Biotechnology · doi:10.3389/fbioe.2021.628137 · Cited by 549 · see citing works

Lipid Nanoparticle Technology for Clinical Translation of siRNA Therapeutics

Kulkarni, J. A., Witzigmann, D., Chen, S., Cullis, P. R., & van der Meel, R. (2019) · Accounts of Chemical Research · doi:10.1021/acs.accounts.9b00368 · Cited by 534 · see citing works

Anti-PCSK9 Antibody Effectively Lowers Cholesterol in Patients With Statin Intolerance

Stroes, E., Colquhoun, D., Sullivan, D., Civeira, F., Rosenson, R. S., Watts, G. F., Bruckert, E., Cho, L., Dent, R., Knusel, B., et al. (2014) · Journal of the American College of Cardiology · doi:10.1016/j.jacc.2014.03.019 · Cited by 526 · see citing works

The asialoglycoprotein receptor: relationships between structure, function, and expression

Stockert, R. J. (1995) · Physiological Reviews · doi:10.1152/physrev.1995.75.3.591 · Cited by 524 · see citing works

Efficacy and Tolerability of Evolocumab vs Ezetimibe in Patients With Muscle-Related Statin Intolerance: The GAUSS-3 Randomized Clinical Trial

Nissen, S. E., Stroes, E., Dent-Acosta, R. E., Rosenson, R. S., Lehman, S. J., Sattar, N., Preiss, D., Bruckert, E., Ceška, R., Lepor, N., et al. (2016) · JAMA · doi:10.1001/jama.2016.3608 · Cited by 522 · see citing works

Self-assembled mRNA vaccines

Kim, J., Eygeris, Y., Gupta, M., & Sahay, G. (2021) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2020.12.014 · Cited by 514 · see citing works

Influence of Polyethylene Glycol Lipid Desorption Rates on Pharmacokinetics and Pharmacodynamics of siRNA Lipid Nanoparticles

Mui, B. L., Tam, Y. K., Jayaraman, M., Ansell, S. M., Du, X., Tam, Y. Y. C., Lin, P. J. C., Chen, S., Narayanannair, J. K., Rajeev, K. G., et al. (2013) · Molecular Therapy — Nucleic Acids · doi:10.1038/mtna.2013.66 · Cited by 509 · see citing works

Safety and Efficacy of a Monoclonal Antibody to Proprotein Convertase Subtilisin/Kexin Type 9 Serine Protease, SAR236553/REGN727, in Patients With Primary Hypercholesterolemia Receiving Ongoing Stable Atorvastatin Therapy

McKenney, J. M., Koren, M. J., Kereiakes, D. J., Hanotin, C., Ferrand, A.-C., & Stein, E. A. (2012) · Journal of the American College of Cardiology · doi:10.1016/j.jacc.2012.03.007 · Cited by 507 · 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

The asialoglycoprotein receptor: a model for endocytic transport receptors

Spiess, M. (1990) · Biochemistry · doi:10.1021/bi00495a001 · Cited by 488 · see citing works

Atorvastatin with or without an Antibody to PCSK9 in Primary Hypercholesterolemia

Roth, E. M., McKenney, J. M., Hanotin, C., Asset, G., & Stein, E. A. (2012) · New England Journal of Medicine · doi:10.1056/nejmoa1201832 · Cited by 459 · see citing works

Efficient encapsulation of antisense oligonucleotides in lipid vesicles using ionizable aminolipids: formation of novel small multilamellar vesicle structures

Semple, S. C., Klimuk, S. K., Harasym, T. O., Dos Santos, N., Ansell, S. M., Wong, K. F., Maurer, N., Stark, H., Cullis, P. R., Hope, M. J., et al. (2001) · Biochimica et Biophysica Acta (BBA) - Biomembranes · doi:10.1016/s0005-2736(00)00343-6 · Cited by 454 · see citing works

Current progress of siRNA/shRNA therapeutics in clinical trials

Burnett, J., Rossi, J. J., & Tiemann, K. (2011) · Biotechnology Journal · doi:10.1002/biot.201100054 · Cited by 445 · see citing works

Hepatic Drug Targeting: Phase I Evaluation of Polymer-Bound Doxorubicin

Seymour, L. W., Ferry, D. R., Anderson, D., Hesslewood, S., Julyan, P. J., Poyner, R., Doran, J., Young, A., Burtles, S. S., & Kerr, D. J. (2002) · Journal of Clinical Oncology · doi:10.1200/jco.20.6.1668 · Cited by 435 · see citing works

Liposomes in tissue engineering and regenerative medicine

Monteiro, N., Martins, A. M., Reis, R. L., & Neves, N. M. (2014) · Journal of The Royal Society Interface · doi:10.1098/rsif.2014.0459 · Cited by 429 · see citing works

The druggable genome: an update

Russ, A., & Lampel, S. (2005) · Drug Discovery Today · doi:10.1016/s1359-6446(05)03666-4 · Cited by 416 · see citing works

Delivery of Oligonucleotides to the Liver with GalNAc: From Research to Registered Therapeutic Drug

Debacker, A. J., Voutila, J., Catley, M. C., Blakey, D. C., & Habib, N. A. (2020) · Molecular Therapy · doi:10.1016/j.ymthe.2020.06.015 · Cited by 403 · see citing works

Efficient Targeting and Activation of Antigen-Presenting Cells In Vivo after Modified mRNA Vaccine Administration in Rhesus Macaques

Liang, F., Lindgren, G., Lin, A., Thompson, E. A., Ols, S., Röhss, J., John, S., Hassett, K., Yuzhakov, O., Bahl, K., et al. (2017) · Molecular Therapy · doi:10.1016/j.ymthe.2017.08.006 · Cited by 395 · see citing works

Cytosolic delivery of nucleic acids: The case of ionizable lipid nanoparticles

Schlich, M., Palomba, R., Costabile, G., Mizrahy, S., Pannuzzo, M., Peer, D., & Decuzzi, P. (2021) · Bioengineering &amp; Translational Medicine · doi:10.1002/btm2.10213 · Cited by 387 · see citing works

Influence of particle size on the in vivo potency of lipid nanoparticle formulations of siRNA

Chen, S., Tam, Y. Y. C., Lin, P. J. C., Sung, M. M. H., Tam, Y. K., & Cullis, P. R. (2016) · Journal of Controlled Release · doi:10.1016/j.jconrel.2016.05.059 · Cited by 366 · see citing works

Development of Lipidoid–siRNA Formulations for Systemic Delivery to the Liver

Akinc, A., Goldberg, M. S., Qin, J., Dorkin, J. R., Gamba‐Vitalo, C., Maier, M. A., Jayaprakash, K. N., Jayaraman, M., Rajeev, K. G., Manoharan, M., et al. (2009) · Molecular Therapy · doi:10.1038/mt.2009.36 · Cited by 353 · 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

The Ashwell-Morell receptor regulates hepatic thrombopoietin production via JAK2-STAT3 signaling

Grozovsky, R., Begonja, A. J., Liu, K., Visner, G. A., Hartwig, J. H., Falet, H., & Hoffmeister, K. M. (2014) · Nature Medicine · doi:10.1038/nm.3770 · Cited by 338 · see citing works

RNA interference-based therapy and its delivery systems

Chen, X., Mangala, L. S., Rodriguez‐Aguayo, C., Kong, X., Lopez‐Berestein, G., & Sood, A. K. (2017) · Cancer and Metastasis Reviews · doi:10.1007/s10555-017-9717-6 · Cited by 333 · see citing works

Gene transfer into hepatocytes using asialoglycoprotein receptor mediated endocytosis of DNA complexed with an artificial tetra-antennary galactose ligand

Plank, C., Zatloukal, K., Cotten, M. L., Mechtler, K., & Wagner, E. (1992) · Bioconjugate Chemistry · doi:10.1021/bc00018a012 · Cited by 324 · see citing works

Nanotechnology-based drug delivery systems for the treatment of Alzheimer’s disease

Fonseca‐Santos, B., Chorilli, M., & Gremião, M. P. D. (2015) · International Journal of Nanomedicine · doi:10.2147/ijn.s87148 · Cited by 313 · see citing works

Clinical Utility of Bile Acid Sequestrants in the Treatment of Dyslipidemia: A Scientific Review

Insull, W. (2006) · Southern Medical Journal · doi:10.1097/01.smj.0000208120.73327.db · Cited by 308 · see citing works

siRNA therapeutics: big potential from small RNAs

Ryther, R. C. C., Flynt, A. S., Phillips, J. A., & Patton, J. G. (2004) · Gene Therapy · doi:10.1038/sj.gt.3302356 · Cited by 301 · see citing works

The Multirole of Liposomes in Therapy and Prevention of Infectious Diseases

Nisini, R., Poerio, N., Mariotti, S., Santis, F. D., & Fraziano, M. (2018) · Frontiers in Immunology · doi:10.3389/fimmu.2018.00155 · Cited by 299 · see citing works

PCSK9 inhibitors: A new era of lipid lowering therapy

Chaudhary, R., Garg, J., Shah, N., & Sumner, A. (2017) · World Journal of Cardiology · doi:10.4330/wjc.v9.i2.76 · Cited by 299 · see citing works

Hepatic Drug Targeting: Phase I Evaluation of Polymer-Bound Doxorubicin

Seymour, L. W., Ferry, D. R., Anderson, D., et al. (2002) · Journal of Clinical Oncology · doi:10.1200/jco.2002.20.6.1668 · Cited by 287 · see citing works

RNAi‐based therapeutics–current status, challenges and prospects

Tiemann, K., & Rossi, J. J. (2009) · EMBO Molecular Medicine · doi:10.1002/emmm.200900023 · Cited by 285 · see citing works

Action and Reaction: The Biological Response to siRNA and Its Delivery Vehicles

Kanasty, R. L., Whitehead, K. A., Vegas, A. J., & Anderson, D. G. (2012) · Molecular Therapy · doi:10.1038/mt.2011.294 · Cited by 281 · see citing works

CryptoSite: Expanding the Druggable Proteome by Characterization and Prediction of Cryptic Binding Sites

Cimermančič, P., Weinkam, P., Rettenmaier, T. J., Bichmann, L., Keedy, D. A., Woldeyes, R. A., Schneidman‐Duhovny, D., Demerdash, O., Mitchell, J. C., Wells, J. A., et al. (2016) · Journal of Molecular Biology · doi:10.1016/j.jmb.2016.01.029 · Cited by 277 · 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

Boosting intracellular delivery of lipid nanoparticle-encapsulated mRNA

Patel, S., Ashwanikumar, N., Robinson, E., DuRoss, A., Sun, C., Murphy-Benenato, K. E., Mihai, C., Almarsson, Ö., & Sahay, G. (2017) · Nano Letters · doi:10.1021/acs.nanolett.7b02664 · Cited by 251 · see citing works

Galactose and N-acetylgalactosamine-specific endocytosis of glycopeptides by isolated rat hepatocytes

Baenziger, J., & Fiete, D. (1980) · Cell · doi:10.1016/0092-8674(80)90371-2 · Cited by 248 · see citing works

Liposomes as Potential Drug Carrier Systems for Drug Delivery

Çağdaş, M., Sezer, A. D., & Bucak, Ş. (2014) · InTech eBooks · doi:10.5772/58459 · Cited by 248 · see citing works

Monotherapy with the PCSK9 inhibitor alirocumab versus ezetimibe in patients with hypercholesterolemia: Results of a 24week, double-blind, randomized Phase 3 trial

Roth, E. M., Taskinen, M., Ginsberg, H. N., Kastelein, J. J. P., Colhoun, H. M., Robinson, J. G., Merlet, L., Pordy, R. C., & Baccara‐Dinet, M. T. (2014) · International Journal of Cardiology · doi:10.1016/j.ijcard.2014.06.049 · Cited by 247 · see citing works

Small interfering RNAs (siRNAs) in cancer therapy: a nano-based approach

Chalbatani, G. M., Dana, H., Gharagouzloo, E., Grijalvo, S., Eritja, R., Logsdon, C. D., Memari, F., Miri, S. R., Rad, M. R., & Marmari, V. (2019) · International Journal of Nanomedicine · doi:10.2147/ijn.s200253 · Cited by 238 · see citing works

Spontaneous Entrapment of Polynucleotides upon Electrostatic Interaction with Ethanol-Destabilized Cationic Liposomes

Maurer, N., Wong, K. F., Stark, H., Louie, L. J., McIntosh, D. P., Wong, T., Scherrer, P., Semple, S. C., & Cullis, P. R. (2001) · Biophysical Journal · doi:10.1016/s0006-3495(01)76202-9 · Cited by 232 · see citing works

Development of Triantennary N‑Acetylgalactosamine Conjugates as Degraders for Extracellular Proteins

Zhou, Y., Teng, P., Montgomery, N. T., Li, X., & Tang, W. (2021) · ACS Central Science · doi:10.1021/acscentsci.1c00146 · Cited by 228 · see citing works

Ionizable lipid nanoparticles for in utero mRNA delivery

Riley, R. S., Kashyap, M. V., Billingsley, M. M., White, B., Alameh, M.-G., Bose, S. K., Zoltick, P. W., Li, H., Zhang, R., Cheng, A. Y., et al. (2021) · Science Advances · doi:10.1126/sciadv.aba1028 · Cited by 222 · see citing works

Biochemistry of carbohydrate‐protein interaction 1

Lee, Y. C. (1992) · The FASEB Journal · doi:10.1096/fasebj.6.13.1397841 · Cited by 209 · see citing works

Stabilized plasmid–lipid particles containing PEG-diacylglycerols exhibit extended circulation lifetimes and tumor selective gene expression

Ambegia, E., Ansell, S. M., Cullis, P. R., Heyes, J. A., Palmer, L., & MacLachlan, I. (2005) · Biochimica et Biophysica Acta (BBA) - Biomembranes · doi:10.1016/j.bbamem.2005.02.001 · Cited by 203 · see citing works

Crystal Structure of the Carbohydrate Recognition Domain of the H1 Subunit of the Asialoglycoprotein Receptor

Meier, M., Bider, M. D., Malashkevich, V. N., Spiess, M., & Burkhard, P. (2000) · Journal of Molecular Biology · doi:10.1006/jmbi.2000.3853 · Cited by 192 · see citing works

Novel mutations of thePCSK9 gene cause variable phenotype of autosomal dominant hypercholesterolemia

Allard, D., Amsellem, S., Abifadel, M., Trillard, M., Devillers, M., Luc, G., Krempf, M., Reznik, Y., Girardet, J.-P., Fredenrich, A., et al. (2005) · Human Mutation · doi:10.1002/humu.9383 · Cited by 190 · see citing works

Targeting hepatocytes for drug and gene delivery emerging novel approaches and applications

Wu, J. (2002) · Frontiers in bioscience · doi:10.2741/a806 · Cited by 190 · 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

Stabilin-1 and Stabilin-2 are specific receptors for the cellular internalization of phosphorothioate-modified antisense oligonucleotides (ASOs) in the liver

Miller, C. M., Donner, A. J., Blank, E. K., Egger, A. W., Kellar, B. M., Østergaard, M. E., Seth, P. P., & Harris, E. N. (2016) · Nucleic Acids Research · doi:10.1093/nar/gkw112 · Cited by 171 · see citing works

Statins and PCSK9 inhibitors: A new lipid-lowering therapy

Gallego-Colón, E., Daum, A., & Yosefy, C. (2020) · European Journal of Pharmacology · doi:10.1016/j.ejphar.2020.173114 · Cited by 166 · see citing works

A review on cationic lipids with different linkers for gene delivery

Zhi, D., Bai, Y., Yang, J., Cui, S., Zhao, Y., Chen, H., & Zhang, S. (2017) · Advances in Colloid and Interface Science · doi:10.1016/j.cis.2017.12.006 · Cited by 163 · see citing works

Lipid nanoparticles for short interfering RNA delivery

Leung, A. K. K., Tam, Y. Y. C., & Cullis, P. R. (2014) · Advances in genetics · doi:10.1016/b978-0-12-800148-6.00004-3 · Cited by 158 · see citing works

Clinical advances of siRNA therapeutics

Hu, B., Weng, Y., Xia, X., Liang, X., & Huang, Y. (2019) · The Journal of Gene Medicine · doi:10.1002/jgm.3097 · Cited by 157 · see citing works

Liver fat accumulation is associated with circulating PCSK9

Ruscica, M., Ferri, N., Macchi, C., Meroni, M., Lanti, C., Ricci, C., Maggioni, M., Fracanzani, A. L., Badiali, S., Fargion, S. R., et al. (2016) · Annals of Medicine · doi:10.1080/07853890.2016.1188328 · Cited by 154 · see citing works

Editorial focus: understanding off-target effects as the key to successful RNAi therapy

Bartoszewski, R., & Sikorski, A. F. (2019) · Cellular & Molecular Biology Letters · doi:10.1186/s11658-019-0196-3 · Cited by 150 · see citing works

Modulation of Membrane Fusion by Asymmetric Transbilayer Distributions of Amino Lipids

Bailey, A. L., & Cullis, P. R. (1994) · Biochemistry · doi:10.1021/bi00208a007 · Cited by 143 · see citing works

The Ashwell–Morell Receptor

Grewal, P. K. (2010) · Methods in enzymology on CD-ROM/Methods in enzymology · doi:10.1016/s0076-6879(10)79013-3 · Cited by 136 · see citing works

Encapsulation and release of a hydrophobic drug from hydroxyapatite coated liposomes

Xu, Q., Tanaka, Y., & Czernuszka, J. T. (2007) · Biomaterials · doi:10.1016/j.biomaterials.2007.02.007 · Cited by 136 · see citing works

Factors influencing the retention and chemical stability of poly(ethylene glycol)-lipid conjugates incorporated into large unilamellar vesicles

Parr, M. J., Ansell, S. M., Choi, L. S. L., & Cullis, P. R. (1994) · Biochimica et Biophysica Acta (BBA) - Biomembranes · doi:10.1016/0005-2736(94)90004-3 · Cited by 134 · see citing works

Surface De-PEGylation Controls Nanoparticle-Mediated siRNA Delivery In Vitro and In Vivo

Zhu, X., Tao, W., Liu, D. X., Wu, J., Guo, Z., Ji, X., Bharwani, Z., Zhao, L., Zhao, X., Farokhzad, O. C., et al. (2017) · Theranostics · doi:10.7150/thno.18136 · Cited by 134 · see citing works

Comprehensive Structure–Activity Relationship of TriantennaryN-Acetylgalactosamine Conjugated Antisense Oligonucleotides for Targeted Delivery to Hepatocytes

Prakash, T. P., Yu, J., Migawa, M. T., Kinberger, G. A., Wan, W. B., Østergaard, M. E., Carty, R. L., Vasquez, G., Low, A., Chappell, A. E., et al. (2016) · Journal of Medicinal Chemistry · doi:10.1021/acs.jmedchem.5b01948 · Cited by 132 · see citing works

Cubic Phases in Phosphatidylcholine-Cholesterol Mixtures: Cholesterol as Membrane “Fusogen”

Tenchov, B., MacDonald, R. C., & Siegel, D. P. (2006) · Biophysical Journal · doi:10.1529/biophysj.106.083766 · Cited by 130 · see citing works

Nanoplatforms for mRNA Therapeutics

Meng, C., Chen, Z., Li, G., Welte, T., & Shen, H. (2021) · Advanced Therapeutics · doi:10.1002/adtp.202000099 · Cited by 130 · see citing works

Synthesis and characterization of novel poly(ethylene glycol)-lipid conjugates suitable for use in drug delivery

Heyes, J. A., Hall, K., Tailor, V., Lenz, R., & MacLachlan, I. (2006) · Journal of Controlled Release · doi:10.1016/j.jconrel.2006.02.012 · Cited by 123 · see citing works

Inclisiran—New hope in the management of lipid disorders?

Dyrbuś, K., Gąsior, M., Penson, P., Ray, K. K., & Banach, M. (2020) · Journal of Clinical Lipidology · doi:10.1016/j.jacl.2019.11.001 · Cited by 120 · see citing works

Evaluation of GalNAc-siRNA Conjugate Activity in Pre-clinical Animal Models with Reduced Asialoglycoprotein Receptor Expression

Willoughby, J. L. S., Chan, A., Sehgal, A., Butler, J. S., Nair, J. K., Racie, T., Shulga‐Morskaya, S., Nguyen, T. M., Qian, K., Yucius, K., et al. (2017) · Molecular Therapy · doi:10.1016/j.ymthe.2017.08.019 · Cited by 117 · see citing works

Effect of inclisiran on lipids in primary prevention: the ORION-11 trial

Ray, K. K., Kallend, D., Leiter, L. A., Raal, F. J., Koenig, W., Jaros, M. J., Schwartz, G. G., Landmesser, U., Garcia Conde, L., Wright, R. S., et al. (2022) · European Heart Journal · doi:10.1093/eurheartj/ehac615 · Cited by 116 · see citing works

Asialoglycoprotein receptor (ASGPR) as target autoantigen in liver autoimmunity: Lost and found

Rigopoulou, E. I., Roggenbuck, D., Smyk, D. S., Liaskos, C., Mytilinaiou, M. G., Feist, E., Conrad, K., & Bogdanos, D. P. (2012) · Autoimmunity Reviews · doi:10.1016/j.autrev.2012.04.005 · Cited by 108 · see citing works

Niacin Treatment of Hypercholesterolemia in Children

Colletti, R. B., Roff, N. K., Neufeld, E. J., Baker, A., Newburger, J. W., & McAuliffe, T. L. (1993) · PEDIATRICS · doi:10.1542/peds.92.1.78 · Cited by 106 · see citing works

Systemic delivery of RafsiRNA using cationic cardiolipin liposomes silences Raf-1 expression and inhibits tumor growth in xenograft model of human prostate cancer

Pal, A., Ahmad, A., Khan, S., Sakabe, I., Zhang, C., Kasid, U. N., & Ahmad, I. (2005) · International Journal of Oncology · doi:10.3892/ijo.26.4.1087 · Cited by 104 · see citing works

Two asialoglycoprotein receptor polypeptides in human hepatoma cells.

Bischoff, J. E., & Lodish, H. F. (1987) · Journal of Biological Chemistry · doi:10.1016/s0021-9258(18)60888-9 · Cited by 101 · see citing works

A Comprehensive Review on the Applications of Exosomes and Liposomes in Regenerative Medicine and Tissue Engineering

Shafiei, M., Ansari, M. N. M., Razak, S. I. A., & Khan, M. U. A. (2021) · Polymers · doi:10.3390/polym13152529 · Cited by 100 · see citing works

Design and Synthesis of Novel N-Acetylgalactosamine-Terminated Glycolipids for Targeting of Lipoproteins to the Hepatic Asialoglycoprotein Receptor

Rensen, P. C. N., van Leeuwen, S. H., Sliedregt, L. A. J. M., van Berkel, T. J. C., & Biessen, E. A. L. (2004) · Journal of Medicinal Chemistry · doi:10.1021/jm049481d · Cited by 87 · see citing works

Asialoglycoprotein receptor (ASGPR): a peculiar target of liver-specific autoimmunity

Roggenbuck, D., Mytilinaiou, M. G., Lapin, S. V., Reinhold, D., & Conrad, K. (2012) · Autoimmunity Highlights · doi:10.1007/s13317-012-0041-4 · Cited by 85 · see citing works

PCSK9 Inhibitors in Lipid Management of Patients With Diabetes Mellitus and High Cardiovascular Risk: A Review

Handelsman, Y., & Lepor, N. E. (2018) · Journal of the American Heart Association · doi:10.1161/jaha.118.008953 · Cited by 85 · see citing works

Variable asialoglycoprotein receptor 1 expression in liver disease: Implications for therapeutic intervention

Witzigmann, D., Quagliata, L., Schenk, S., Quintavalle, C., Terracciano, L., & Huwyler, J. (2015) · Hepatology Research · doi:10.1111/hepr.12599 · Cited by 80 · see citing works

siRNA Therapeutics against Respiratory Viral Infections—What Have We Learned for Potential COVID‐19 Therapies?

Mehta, A., Michler, T., & Merkel, O. M. (2021) · Advanced Healthcare Materials · doi:10.1002/adhm.202001650 · Cited by 74 · see citing works

Enhanced inhibition of virus DNA synthesis in hepatocytes by trifluorothymidine coupled to asialofetuin

FIUME, L., Mattioli, A., Balboni, P. G., Tognon, M. G., Barbanti-Brodano, G., Vries, J. D., & Wieland, T. (1979) · FEBS Letters · doi:10.1016/0014-5793(79)81247-8 · Cited by 66 · see citing works

Cell-Type Specific and Ligand Specific Enhancement of Cellular Uptake of Oligodeoxynucleoside Methylphosphonates Covalently Linked with a Neoglycopeptide, YEE(ah-GalNAc)3

Hangeland, J. J., Levis, J. T., Lee, Y. C., & Ts’o, P. O. P. (1995) · Bioconjugate Chemistry · doi:10.1021/bc00036a006 · Cited by 63 · see citing works

Selective inhibition of ectromelia virus DNA synthesis in hepatocytes by adenine‐9‐β‐D‐arabinofuranoside (ara‐A) and adenine‐9‐β‐D‐arabinofuranoside 5′‐monophosphate (ara‐AMP) conjugated to asialofetuin

FIUME, L., Mattioli, A., Busi, C., Balboni, P. G., Barbanti-Brodano, G., Vries, J. D., Altmann, R., & Wieland, T. (1980) · FEBS Letters · doi:10.1016/0014-5793(80)80639-9 · Cited by 61 · see citing works

Inclisiran for the Treatment of Cardiovascular Disease: A Short Review on the Emerging Data and Therapeutic Potential

Kosmas, C. E., Estrella, A. M., Skavdis, A., Genao, E. P., Martinez, I., & Guzman, E. (2020) · Therapeutics and Clinical Risk Management · doi:10.2147/tcrm.s230592 · Cited by 54 · see citing works

Inclisiran: a small interfering RNA strategy targeting PCSK9 to treat hypercholesterolemia

Banerjee, Y., Pantea Stoian, A., Cicero, A. F. G., Fogacci, F., Nikolic, D., Sachinidis, A., Rizvi, A. A., Janez, A., & Rizzo, M. (2022) · Expert Opinion on Drug Safety · doi:10.1080/14740338.2022.1988568 · Cited by 54 · see citing works

Clinical development of monoclonal antibody-based drugs in HIV and HCV diseases

Flego, M., Ascione, A., Cianfriglia, M., & Vella, S. (2013) · BMC Medicine · doi:10.1186/1741-7015-11-4 · Cited by 51 · see citing works

Alirocumab for Hyperlipidemia: Physiology of PCSK9 inhibition, Pharmacodynamics and Phase I and Ii Clinical Trial Results of a PCSK9 Monoclonal Antibody

Roth, E. M., & Diller, P. M. (2014) · Future Cardiology · doi:10.2217/fca.13.107 · Cited by 49 · see citing works

Inclisiran for the treatment of hypercholesterolaemia: implications and unanswered questions from the ORION trials

Cupido, A. J., & Kastelein, J. J. P. (2020) · Cardiovascular Research · doi:10.1093/cvr/cvaa212 · Cited by 48 · see citing works

Interrogating the druggable genome with structural informatics

Hambly, K., Danzer, J. F., Muskal, S. M., & Debe, D. A. (2006) · Molecular Diversity · doi:10.1007/s11030-006-9035-3 · Cited by 48 · see citing works

Inclisiran in lipid management: A Literature overview and future perspectives

Scicchitano, P., Milo, M., Mallamaci, R., Palo, M. D., Caldarola, P., Massari, F., Gabrielli, D., Colivicchi, F., & Ciccone, M. M. (2021) · Biomedicine & Pharmacotherapy · doi:10.1016/j.biopha.2021.112227 · Cited by 47 · see citing works

Untitled work

Heel (1980) · doi:10.2165/00003495-198019030-00001 · Cited by 46 · see citing works

A Critical Review of the Efficacy and Safety of Inclisiran

Hardy, J., Niman, S., Pereira, E., Lewis, T., Reid, J., Choksi, R., & Goldfaden, R. F. (2021) · American Journal of Cardiovascular Drugs · doi:10.1007/s40256-021-00477-7 · Cited by 44 · see citing works

Conjugation of mono and di-GalNAc sugars enhances the potency of antisense oligonucleotides via ASGR mediated delivery to hepatocytes

Kinberger, G. A., Prakash, T. P., Yu, J., Vasquez, G., Low, A., Chappell, A. E., Schmidt, K., Murray, H. M., Gaus, H. J., Swayze, E. E., et al. (2016) · Bioorganic & Medicinal Chemistry Letters · doi:10.1016/j.bmcl.2016.05.084 · Cited by 42 · see citing works

siRNA technology

Schütze, N. (2003) · Molecular and Cellular Endocrinology · doi:10.1016/j.mce.2003.10.078 · Cited by 42 · see citing works

Exaggerated levothyroxine malabsorption due to calcium carbonate supplementation in gastrointestinal disorders

Csako, G., McGriff, N. J., Rotman-Pikielny, P., Sarlis, N. J., & Pucino, F. (2001) · Annals of Pharmacotherapy · doi:10.1345/aph.1a031 · Cited by 40 · see citing works

Pharmacokinetics and pharmacodynamics of vincristine sulfate liposome injection (VSLI) in adults with acute lymphoblastic leukemia

Silverman, J. A., Reynolds, L., & Deitcher, S. R. (2013) · The Journal of Clinical Pharmacology · doi:10.1002/jcph.155 · Cited by 38 · see citing works

Solubilization of hepatic binding sites for asialo-glycoproteins

Morell, A. G., & Scheinberg, I. H. (1972) · Biochemical and Biophysical Research Communications · doi:10.1016/0006-291x(72)90679-1 · Cited by 38 · see citing works

Fenofibrate or Gemfibrozil for Treatment of Types IIa and IIb Primary Hyperlipoproteinemia: A Randomized, Double-Blind, Crossover Study

Insua, A., Massari, F., Moncalvo, J. J. R., Zanchetta, J., & Insua, A. M. (2002) · Endocrine Practice · doi:10.4158/ep.8.2.96 · Cited by 36 · see citing works

PCSK9 and lipid lowering drugs

Guo, Y., Zhang, W., & Li, J. (2014) · Clinica Chimica Acta · doi:10.1016/j.cca.2014.07.008 · Cited by 36 · see citing works

Design of a Targeted Peptide Nucleic Acid Prodrug To Inhibit Hepatic Human Microsomal Triglyceride Transfer Protein Expression in Hepatocytes

Biessen, E. A. L., Sliedregt, K., Hoen, P. A. C. ’., Prince, P., van de Bilt, E., Valentijn, A. R. P. M., Meeuwenoord, N. J., Princen, H. M. G., Bijsterbosch, M. K., van der MAREL, G. A., et al. (2002) · Bioconjugate Chemistry · doi:10.1021/bc015550g · Cited by 35 · see citing works

Long-Term Safety and Efficacy of Triple Combination Ezetimibe/Simvastatin Plus Extended-Release Niacin in Patients With Hyperlipidemia

Fazio, S., Guyton, J. R., Polis, A. B., Adewale, A. J., Tomassini, J. E., Ryan, N. W., & Tershakovec, A. M. (2009) · The American Journal of Cardiology · doi:10.1016/j.amjcard.2009.10.001 · Cited by 34 · see citing works

Efficacy and safety of inclisiran a newly approved FDA drug: a systematic review and pooled analysis of available clinical studies

Cicero, A. F. G., Fogacci, F., Zambon, A., Toth, P. P., & Borghi, C. (2022) · American Heart Journal Plus: Cardiology Research and Practice · doi:10.1016/j.ahjo.2022.100127 · Cited by 34 · see citing works

Safety of Niacin and Simvastatin Combination Therapy

Bays, H. E. (2008) · The American Journal of Cardiology · doi:10.1016/j.amjcard.2008.02.026 · Cited by 32 · see citing works

RT-qPCR Methods to Support Pharmacokinetics and Drug Mechanism of Action to Advance Development of RNAi Therapeutics

Castellanos-Rizaldos, E., Brown, C. R., Dennin, S., Kim, J., Gupta, S., Najarian, D., Gu, Y., Aluri, K. C., Enders, J., Brown, K. M., et al. (2020) · Nucleic Acid Therapeutics · doi:10.1089/nat.2019.0840 · Cited by 30 · see citing works

Rare versus common diseases: a false dichotomy in precision medicine

Chung, B. H., Chau, J. F. T., & Wong, G. K. (2021) · npj Genomic Medicine · doi:10.1038/s41525-021-00176-x · Cited by 30 · see citing works

Synthesis of some cluster galactosides and their effect on the hepatic galactose-binding system

Kawaguchi, K., Kuhlenschmidt, M. S., Roseman, S., & Lee, Y. C. (1980) · Archives of Biochemistry and Biophysics · doi:10.1016/0003-9861(80)90121-6 · Cited by 29 · see citing works

The Relationship between Bicontinuous Inverted Cubic Phases and Membrane Fusion

Siegel, D. P. (2005) · Surfactant science · doi:10.1201/9781420027709.ch4 · Cited by 26 · see citing works

Inclisiran: How Widely and When Should We Use It?

Pirillo, A., & Catapano, A. L. (2022) · Current Atherosclerosis Reports · doi:10.1007/s11883-022-01056-0 · Cited by 26 · see citing works

Evaluation of the synergistic adverse effects of concomitant therapy with statins and fibrates on rhabdomyolysis

Matzno, S., Tazuya-Murayama, K., Tanaka, H., Yasuda, S., Mishima, M., Uchida, T., Nakabayashi, T., & Matsuyama, K. (2003) · Journal of Pharmacy and Pharmacology · doi:10.1211/002235703765951401 · Cited by 26 · see citing works

Composition, Antigenic Properties and Hepatocyte Surface Expression of the Woodchuck Asialoglycoprotein Receptor

Diao, J., & Michalak, T. I. (1996) · Journal of Receptors and Signal Transduction · doi:10.3109/10799899609039951 · Cited by 12 · see citing works

Cholesterol lowering with inclisiran: a new chapter in the PCSK9 story book

Santos, R. D., & Rocha, V. Z. (2022) · European Heart Journal · doi:10.1093/eurheartj/ehac656 · Cited by 9 · see citing works

Ezetimibe (Zetia)

Brar, K. (2004) · Medical Journal Armed Forces India · doi:10.1016/s0377-1237(04)80019-4 · Cited by 9 · see citing works

siRNA Design and GalNAc-Empowered Hepatic Targeted Delivery

Lu, M., Zhang, M., Hu, B., & Huang, Y. (2021) · Methods in molecular biology · doi:10.1007/978-1-0716-1298-9_6 · Cited by 8 · see citing works

[PCSK9 inhibition--A new era in cholesterol treatment].

OLSSON, A. G. (2015) · PubMed · Cited by 2 · see citing works

Targeting proprotein convertase subtilisin/kexin type 9 (PCSK9): From bench to bedside

Bao, X., Liang, Y., Chang, H., Cai, T., Feng, B., Gordon, K., Zhu, Y., Shi, H., He, Y., & Xie, L. (2024) · Signal Transduction and Targeted Therapy · doi:10.1038/s41392-023-01690-3 · Cited by 187 · see citing works

An Update: Enzymatic Synthesis for Industrial Applications

Bayer, T., Wu, S., Šnajdrová, R., Baldenius, K., & Bornscheuer, U. T. (2025) · Angewandte Chemie International Edition · doi:10.1002/anie.202505976 · Cited by 98 · see citing works

Advances with Lipid-Based Nanosystems for siRNA Delivery to Breast Cancers

Subhan, M. A., Filipczak, N., & Torchilin, V. P. (2023) · Pharmaceuticals · doi:10.3390/ph16070970 · Cited by 66 · see citing works

Recent Update on siRNA Therapeutics

Ebenezer, O., Oyebamiji, A. K., Olanlokun, J. O., Tuszyński, J. A., & Wong, G. K. (2025) · International Journal of Molecular Sciences · doi:10.3390/ijms26083456 · Cited by 66 · see citing works

The recent blooming of therapeutic aptamers

Cesarini, V., Appleton, S. L., de Franciscis, V., & Catalucci, D. (2025) · Molecular Aspects of Medicine · doi:10.1016/j.mam.2025.101350 · Cited by 46 · see citing works

Applications and Research Advances in the Delivery of CRISPR/Cas9 Systems for the Treatment of Inherited Diseases

Lu, X., Zhang, M., Li, G., Zhang, S., Zhang, J., Fu, X., & Sun, F. (2023) · International Journal of Molecular Sciences · doi:10.3390/ijms241713202 · Cited by 34 · see citing works

PCSK9 Inhibitors: Focus on Evolocumab and Its Impact on Atherosclerosis Progression

Abduljabbar, M. H. (2024) · Pharmaceuticals · doi:10.3390/ph17121581 · Cited by 20 · see citing works

Types of RNA therapeutics

Goleij, P., Babamohamadi, M., Rezaee, A., Sanaye, P. M., Tabari, M. A. K., Sadreddini, S., Arefnezhad, R., & Motedayyen, H. (2024) · Progress in molecular biology and translational science · doi:10.1016/bs.pmbts.2023.12.022 · Cited by 19 · see citing works

Improved biocompatibility of dendrimer-based gene delivery by histidine-modified nuclear localization signals

Lee, J., Kwon, Y., Edwards, S., Guim, H., & Jeong, K. J. (2023) · International Journal of Pharmaceutics · doi:10.1016/j.ijpharm.2023.123299 · Cited by 12 · see citing works

Treatment with Proprotein Convertase Subtilisin/Kexin Type 9 Inhibitors (PCSK9i): Current Evidence for Expanding the Paradigm?

Giglio, R. V., Muzurović, E. M., Patti, A. M., Toth, P. P., Agarwal, M. A., Almahmeed, W. A., Klisic, A. N., Ciaccio, M., & Rizzo, M. (2023) · Journal of Cardiovascular Pharmacology and Therapeutics · doi:10.1177/10742484231186855 · Cited by 11 · see citing works

An update on inclisiran for the treatment of elevated LDL cholesterol

Barkas, F., & Ray, K. (2024) · Expert Opinion on Pharmacotherapy · doi:10.1080/14656566.2024.2337253 · Cited by 9 · see citing works

Graphene oxide and silicene as 2D platforms for complexation and intracellular delivery of siRNA

Razavi, L., Raissi, ‪., & Farzad, F. (2024) · Journal of Drug Delivery Science and Technology · doi:10.1016/j.jddst.2024.105514 · Cited by 8 · see citing works

Cutting-edge lipid-lowering pharmacological therapies: Improving lipid control beyond statins

Capuozzo, M., Ottaiano, A., Cinque, C., Farace, S., & Ferrara, F. (2025) · Hipertensión y Riesgo Vascular · doi:10.1016/j.hipert.2024.12.002 · Cited by 8 · see citing works

Inclisiran and cardiovascular events: a comprehensive review of efficacy, safety, and future perspectives

Mohamed, A. A. M., & Ray, K. K. (2023) · Current Opinion in Cardiology · doi:10.1097/hco.0000000000001074 · Cited by 7 · see citing works

The Fomivirsen, Patisiran, and Givosiran Odyssey: How the Success Stories May Pave the Way for Future Clinical Translation of Nucleic Acid Drugs

Mansouri, M., Mansouri, K., Taheri, Z., Alhashemi, S. H., & Dehshahri, A. (2025) · BioDrugs · doi:10.1007/s40259-025-00711-7 · Cited by 7 · see citing works

Inclisiran SiRNA therapy for durable LDL-C reduction: a systematic review and meta-analysis highlighting a breakthrough in long-term cardiovascular risk management

Satyam, S. M., El-Tanani, M., Patni, M. A., Rehman, A., Irshad, S. M., Raheem, R., Junais, E., John, A. A., Rassal, R. Y., Kumari, R., et al. (2026) · BMC Cardiovascular Disorders · doi:10.1186/s12872-026-05515-3 · Cited by 5 · see citing works

Update: Enzymatische Synthese für industrielle Anwendungen

Bayer, T., Wu, S., Šnajdrová, R., Baldenius, K., & Bornscheuer, U. T. (2025) · Angewandte Chemie · doi:10.1002/ange.202505976 · Cited by 4 · see citing works

Therapeutic precision gene editing of cholesterol pathways as a gene therapy strategy for cardiovascular disease

Erbasan, E., Aliciaslan, M., Erendor, F., Şanlioğlu, A. D., & Şanlıoğlu, S. (2025) · Gene Therapy · doi:10.1038/s41434-025-00575-0 · Cited by 4 · see citing works

Blocking Translation of Oncogenic mRNA

Christie, K. N. (2023) · Journal of Cancer Therapy · doi:10.4236/jct.2023.146021 · Cited by 4 · see citing works

2024 KSoLA Update on New Lipid-Lowering Agents: Inclisiran and Bempedoic Acid

Kim, H., Cha, J., & Lee, S. (2025) · Journal of Lipid and Atherosclerosis · doi:10.12997/jla.2025.14.2.135 · Cited by 6 · see citing works

A Facile and Promising Delivery Platform for siRNA to Solid Tumors

Leng, Q., Anand, A., & Mixson, A. J. (2024) · Molecules · doi:10.3390/molecules29235541 · Cited by 3 · see citing works

A Genome-Wide CRISPR Screen Identifies Sortilin as the Receptor Responsible for Galectin-1 Lysosomal TraffickingPreprint

Donnelly, J., Kamber, R. A., Wisnovsky, S., Roberts, D. S., Peltan, E. L., Bassik, M. C., & Bertozzi, C. R. (2024) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.1101/2024.01.03.574113 · Cited by 3 · see citing works

Designing of potential siRNA molecules for African norovirus gene silencing: A computational approach

Ebenezer, O., Oyebamiji, A. K., Olanrewaju, A. A., Akinola, O. T., Afolabi, S. O., Olaseinde, A. A., & Tuszyński, J. A. (2024) · Pharmacological Research - Reports · doi:10.1016/j.prerep.2024.100021 · Cited by 2 · see citing works

A Novel Pot-Economy Approach to the Synthesis of Triantennary GalNAc-Oligonucleotide

Gusev, A. E., Ivanov, V., Dmitriev, N. A., Kholstov, A. V., Vasilichin, V. A., Кофиади, И. А., & Khaitov, M. R. (2024) · Molecules · doi:10.3390/molecules29245959 · Cited by 2 · see citing works

Unveiling the Interaction Mechanism of siRNA with Lipid Bilayers of Different Types for siRNA-Based Therapy

Zheng, D., Lu, Z. G., Li, J., Dong, J., Zhang, X., Zhang, X., & Cao, D. (2025) · The Journal of Physical Chemistry B · doi:10.1021/acs.jpcb.4c07520 · Cited by 2 · see citing works

Advancements In Pharmacotherapy for Hyperlipidemia: A Comprehensive Review of Recent Drug Innovations and Clinical Outcomes

Dey, H., Agarwal, V., Ali, M. N., Upadhyay, A., Shafieq, S., Dixit, B., Tomar, S., & Tarannum, M. (2024) · Journal of Drug Delivery and Therapeutics · doi:10.22270/jddt.v14i4.6533 · Cited by 1 · see citing works

Long-Acting RNA Interference Therapies for Cardiovascular Risk Reduction: Current Evidence and Emerging Targets

Fadah, K., Alomari, M., Elchouemi, M., & Lange, R. A. (2026) · Current Cardiology Reports · doi:10.1007/s11886-026-02362-8 · Cited by 1 · see citing works

Гиполипидемическая терапия в особых ситуациях

Арутюнов, Г. П., Владиславович, Е. М., Alieva, A. S., Иосифович, Ш. И., Orlova, Y. A., Koziolova, N. A., Тарловская, Е. И., Григорьева, Н. Ю., & Chesnikova, A. I. (2024) · Атеросклероз и дислипидемии · doi:10.34687/2219-8202.jad.2024.03.0008 · Cited by 1 · see citing works

"Potential Application of Oligonucleotide Therapeutics in SARS-CoV-2 Genomic RNA Knockdown"

Inagaki, M. (2023) · Biomedical Journal of Scientific & Technical Research · doi:10.26717/bjstr.2023.53.008460

Supercharged coiled‐coil protein with N‐terminal decahistidine tag boosts siRNA complexation and delivery efficiency of a lipoproteoplex

Sun, J. W., Thomas, J. S., Monkovic, J., Gibson, H., Nagapurkar, A., Frezzo, J. A., Katyal, P., Punia, K., Mahmoudinobar, F., Renfrew, P. D., et al. (2024) · Journal of Peptide Science · doi:10.1002/psc.3594

Novel therapies for lowering low-density lipoprotein levels for atherosclerotic cardiovascular disease prevention: reaching the target where others have failed

Dziewierz, A., Bobrowska, B., Streb, W., Pelliccia, F., Bartuś, S., & Rajtar-Salwa, R. (2025) · Expert Review of Cardiovascular Therapy · doi:10.1080/14779072.2025.2570839

RNA-Based Therapies for Hypercholesterolemia and Coronary Artery Disease

Mendoza, M. J. L., Figueroa, N. C., Mena, M. J. V., Ulloa, A., Vindas, J. J., & Estrada, M. A. S. (2026) · Cureus · doi:10.7759/cureus.103877

O-Glycosylation as an energy sensor: Related molecular mechanisms, disease associations, and therapeutic opportunities in type 2 diabetes

Shu, T., Han, X., & Zhu, Y. (2026) · Genes & Diseases · doi:10.1016/j.gendis.2026.102165

Nanoparticle-Based PCSK9 Inhibition in Viral Infections: Evaluating Clinical Limitations and Advancing Therapeutic Strategies

Dababneh, M. F., Alhasso, B., Shareef, A., Kyada, A., Malathi, H., Maharana, L., Puri, D., Gupta, H., Yarmukhamedova, N. A., Sameer, H. N., et al. (2026) · Journal of Pharmaceutical Innovation · doi:10.1007/s12247-026-10651-6

Dyslipidaemia: Pathophysiology, Epidemiology, Diagnostic Advances, and Emerging Therapeutic Strategies

Qadir, A. M., Omer, R. A., Barzani, H. A. H., & Sulaiman, S. H. (2026) · Russian Journal of Bioorganic Chemistry · doi:10.1134/s1068162025603581

Bio-intelligent delivery systems for regenerative medicine: From living biomaterials to clinical translation

Singh, M., Tripathi, D., & Maity, D. (2026) · International Journal of Pharmaceutics · doi:10.1016/j.ijpharm.2026.127410

Investigation for the cause of vial breakage during frozen storage of nucleic acid drug solution and the effect of the internal coating on the vial strain

Taga, H., Kodama, T., Ogawa, T., Hasegawa, S., Arai, H., Hirota, K., & Fukami, T. (2026) · Journal of Pharmaceutical Sciences · doi:10.1016/j.xphs.2026.104526

Harnessing the Potential of Phospholipids as Pharmaceutical Excipients: A Versatile Platform for Customized Drug Delivery Systems

Joseph, A. T., & Ravindran, S. (2026) · Drug Development and Industrial Pharmacy · doi:10.1080/03639045.2026.2752934

Inclisiran: a new promising agent in the management of hypercholesterolemia

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

Small Interfering RNA Therapeutic Inclisiran: A New Approach to Targeting PCSK9

German, C. A., & Shapiro, M. D. (2019) · BioDrugs · doi:10.1007/s40259-019-00399-6 · Cited by 115 · see citing works

Small Interfering Ribonucleic Acid as Lipid-Lowering Therapy: Inclisiran in Focus

Rakočević, J., Dobric, M. R., Vučić, R. M., Furtula, M., Zaletel, I. V., Milutinović, K., Ilijevski, A., Labudović‐Borović, M., Tomasevic, M. V., & Bajčetić, M. (2023) · International Journal of Molecular Sciences · doi:10.3390/ijms24066012 · Cited by 4 · see citing works

Inclisiran: a small interfering RNA strategy targeting PCSK9 to treat hypercholesterolemia

Banerjee, Y., Pantea Stoian, A., Cicero, A. F. G., Fogacci, F., Nikolic, D., Sachinidis, A., Rizvi, A. A., Janez, A., & Rizzo, M. (2022) · Expert Opinion on Drug Safety · doi:10.1080/14740338.2022.1988568 · Cited by 54 · see citing works

Inclisiran—Safety and Effectiveness of Small Interfering RNA in Inhibition of PCSK-9

Wołowiec, Ł., Osiak, J., Wołowiec, A., Wijata, A., Grześk, E., Kozakiewicz, M., Banach, J., Nowaczyk, A., Nowaczyk, J., & Grześk, G. (2023) · Pharmaceutics · doi:10.3390/pharmaceutics15020323 · Cited by 43 · see citing works