The DOI matches its Crossref record; 254 of the 254 references it cites were found in a registry or an index.
Lipid-Based DNA Therapeutics: Hallmarks of Non-Viral Gene Delivery
Abstract
Gene therapy is a promising strategy for the treatment of monogenic disorders. Non-viral gene delivery systems including lipid-based DNA therapeutics offer the opportunity to deliver an encoding gene sequence specifically to the target tissue and thus enable the expression of therapeutic proteins in diseased cells. Currently, available gene delivery approaches based on DNA are inefficient and require improvements to achieve clinical utility. In this Review, we discuss state-of-the-art lipid-based DNA delivery systems that have been investigated in a preclinical setting. We emphasize factors influencing the delivery and subsequent gene expression in vitro, ex vivo, and in vivo. In addition, we cover aspects of nanoparticle engineering and optimization for DNA therapeutics. Finally, we highlight achievements of lipid-based DNA therapies in clinical trials.
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Cites
Items 201–254 of 254
Structure, Function, and Evolution of Proton-ATPases
Nelson, N. (1988) · PLANT PHYSIOLOGY · doi:10.1104/pp.86.1.1 · Cited by 83
Large scale production of DC-Chol cationic liposomes by microfluidization
Sorgi, F. L., & Huang, L. (1996) · International Journal of Pharmaceutics · doi:10.1016/s0378-5173(96)04733-3 · Cited by 80
Targeted Expression of BikDD Eradicates Pancreatic Tumors in Noninvasive Imaging Models
Xie, X., Xia, W., Li, Z., Kuo, H.-P., Liu, Y., Li, Z., Ding, Q., Zhang, S., Spohn, B., Yang, Y., et al. (2007) · Cancer Cell · doi:10.1016/j.ccr.2007.05.009 · Cited by 77
Effect of liposomalization on the antitumor activity, side-effects and tissue distribution of CPT-11
Sadzuka, Y., Hirotsu, S., & Hirota, S. (1998) · Cancer Letters · doi:10.1016/s0304-3835(98)00031-7 · Cited by 74
Growth of dissociated neurons in culture dishes coated with synthetic polymeric amines
Rüegg, U. T., & Hefti, F. F. (1984) · Neuroscience Letters · doi:10.1016/0304-3940(84)90309-4 · Cited by 72
Combinatorial Library of Lipidoids for In Vitro DNA Delivery
Sun, S., Wang, M., Knupp, S. A., Soto‐Feliciano, Y. M., Hu, X., Kaplan, D. L., Langer, R., Anderson, D. G., & Xu, Q. (2011) · Bioconjugate Chemistry · doi:10.1021/bc200572w · Cited by 72
Minicircle DNA Provides Enhanced and Prolonged Transgene Expression Following Airway Gene Transfer
Munye, M. M., Tagalakis, A. D., Barnes, J. L., Brown, R. E., McAnulty, R. J., Howe, S. J., & Hart, S. L. (2016) · Scientific Reports · doi:10.1038/srep23125 · Cited by 72
Mukherjee, A. B., Orloff, S., Butler, J. D., Triche, T. J., Lalley, P. A., & Schulman, J. D. (1978) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.75.3.1361 · Cited by 70
Chen, S., Zaifman, J., Kulkarni, J. A., Zhigaltsev, I. V., Tam, Y. K., Ciufolini, M. A., Tam, Y. Y. C., & Cullis, P. R. (2018) · Journal of Controlled Release · doi:10.1016/j.jconrel.2018.07.026 · Cited by 68
Self-citationDiscontinued in 2013: oncology drugs
Williams, R. (2014) · Expert Opinion on Investigational Drugs · doi:10.1517/13543784.2015.971154 · Cited by 67
A Phase l Study of a Tumor-targeted Systemic Nanodelivery System, SGT-94, in Genitourinary Cancers
Siefker-Radtke, A. O., Zhang, X.-Q., Guo, C. C., Shen, Y., Pirollo, K. F., Sabir, S. H., Leung, C., Leong-Wu, C. A., Ling, C.-M., Chang, E. H., et al. (2016) · Molecular Therapy · doi:10.1038/mt.2016.118 · Cited by 66
CAR-T cell therapy in ovarian cancer: from the bench to the bedside
Zhu, X., Cai, H., Zhao, L., Ning, L., & Lang, J. (2017) · Oncotarget · doi:10.18632/oncotarget.19929 · Cited by 66
Lai, S. Y., Koppikar, P., Thomas, S. M., Childs, E. E., Egloff, A. M., Seethala, R. R., Branstetter, B. F., Gooding, W. E., Muthukrishnan, A., Mountz, J. M., et al. (2009) · Journal of Clinical Oncology · doi:10.1200/jco.2008.17.8251 · Cited by 65
Therapeutic implications of tumor interstitial fluid pressure in subcutaneous RG-2 tumors
Navalitloha, Y. (2006) · Neuro-Oncology · doi:10.1215/15228517-2006-007 · Cited by 64
Densmore, C. L., Giddings, T. H., Waldrep, J. C., Kinsey, B. M., & Knight, V. (1999) · The Journal of Gene Medicine · Cited by 62
Combinatorial Approach to Nanoarchitectonics for Nonviral Delivery of Nucleic Acids
Molla, M. R., & Levkin, P. A. (2015) · Advanced Materials · doi:10.1002/adma.201502888 · Cited by 62
Phase 2 trial of Allovectin-7 in advanced metastatic melanoma
Gonzalez, R. C., Hutchins, L. E., Nemunaitis, J. J., et al. (2006) · Melanoma Research · doi:10.1097/01.cmr.0000232299.44902.41 · Cited by 61
Differential Endosomal Pathways for Radically Modified Peptide Vectors
Arukuusk, P., Pärnaste, L., Margus, H., Eriksson, J., Vasconcelos, D., Padari, K., Pooga, M., & Langel, Ü. (2013) · Bioconjugate Chemistry · doi:10.1021/bc4002757 · Cited by 60
Gopalan, B., Ito, I., Branch, C. D., Stephens, C. L., Roth, J. A., & Ramesh, R. (2004) · Technology in Cancer Research & Treatment · doi:10.1177/153303460400300615 · Cited by 59
Meisel, J. W., & Gokel, G. W. (2016) · Scientific Reports · doi:10.1038/srep27662 · Cited by 59
Pirollo, K. F., Rait, A. S., Zhou, Q., et al. (2008) · Clinical Cancer Research · doi:10.1158/1078-0432.ccr-07-1951 · Cited by 56
Guerra‐Crespo, M. (2003) · Journal of Neuroscience Methods · doi:10.1016/s0165-0270(03)00125-0 · Cited by 54
Yuba, E., Nakajima, Y., Tsukamoto, K., Iwashita, S., Kojima, C., Harada, A., & Kono, K. (2012) · Journal of Controlled Release · doi:10.1016/j.jconrel.2012.04.002 · Cited by 53
Aerosol Delivery of Lipid:DNA Complexes to Lungs of Rhesus Monkeys
McDonald, R. J., Liggitt, D., Roche, L. M., Nguyen, H. T., Pearlman, R., Raabe, O. G., Bussey, L. B., & Gorman, C. M. (1998) · Pharmaceutical Research · doi:10.1023/a:1011902532163 · Cited by 51
Intracellular trafficking of nucleic acids
Zhou, R., Geiger, R., & Dean, D. A. (2004) · Expert Opinion on Drug Delivery · doi:10.1517/17425247.1.1.127 · Cited by 50
Use of CRISPR/Cas9 gene-editing tools for developing models in drug discovery
Ahmad, G., & Amiji, M. M. (2018) · Drug Discovery Today · doi:10.1016/j.drudis.2018.01.014 · Cited by 49
Zebrafish as a predictive screening model to assess macrophage clearance of liposomes in vivo
Sieber, S., Grossen, P., Uhl, P., Detampel, P., Mier, W., Witzigmann, D., & Huwyler, J. (2019) · Nanomedicine Nanotechnology Biology and Medicine · doi:10.1016/j.nano.2018.11.017 · Cited by 49
Self-citationStructural and Formulation Factors Influencing Pyridinium Lipid-Based Gene Transfer
Zhu, L., Lü, Y., Miller, D. D., & Mahato, R. I. (2008) · Bioconjugate Chemistry · doi:10.1021/bc8004039 · Cited by 48
Zhong, Chen, Sun, Lü, Y., Tao, T., Huang, Zhang, Yu, Y., Gao, J., & Zou (2013) · International Journal of Nanomedicine · doi:10.2147/ijn.s42800 · Cited by 45
Malloggi, C., Pezzoli, D., Magagnin, L., Nardo, L. D., Mantovani, D., Tallarita, E., & Candiani, G. (2015) · Polymer Chemistry · doi:10.1039/c5py00915d · Cited by 44
Alton, E. W. F. W., Armstrong, D. K., Ashby, D., Bayfield, K. J., Bilton, D., Bloomfield, E. V., Boyd, A., Brand, J., Buchan, R., Calcedo, R., et al. (2016) · Efficacy and Mechanism Evaluation · doi:10.3310/eme03050 · Cited by 44
Ball, R. L., Knapp, C. M., & Whitehead, K. A. (2015) · PLoS ONE · doi:10.1371/journal.pone.0133154 · Cited by 43
Development of Cationic Quaternary Ammonium Sulfonamide Amino Lipids for Nucleic Acid Delivery
Miller, J. B., Kós, P., Tieu, V., Zhou, K., & Siegwart, D. J. (2017) · ACS Applied Materials & Interfaces · doi:10.1021/acsami.7b15982 · Cited by 43
Sang, Y., Xie, K., Mu, Y., Lei, Y., Zhang, B., Xiong, S., Chen, Y., & Qi, N. (2013) · Cytotechnology · doi:10.1007/s10616-013-9658-z · Cited by 42
Malley, B. W. O., Li, D., McQuone, S. J., & Ralston, R. (2005) · The Laryngoscope · doi:10.1097/00005537-200503000-00002 · Cited by 40
Studies of direct intratumoral gene transfer using cationic lipid-complexed plasmid DNA
Clark, P. R., Stopeck, A., Ferrari, M. E., Parker, S. E., & Hersh, E. M. (2000) · Cancer Gene Therapy · doi:10.1038/sj.cgt.7700184 · Cited by 35
The effect of cationic liposome pretreatment and centrifugation on retrovirus-mediated gene transfer
Swaney, W., Sorgi, F., Bahnson, A., & Barranger, J. A. (1997) · Gene Therapy · doi:10.1038/sj.gt.3300529 · Cited by 34
Drug Delivery–mediated Control of RNA Immunostimulation
Nguyen, D. N., Chen, S., Lu, J. J., Goldberg, M. S., Kim, P., Sprague, A., Novobrantseva, T., Sherman, J., Shulga-Morskaya, S., de Fougerolles, A., et al. (2009) · Molecular Therapy · doi:10.1038/mt.2009.147 · Cited by 34
Functional delivery of large genomic DNA to human cells with a peptide‐lipid vector
White, R. E., Wade‐Martins, R., Hart, S. L., Frampton, J., Huey, B. D., Desai‐Mehta, A., Cerosaletti, K. M., CONCANNON, P. J., & James, M. R. (2003) · The Journal of Gene Medicine · doi:10.1002/jgm.420 · Cited by 32
Biocompatible Polymer–Peptide Hybrid-Based DNA Nanoparticles for Gene Delivery
Witzigmann, D., Wu, D., Schenk, S., Balasubramanian, V., Meier, W. P., & Huwyler, J. (2015) · ACS Applied Materials & Interfaces · doi:10.1021/acsami.5b01684 · Cited by 31
Self-citationTumor suppressor candidate 2 (TUSC2, FUS-1) and human cancers.
Rimkus, T. K., Sirkisoon, S. R., Harrison, A., & Lo, H. (2017) · PubMed · Cited by 28
Cao, X., Majidi, M., Feng, M., Shao, R., Wang, J., Zhao, Y., Baladandayuthapani, V., Song, J., Fang, B., Ji, L., et al. (2016) · Scientific Reports · doi:10.1038/srep35741 · Cited by 26
Recent Advances in Nucleic Acid-Based Delivery: From Bench to Clinical Trials in Genetic Diseases.
Oliveira, C., Ribeiro, A., Veiga, F., & Silveira, I. (2016) · Journal of Biomedical Nanotechnology · doi:10.1166/jbn.2016.2245 · Cited by 25
Improving transfection efficiency of ultrapure oligochitosan/DNA polyplexes by medium acidification
Agirre, M., Sesma, J. Z., Puras, G., Ojeda, E., & Pedraz, J. L. (2014) · Drug Delivery · doi:10.3109/10717544.2013.871373 · Cited by 24
Optimization of transfection methods for Huh-7 and Vero cells: A comparative study
Hashemi, A., Roohvand, F., Ghahremani, M. H., et al. (2012) · Cytology and Genetics · doi:10.3103/s0095452712060035 · Cited by 23
Combinatorial Synthesis and High-Throughput Screening of Alkyl Amines for Nonviral Gene Delivery
Li, L. X., Wang, F.-J., Wu, Y., Davidson, G. R., & Levkin, P. A. (2013) · Bioconjugate Chemistry · doi:10.1021/bc400158w · Cited by 23
Ju, J. Q., Huan, M., Wan, N., Hou, Y., Ma, X.-X., Jia, Y., Li, C., Zhou, S., & Zhang, B. (2016) · Bioorganic & Medicinal Chemistry Letters · doi:10.1016/j.bmcl.2016.04.007 · Cited by 23
Transfection of primary tumor cells and tumor cell lines with plasmid DNA/lipid complexes.
Stopeck, A., Hersh, E. M., Brailey, J. L., et al. (1998) · PubMed · Cited by 21
Tam, Y. K., Madden, T. D., & Hope, M. J. (2016) · Journal of Drug Targeting · doi:10.1080/1061186x.2016.1221955 · Cited by 20
Joris, F., Smedt, S. C. D., & Raemdonck, K. (2017) · Nano Today · doi:10.1016/j.nantod.2017.06.012 · Cited by 17
Assessment of a Modular Transfection System Based upon Cellular Localization of DNA
Guo, C., & Gemeinhart, R. A. (2004) · Molecular Pharmaceutics · doi:10.1021/mp049969a · Cited by 12
Single-Tailed Lipidoids Enhance the Transfection Activity of Their Double-Tailed Counterparts
Wu, Y., Li, L. X., Chen, Q., Su, Y., Levkin, P. A., & Davidson, G. R. (2015) · ACS Combinatorial Science · doi:10.1021/acscombsci.5b00117 · Cited by 11
Technology evaluation: gene therapy (IL-2), Valentis Inc.
Morse, M. A. (2000) · PubMed · Cited by 11
Harvie (2000) · doi:10.1002/(sici)1520-6017(200005)89:5<652::aid-jps11>3.0.co;2-h
Cited by
Drug delivery systems for RNA therapeutics
Paunovska, K., Loughrey, D., & Dahlman, J. E. (2022) · Nature Reviews Genetics · doi:10.1038/s41576-021-00439-4 · Cited by 1,349
Lipid nanoparticle technology for therapeutic gene regulation in the liver
Witzigmann, D., Kulkarni, J. A., Leung, J., Chen, S., Cullis, P. R., & van der Meel, R. (2020) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2020.06.026 · Cited by 376
Self-citationPolymeric Delivery of Therapeutic Nucleic Acids
Kumar, R., Santa Chalarca, C. F., Bockman, M. R., Bruggen, C. V., Grimme, C. J., Dalal, R. J., Hanson, M. G., Hexum, J. K., & Reineke, T. M. (2021) · Chemical Reviews · doi:10.1021/acs.chemrev.0c00997 · Cited by 365
Neutral Lipopolyplexes for In Vivo Delivery of Conventional and Replicative RNA Vaccine
Perche, F., Clemençon, R., Schulze, K., Ebensen, T., Guzmán, C. A., & Pichon, C. (2019) · Molecular Therapy - Nucleic Acids · doi:10.1016/j.omtn.2019.07.014 · Cited by 55
An inflammation self-adaptive nanocarrier for highly efficient gene therapy
Jia, H., Yang, Y., Li, M., Li, Y., Han, X., Li, J., Zhang, X., Fan, C., Wu, T., Cui, C., et al. (2020) · Materials Today Chemistry · doi:10.1016/j.mtchem.2020.100287 · Cited by 3
Metadata
- Authors
- Buck, J., Grossen, P., Cullis, P. R., Huwyler, J., & Witzigmann, D.
- Venue
- ACS Nano
- Publisher
- American Chemical Society (ACS)
- Published
- 2019-03-25
- Volume
- 13
- Issue
- 4
- Pages
- 3754-3782
- ISSN
- 1936-0851 (ISSN-L)
- 1936-086X
- OpenAlex
- W2925126737
- Cited by
- 312
- References
- 254
- FWCI
- 11.67
- Type
- journal article
- Language
- en
- Open access
- closed
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