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Lipid-based Nanoparticles For Nucleic Acid Delivery

Weijun Li, F. Szoka
Published 2006 · Chemistry, Medicine

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AbstractLipid-based colloidal particles have been extensively studied as systemic gene delivery carriers. The topic that we would like to emphasize is the formulation/assembly of lipid-based nanoparticles (NP) with diameter under 100 nm for delivering nucleic acid in vivo. NP are different from cationic lipid–nucleic acid complexes (lipoplexes) and are vesicles composed of lipids and encapsulated nucleic acids with a diameter less than 100 nm. The diameter of the NP is an important attribute to enable NP to overcome the various in vivo barriers for systemic gene delivery such as: the blood components, reticuloendothelial system (RES) uptake, tumor access, extracellular matrix components, and intracellular barriers. The major formulation factors that impact the diameter and encapsulation efficiency of DNA-containing NP include the lipid composition, nucleic acid to lipid ratio and formulation method. The particle assembly step is a critical one to make NP suitable for in vivo gene delivery. NP are often prepared using a dialysis method either from an aqueous-detergent or aqueous-organic solvent mixture. The resulting particles have diameters about 100 nm and nucleic acid encapsulation ratios are >80%. Additional components can then be added to the particle after it is formed. This ordered assembly strategy enables one to optimize the particle physico-chemical attributes to devise a biocompatible particle with increased gene transfer efficacy in vivo. The components included in the sequentially assembled NP include: poly(ethylene glycol) (PEG)-shielding to improve the particle pharmacokinetic behavior, a targeting ligand to facilitate the particle–cell recognition and in some case a bioresponsive lipid or pH-triggered polymer to enhance nucleic acid release and intracellular trafficking. A number of groups have observed that a PEG-shielded NP is a robust and modestly effective system for systemic gene or small interfering RNA (siRNA) delivery.
This paper references
10.1016/S0006-3495(03)74986-8
Mechanism of pH-triggered collapse of phosphatidylethanolamine liposomes stabilized by an ortho ester polyethyleneglycol lipid.
Xin Guo (2003)
a new barrier for non-viral gene delivery
E. Dauty (2005)
formulation and transfection properties
Y. P. Zhang (1999)
implications for membrane fusion mechanisms
D. P. Siegel (1997)
10.3109/08982109609031137
How are Nucleic Acids Released in Cells from Cationic Lipid-Nucleic Acid Complexes?
F. Szoka (1996)
Receptor - specific delivery of liposomes via folatePEG
T. Lee (2000)
construction and characterization
J. J. Wheeler (1999)
10.1074/JBC.271.14.8481
Folate-targeted, Anionic Liposome-entrapped Polylysine-condensed DNA for Tumor Cell-specific Gene Transfer (*)
R. Lee (1996)
10.1201/9780203833445
Molecular Biology of the Cell
J. Davies (1983)
10.1038/sj.gt.3300821
Stabilized plasmid-lipid particles: construction and characterization
J J Wheeler (1999)
10.1016/J.BBAMEM.2005.02.001
Stabilized plasmid-lipid particles containing PEG-diacylglycerols exhibit extended circulation lifetimes and tumor selective gene expression.
E. Ambegia (2005)
10.4324/9780203300961
Pharmaceutical Perspectives of Nucleic Acid-Based Therapeutics
R. I. Mahato (2002)
10.1016/J.TAAP.2004.04.017
Toxicology of antisense therapeutics.
T. Jason (2004)
Tumor - targeted p 53gene therapy enhances the efficacy of conventional chemo / radiother
K. F. Pirollo L. Xu (2001)
10.1385/1592591396
Nonviral vectors for gene therapy
L. Huang (1999)
10.1021/BC025625W
Low-pH-sensitive PEG-stabilized plasmid-lipid nanoparticles: preparation and characterization.
J. S. Choi (2003)
10.1038/sj.gt.3300965
Stabilized plasmid-lipid particles for regional gene therapy: formulation and transfection properties
Y. Zhang (1999)
10.1002/(SICI)1520-6017(200005)89:5<652::AID-JPS11>3.0.CO;2-H
Use of poly(ethylene glycol)-lipid conjugates to regulate the surface attributes and transfection activity of lipid-DNA particles.
P. Harvie (2000)
a systemic gene therapy vector
D. B. Fenske (2002)
10.1038/sj.gt.3301308
Stabilized plasmid-lipid particles for systemic gene therapy
P. Tam (2000)
[Cationic liposomes in gene delivery].
T. Yotsuyanagi (1998)
10.1021/JA015867R
Dimerizable cationic detergents with a low cmc condense plasmid DNA into nanometric particles and transfect cells in culture.
E. Dauty (2001)
10.1016/0304-4157(92)90038-C
Sterically stabilized liposomes.
M. Woodle (1992)
10.1002/jgm.172
Enhanced gene delivery to human airway epithelial cells using an integrin‐targeting lipoplex
E. S. Scott (2001)
10.1002/jgm.619
Gene therapy clinical trials worldwide 1989–2004—an overview
M. Edelstein (2004)
10.1038/nrd1960
Artificial viruses: a nanotechnological approach to gene delivery
Enrico Mastrobattista (2006)
10.1016/J.YMTHE.2005.09.014
Hypersensitivity and loss of disease site targeting caused by antibody responses to PEGylated liposomes.
A. Judge (2006)
10.1126/SCIENCE.275.5301.810
Structure of DNA-Cationic Liposome Complexes: DNA Intercalation in Multilamellar Membranes in Distinct Interhelical Packing Regimes
J. Rädler (1997)
10.1016/S0006-3495(99)76894-3
Physicochemical characterization and purification of cationic lipoplexes.
Y. Xu (1999)
PREPARATION AND CHARACTERIZATION OF
Cecilia Paglier (1996)
10.1038/sj.gt.3302699
Genospheres: self-assembling nucleic acid-lipid nanoparticles suitable for targeted gene delivery
M. E. Hayes (2006)
Nonviral Vectors for Gene Therapy. Academic
L Huang (1999)
Long-circulating vectors for the systemic delivery of genes.
D. Fenske (2001)
10.1038/nature04688
RNAi-mediated gene silencing in non-human primates
T. Zimmermann (2006)
10.1101/pdb.prot4450
Bioresponsive targeted charge neutral lipid vesicles for systemic gene delivery.
Weijun Li (2006)
10.1006/MTHE.2001.0425
Intratumoral delivery of p2CMVmIL-12 using water-soluble lipopolymers.
R. Mahato (2001)
10.1093/NAR/29.17.3694
Development of an effective gene delivery system: a study of complexes composed of a peptide-based amphiphilic DNA compaction agent and phospholipid.
E. Murphy (2001)
10.1093/OXFORDJOURNALS.JBCHEM.A134989
Use of n-octyl-beta-D-thioglucoside, a new nonionic detergent, for solubilization and reconstitution of membrane proteins.
T. Tsuchiya (1984)
10.1074/JBC.M412374200
Actin Cytoskeleton as the Principal Determinant of Size-dependent DNA Mobility in Cytoplasm
Emmanuel Dauty (2005)
10.1038/nbt1122
Potent and persistent in vivo anti-HBV activity of chemically modified siRNAs
David V Morrissey (2005)
10.1016/J.YMTHE.2004.10.013
Thiocholesterol-based lipids for ordered assembly of bioresponsive gene carriers.
Z. Huang (2005)
10.1038/sj.gt.3302304
A sterically stabilized immunolipoplex for systemic administration of a therapeutic gene
W. Yu (2004)
10.1016/B978-012358465-6/50023-2
The Perplexing Delivery Mechanism of Lipoplexes
L. Barron (1999)
10.1016/S0076-6879(02)46048-X
Stabilized plasmid-lipid particles: a systemic gene therapy vector.
D. Fenske (2002)
10.1016/J.YMTHE.2005.11.002
Design of noninflammatory synthetic siRNA mediating potent gene silencing in vivo.
A. Judge (2006)
10.1517/17425247.2.2.255
Electroporation for targeted gene transfer
L. Heller (2005)
10.1038/nbt1081
Sequence-dependent stimulation of the mammalian innate immune response by synthetic siRNA
Adam D Judge (2005)
10.1021/BC000120W
Water-soluble lipopolymer for gene delivery.
Han So (2001)
10.1073/PNAS.84.22.7851
pH-sensitive immunoliposomes mediate target-cell-specific delivery and controlled expression of a foreign gene in mouse.
C. Y. Wang (1987)
10.3109/10611860009102218
Stabilized Plasmid–Lipid Particles: Pharmacokinetics and Plasmid Delivery to Distal Tumors following Intravenous Injection
M. Monck (2000)
Biological membranes : a practical approach
J. Findlay (1987)
10.1073/PNAS.93.14.7305
Formation of stable cationic lipid/DNA complexes for gene transfer.
H. E. Hofland (1996)
10.1016/S0005-2736(00)00343-6
Efficient encapsulation of antisense oligonucleotides in lipid vesicles using ionizable aminolipids: formation of novel small multilamellar vesicle structures.
S. Semple (2001)
10.1002/jgm.449
In vivo knockdown of gene expression in brain cancer with intravenous RNAi in adult rats
Y. Zhang (2003)
10.1021/BC000110V
Steric stabilization of fusogenic liposomes by a low-pH sensitive PEG--diortho ester--lipid conjugate.
X. Guo (2001)
10.1021/BC990101Q
Controlled template-assisted assembly of plasmid DNA into nanometric particles with high DNA concentration.
M. Ouyang (2000)
10.1517/14712598.3.6.911
Hydrodynamic delivery of DNA
B. Hodges (2003)
self-assembling nucleic acidYlipid nanoparticles suitable for targeted gene delivery
M. E. Hayes (2006)
10.1038/sj.cgt.7700311
Lipid-coated polyplexes for targeted gene delivery to ovarian carcinoma cells
E. Mastrobattista (2001)
10.1002/BIP.360360309
DNA condensation by cobalt hexaammine(III) in alcohol–water mixtures: Dielectric constant and other solvent effects
P. G. Arscott (1995)
10.1021/JA0522332
Monomolecular DNA nanoparticles for intravenous delivery of genes.
Chandrashekhar Chittimalla (2005)
10.3109/08982100009031102
Dimerizable Detergents as Gene Transfer Vectors
T. Blessing (2000)
TransferrinYlipoplexes with protamine-condensed DNA for serumresistant gene delivery
C. Tros de Ilarduya (2003)
10.1021/JA051977C
Direct fabrication and harvesting of monodisperse, shape-specific nanobiomaterials.
J. Rolland (2005)
10.1073/PNAS.93.21.11493
Mechanism of oligonucleotide release from cationic liposomes.
O. Zelphati (1996)
10.1021/BI9602019
Mechanism of DNA release from cationic liposome/DNA complexes used in cell transfection.
Y. Xu (1996)
10.1073/pnas.221450098
Brain-specific expression of an exogenous gene after i.v. administration
N. Shi (2001)
10.1073/PNAS.84.21.7413
Lipofection: a highly efficient, lipid-mediated DNA-transfection procedure.
P. Felgner (1987)
10.1016/S0005-2736(00)00264-9
Efficient encapsulation of DNA plasmids in small neutral liposomes induced by ethanol and calcium.
A. Bailey (2000)
10.1615/CRITREVTHERDRUGCARRIERSYST.V19.I3.10
Factors affecting drug and gene delivery: effects of interaction with blood components.
P. Opanasopit (2002)
10.1124/PR.58.1.8
Uptake Pathways and Subsequent Intracellular Trafficking in Nonviral Gene Delivery
I. Khalil (2006)
Water-soluble lipopolymer for gene
S. Han (2001)
10.1002/jgm.634
Low‐pH‐sensitive poly(ethylene glycol) (PEG)‐stabilized plasmid nanolipoparticles: effects of PEG chain length, lipid composition and assembly conditions on gene delivery
Weijun Li (2005)
10.1093/NAR/28.15.2986
Compaction of DNA in an anionic micelle environment followed by assembly into phosphatidylcholine liposomes.
E. Murphy (2000)
10.1146/ANNUREV.IMMUNOL.18.1.813
An address system in the vasculature of normal tissues and tumors.
E. Ruoslahti (2000)
10.1016/J.ADDR.2004.12.004
Implications of pharmacokinetic behavior of lipoplex for its inflammatory toxicity.
Jing-Shi Zhang (2005)
10.3109/08982100009029385
Receptor-Specific Delivery of Liposomes Via Folate-Peg-Chol
W. Guo (2000)
10.1016/S0006-3495(01)76202-9
Spontaneous entrapment of polynucleotides upon electrostatic interaction with ethanol-destabilized cationic liposomes.
N. Maurer (2001)
10.1021/BI952436A
Potentiation of cationic liposome-mediated gene delivery by polycations.
X. Gao (1996)
10.1081/LPR-120026389
Targeting of Lipid-Protamine-DNA (LPD) Lipopolyplexes Using RGD Motifs
P. Harvie (2003)
10.1089/10430349950017680
Lipoplex-mediated gene delivery to the lung occurs within 60 minutes of intravenous administration.
L. Barron (1999)
10.1016/S1525-0016(03)00064-9
Lipid-protamine-DNA-mediated antigen delivery to antigen-presenting cells results in enhanced anti-tumor immune responses.
John Dileo (2003)
Assembly of nucleic acidYlipid nanoparticles from aqueousYorganic monophases
M. E. Hayes (2006)
liposome com 447 Lipidic Nanoparticles for Nucleic Acid Delivery plexes for increased systemic delivery and gene expression
N. S. Templeton (1997)
10.1016/S0076-6879(03)73022-5
Transferrin-lipoplexes with protamine-condensed DNA for serum-resistant gene delivery.
C. Tros de Ilarduya (2003)
10.1016/0005-2736(73)90408-2
Single bilayer liposomes prepared without sonication.
S. Batzri (1973)
10.1038/NBT0797-647
Improved DNA: liposome complexes for increased systemic delivery and gene expression
Nancy Smyth Templeton (1997)
10.1007/s11095-004-1873-z
A Scalable, Extrusion-Free Method for Efficient Liposomal Encapsulation of Plasmid DNA
Lloyd B. Jeffs (2004)
10.1016/S0006-3495(92)81897-0
Sterically stabilized liposomes. Reduction in electrophoretic mobility but not electrostatic surface potential.
M. Woodle (1992)
Lipofection reagents prepared by a simple ethanol injection technique.
M. Campbell (1995)
10.1016/S0005-2736(99)00059-0
Stabilized plasmid-lipid particles: factors influencing plasmid entrapment and transfection properties.
K. Mok (1999)
10.1002/ANIE.200250446
Targeted gene delivery to cancer cells: directed assembly of nanometric DNA particles coated with folic acid.
G. Zuber (2003)
10.1016/S0168-3659(01)00324-8
Tumor-targeted p53-gene therapy enhances the efficacy of conventional chemo/radiotherapy.
L. Xu (2001)
10.1097/00000441-198910000-00013
In vivo transfection of murine lungs with a functioning prokaryotic gene using a liposome vehicle.
K. Brigham (1989)
10.1006/MTHE.2002.0633
Antisense gene therapy of brain cancer with an artificial virus gene delivery system.
Y. Zhang (2002)
10.1038/sj.gt.3301786
Robust and prolonged gene expression from injectable polymeric implants
R. Eliaz (2002)
Low - pHsensitive PEGstabilized plasmid Y lipid nanoparticles : preparation and characterization
J. S. Choi (2003)
10.1016/J.BBAMEM.2006.03.020
Assembly of nucleic acid-lipid nanoparticles from aqueous-organic monophases.
M. E. Hayes (2006)
10.1016/S0006-3495(97)78336-X
The mechanism of lamellar-to-inverted hexagonal phase transitions in phosphatidylethanolamine: implications for membrane fusion mechanisms.
D. P. Siegel (1997)
ferrin Y lipoplexes with protaminecondensed DNA for serumresistant gene delivery
M. A. Arangoa C. Tros de Ilarduya (2003)
10.1016/S0169-409X(02)00042-X
Folate-mediated delivery of macromolecular anticancer therapeutic agents.
Y. Lu (2002)
10.1016/0014-5793(95)01206-T
Aggregated DNA in ethanol solution
J. Piškur (1995)



This paper is referenced by
10.1021/ja903028f
Mesomorphic imidazolium salts: new vectors for efficient siRNA transfection.
W. Dobbs (2009)
10.1002/9783527630233.CH7
Chapter 7. Nanobiotechnology
Rudy J. Koopmans (2010)
10.1016/j.addr.2010.03.008
Engineering RNA for Targeted siRNA Delivery and Medical Application☆
P. Guo (2010)
Scavenger receptors as a target for nucleic acid delivery with peptide vectors
Carmen Tali (2017)
10.1016/j.biomaterials.2010.04.048
The effect of swelling and cationic character on gene transfection by pH-sensitive nanocarriers.
Jin-Oh You (2010)
10.1517/17425247.2010.517519
Using liposomes to target infection and inflammation induced by foreign body injuries or medical implants
Avi Schroeder (2010)
10.1039/c3ob27261c
Cationic lipophosphoramidates with two disulfide motifs: synthesis, behaviour in reductive media and gene transfection activity.
Aurore Fraix (2013)
10.1016/j.ejpb.2019.02.013
Anisamide‐targeted PEGylated gold nanoparticles designed to target prostate cancer mediate: Enhanced systemic exposure of siRNA, tumour growth suppression and a synergistic therapeutic response in combination with paclitaxel in mice
Xue Luan (2019)
10.4028/www.scientific.net/KEM.819.169
Niosomes Containing Spermine-Based Cationic Lipid with Different Linkers for siRNA Delivery
S. Pengnam (2019)
10.1016/j.addr.2009.01.005
Antioxidant enzyme gene transfer for ischemic diseases.
J. Wu (2009)
10.1016/j.biomaterials.2012.07.012
Anisamide-targeted cyclodextrin nanoparticles for siRNA delivery to prostate tumours in mice.
Jianfeng Guo (2012)
10.1016/j.biomaterials.2012.08.057
A biomimetic nanovector-mediated targeted cholesterol-conjugated siRNA delivery for tumor gene therapy.
Y. Ding (2012)
Overcoming Temozolomide Resistance in Glioblastoma Multiforme with MGMT-Targeting Spherical Nucleic Acids
Timothy L. Sita (2017)
10.1021/bm301155w
Parallel lipoplex folding pathways revealed using magnetic tweezers.
Zhiqiang Sun (2012)
Exploring the synthetic possibilities and siRNA delivery potential of small molecule carriers (SMoCs)
M. J. Gooding (2011)
10.1016/j.biomaterials.2009.05.036
Envelope-type lipid nanoparticles incorporating a short PEG-lipid conjugate for improved control of intracellular trafficking and transgene transcription.
Tomoya Masuda (2009)
10.1080/17425247.2020.1713746
Development of nano-carriers for Leishmania vaccine delivery
Anis Askarizadeh (2020)
10.1021/ja302930b
Self-assembly of stable monomolecular nucleic acid lipid particles with a size of 30 nm.
Sophia Rudorf (2012)
10.1049/iet-nbt.2015.0018
Short interfering RNA therapeutics: nanocarriers, prospects and limitations.
N. Mehrotra (2015)
10.1002/CMR.A.21361
Assessment of a Heuristic Model for Characterization of Magnetic Nanoparticles as Contrast Agent in MRI
Nazario Félix-González (2015)
10.1002/adhm.201400842
Engineering Stem Cells for Biomedical Applications.
P. T. Yin (2016)
10.2174/156802612798919141
siRNA delivery with lipid-based systems: promises and pitfalls.
C. Foged (2012)
10.1021/bc2002874
Synthesis and transfection efficiency of cationic oligopeptide lipids: role of linker.
V. Gopal (2011)
Développement de nanoparticules lipidiques pour la délivrance de courtes séquences d'ARN interférents
J. Bruniaux (2014)
10.1089/hum.2011.065
To be targeted: is the magic bullet concept a viable option for synthetic nucleic acid therapeutics?
M. Ogris (2011)
10.1517/14712598.8.7.911
Optimizing gene delivery vectors for the treatment of heart disease
S. J. Gray (2008)
10.1002/9780470923733.CH8
Application of RNA Interference to Viral Diseases
Ralph A Tripp (2010)
Fusion mixture for lipid-containing membrane of any modification of the lipid membrane of a cell membrane, a component of a cell membrane or a separate from the other cellular constituents cell membranes in vivo or in vitro
Agnes Csiszár (2013)
10.13023/ETD.2016.051
Thermodynamics and Kinetics of the Three-Way Junction of Phi29 Motor pRNA and its Assembly into Nanoparticles for Therapeutic Delivery to Prostate Cancer
Daniel W. Binzel (2016)
Enhanced Gene Delivery Mediated by Incorporation of Recombinant Fusion Proteins: Listeriolysin O and Peptides Derived from Protamine.
Na Hyung Kim (2011)
10.21236/ada640844
Development of Sorbents for Extraction and Stabilization of Nucleic Acids
Brandy J. White (2016)
10.1038/SJ.MT.6300326
Breaking the bonds: non-viral vectors become chemically dynamic.
J. Wolff (2008)
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