Recent developments in cationic lipid-mediated gene delivery and gene therapy

Expert Opinion on Therapeutic Patents

Volumn 11, Issue 11, 2001, Pages 1729-1752

  Ilies, M A ^a   Balaban, A T ^a  

^a TEXAS A AND M UNIVERSITY AT GALVESTON   (United States)

Author keywords

Cationic lipid; Gene delivery; Gene therapy; Lipoplexes; Plasmid DNA; Transfection

Indexed keywords

1,2 BIS(OLEOYLOXY) 3 (TRIMETHYLAMMONIUM)PROPANE; 1,4 BIS [N' BUTYL [4 N,N DIMETHYL 2,3 BIS(TETRADECYCLOXY) 1 PROPANAMINIUM]]UREYLBUTANE; 2,3 DIOLEOYLOXY N [2 (SPERMINECARBOXAMIDO)ETHYL] N,N DIMETHYL 1 PROPANAMINIUM TRIFLUOROACETATE; 2,3 DIOLEOYLOXYPROPYL 1 TRIMETHYLAMMONIUM BROMIDE; 3 B[N (N,N,N TRIETHYLAMINOPROPANE) CARBAMOYL] CHOLESTEROL; ALKANE DERIVATIVE; AMIDINE; BROMINE DERIVATIVE; CHOLESTEROL DERIVATIVE; DIOLEOYLPHOSPHATIDYLETHANOLAMINE; GUANIDINE; GUANOSINE PHOSPHATE; LIPOFECTAMINE; LIPOFECTIN; LIPOPOLYAMINE; LIPOSOME; N (2 HYDROXYETHYL) N,N DIMETHYL 2,3 BIS(TETRADECYLOXY) 1 PROPANAMINIUM BROMIDE; N (3 AMINOPROPYL) N,N DIMETHYL 2,3, BIS(DODECYCLOXY) 1 PROPANAMINIUM BROMIDE; POLYAMINE DERIVATIVE; QUARTERNARY AMMONIUM SALT LIPID; QUATERNARY AMMONIUM DERIVATIVE; TRANSFECTAM; UNCLASSIFIED DRUG; VIRUS VECTOR;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ARTERY DISEASE; CANCER; CONTROLLED STUDY; CORONARY ARTERY DISEASE; CYSTIC FIBROSIS; CYTOTOXICITY; DEVELOPMENT; DRUG BIOAVAILABILITY; DRUG DESIGN; DRUG FORMULATION; DRUG MECHANISM; DRUG STRUCTURE; DRUG SYNTHESIS; GENE EXPRESSION; GENE TARGETING; GENE THERAPY; GENE TRANSFER; GENETIC TRANSFECTION; HUMAN; HUMAN CELL; IMMUNOGENICITY; INDUSTRY; MOUSE; NEUROLOGIC DISEASE; NONHUMAN; PHYSICAL CHEMISTRY; PLASMID; PROPHYLAXIS; REVIEW; SIDE EFFECT;

EID: 0034758926     PISSN: 13543776     EISSN: None     Source Type: Journal    
DOI: 10.1517/13543776.11.11.1729     Document Type: Review

References (257)

1. Initial sequencing and analysis of the human genome
2. The sequence of the human genome
3. Therapeutical Mechanisms and Strategies: Templeton NS
4. Gene Therapy: Delivering the Medicines of the 21st Century
5. Application of lipids and plasmid design for gene delivery to mammalian cells
6. Gene therapy: The first decade
7. Synthetic gene-transfer vectors
8. Liposome and polylysine mediated gene transfer
9. The Cambrian period of nonviral gene delivery
10. DNA-induced endocytosis upon local microinjection to giant unilamellar cationic vesicles
11. How realistic is cutaneous gene therapy?
12. Electroporation: A general phenomenon for manipulating cells and tissues
13. Depth-targeted efficient gene delivery and expression in the skin by pulsed electric fields: An approach to gene therapy of skin aging and other diseases
14. In vivo gene delivery by electroporation
15. In vivo efficacy and safety of skin electroporation
16. Electroporation-enhanced gene delivery in mammary tumors
17. Controlled gene gun delivery and expression of DNA within the cornea
18. Uptake of exogenous DNA via the skin
19. In vivo gene gun-mediated DNA delivery into rodent brain tissue
20. Viral vectors for gene therapy
21. Viral gene delivery
22. Viral vectors for gene transfer: A review of their use in the treatment of human diseases
23. Viral vectors for gene therapy: The art of turning infectious agents into vehicles of therapeutics
24. Virus-sized self-assembling lamellar complexes between plasmid DNA and cationic micelles promote gene transfer
25. Recent advances in liposome technologies and their applications for systemic gene delivery
26. Key issues in non-viral gene delivery
27. Biological barriers to cellular delivery of lipid-based DNA carriers
28. Formation of stable cationic lipid/DNA complexes for gene transfer
29. Physical stabilisation of DNA-based therapeutics
30. Nomenclature for synthetic gene delivery systems
31. Biophysical characterization of cationic lipid: DNA complexes
32. Cationic lipid binding to DNA: Characterization of complex formation
33. Evidence for three-dimensional interlayer correlations in cationic lipid-DNA complexes as observed by cryo-electron microscopy
34. Structure, stability, and thermodynamics of lamellar DNA-lipid complexes
35. Lipid-DNA complex formation: Reorganization and rupture of lipid vesicles in the presence of DNA as observed by cryoelectron microscopy
36. Lipofection: A highly efficient, lipid-mediated DNA-transfection procedure
37. Cationic liposome-mediated transfection
38. Cationic liposome-mediated RNA transfection
39. DNA strongly binds to micelles and vesicles containing lipopolyamines or lipointercalants
40. Efficient gene transfer into mammalian primary endocrine cells with lipopolyamine-coated DNA
41. Use of a quaternary ammonium detergent in liposome mediated DNA transfection of mouse L-cells
42. Interactions of mammalian cells with lipid dispersions containing novel metabolizable cationic amphiphiles
43. A novel cationic liposome reagent for efficient transfection of mammalian cells
44. Receptor-mediated in vitro gene transformation by a soluble DNA carrier system
45. Receptor-mediated gene delivery and expression in vivo
46. Transferrin-polycation conjugates as carriers for DNA uptake into cells
47. Lipophylic polylysines mediate efficient DNA transfection in mammalian cells
48. Positively charged lipids: Structure, methods of synthesis and applications
49. Lipidic vector systems for gene transfer
50. Cationic lipids used in gene transfer
51. Nonviral strategies for gene therapy
52. Cationic liposomes for gene therapy
53. Liposome-based gene therapy
54. Gene transfer with lipospermines and polyethylenimines
55. Interpolyelectrolyte and block ionomer complexes for gene delivery: Physico-chemical aspects
56. Taking polycation gene delivery systems from in vitro to in vivo
57. Structure and function of lipid-DNA complexes for gene delivery
58. Delivering DNA with polymer matrices: Application in tissue engineering and gene therapy
59. Poly(amidoamine) (PAMAM) dendrimers: From biomimicry to drug delivery and biomedical applications
60. Nonviral vectors in the new millennium: Delivery barriers in gene transfer
61. Cationic lipid-DNA complexes in gene delivery: From biophysics to biological applications
62. Novel cationic lipids for gene delivery and gene therapy
63. Ligand-targeted receptor-mediated vectors for gene delivery
64. Synthesis of cationic lipids from 1,2,4-butanetriol
65. Synthesis of diether-linked cationic lipids for gene delivery
66. Structural basis of DOTMA for its high intravenous transfection activity in mouse
67. Synthesis and characterization of aromatic ring-based cationic lipids for gene delivery in vitro and in vivo
68. Cationic liposome-mediated gene transfer
69. Targeted gene transfer into hepatoma cells with lipopolyamine-condensed DNA particles presenting galactose ligands: A stage toward artificial viruses
70. In vivo gene delivery to the liver using novel galactosylated cationic liposomes
71. Synthesis of targetable cationic amphiphiles
72. Synthesis of novel cationic lipids with oxyethylene spacers at the linkages between hydrocarbon chains and pseudoglyceryl backbone
73. Enhanced gene delivery and mechanism studies with a novel series of cationic lipid formulations
74. A novel cationic lipid greatly enhances plasmid DNA delivery and expression in mouse lung
75. Cationic phosphonolipids as non viral vectors for DNA transfection
76. Systemic administration of cationic phosphonolipids/DNA complexes and the relationship between formulation and lung transfection efficiency
77. Cationic phosphonolipids as nonviral vectors: In vitro and in vivo applications
78. New biocompatible cationic amphiphiles derivative from glycine betaine: A novel family of efficient nonviral gene transfer agents
79. Cation substitution in cationic phosphonolipids: A new concept to improve transfection activity and decrease cellular toxicity
80. Aerosolization of cationic lipid-DNA complexes: Lipoplex characterization and optimization of aerosol delivery conditions
81. Synthesis and stability study of the new pentammonio lipid pcTG90, a gene transfer agent
82. Effect of liposome-encapsulated clodronate pretreatment on synthetic vector-mediated gene expression in mice
83. New cationic liposomes for gene transfer into mammalian cells with high efficiency and low toxicity
84. Synthesis and evaluation of vitamin D-based cationic lipids for gene delivery in vitro
85. Synthesis and biological properties of new glycosidic cationic lipids for DNA delivery
86. Synthesis, activity, and structure-activity relationship studies of novel cationic lipids for DNA transfer
87. Introduction of a disulfide bond into a cationic lipid enhances transgene expression of plasmid DNA
88. Cationic liposomes containing disulfide bonds in delivery of plasmid DNA
89. Use of dithiodiglycolic acid as a tether for cationic lipids decreases the cytotoxicity and increases transgene expression of plasmid DNA in vitro
90. Reduction-sensitive lipopolyamines as a novel nonviral gene delivery system for modulated release of DNA with improved transgene expression
91. Reducible cationic lipids for gene transfer
92. Lipofectamine reagent: A new higher efficiency polycationic liposome transfection reagent
93. Systemic delivery of secreted protein by grafts of epidermal keratinocytes: Prospects for keratinocyte gene therapy
94. Introduction of cyclic guanidines into cationic lipids for non-viral gene delivery
95. A novel series of amphiphilic imidazolinium compounds for in vitro and in vivo gene delivery
96. Synthesis of pyridinium amphiphiles used for transfection and some characteristics of amphiphile/DNA complex formation
97. Formation of de novo centromeres and construction of first-generation human artificial microchromosomes
98. Structures of lipid-DNA complexes: Supramolecular assembly and gene delivery
99. New structures in complex formation between DNA and cationic liposomes visualized by freeze-fracture electron microscopy
100. Sticky ions in biological systems
101. The counterion influence on cationic lipid-mediated transfection of plasmid DNA
102. Charge density-dependent strength of hydration and biological structure
103. Cationic lipid-mediated gene delivery to murine lung: Correlation of lipid hydration with in vivo transfection activity
104. Electrostatic parameters of cationic liposomes commonly used for gene delivery as determined by 4-heptadecyl-7-hydroxycoumarin
105. Two-dimensional condensation of DNA molecules on cationic lipid membranes
106. Thermodinamics of the binding of a cationic lipid to DNA
107. The role of counterions in the interaction of bifunctional surface active compounds with model membranes
108. Influence of counterions on the interaction of pyridinium salts with model membranes
109. Thermotropic phase behavior of cationic lipid-DNA complexes compared to binary lipid mixtures
110. Hydration of lipoplexes commonly used in gene delivery: Follow-up by laurdan fluorescence changes and quantification by differential scanning calorimetry
111. Physico-chemical characterisation and transfection efficiency of lipid-based gene delivery complexes
112. Structural characteristics of supramolecular assemblies formed by guanidinium-cholesterol reagents for gene transfection
113. Effect of counterions on the influence of dodecyltrimethylammonium halides on thermotropic phase behaviour of phosphatidylcholine bilayers
114. Hydration of alkylammonium salt micelles - Influence of bromide and chloride counterions
115. Characteristics and biodistribution of cationic liposomes and their DNA complexes
116. Interactions between cholesterol and lipids in bilayer membranes. Role of lipid headgroup and hydrocarbon chain-backbone linkage
117. DOTAP cationic liposomes prefer relaxed over supercoiled plasmids
118. Effect of DNA/liposome mixing ratio on the physicochemical characteristics, cellular uptake and intracellular trafficking of plasmid DNA/cationic liposome complexes and subsequent gene expression
119. Biophysical and structural properties of DNA.diC(14)-amidine complexes. Influence of the DNA/lipid ratio
120. Interactions between DNA and synthetic cationic liposomes
121. Atomic force microscopy imaging of DNA-cationic liposome complexes optimised for gene transfection into neuronal cells
122. Physico-chemical analysis of cationic liposome-DNA complexes (lipoplexes) with respect to in vitro and in vivo gene delivery efficiency
123. Isothermal titration calorimetric analysis of the interaction between cationic lipids and plasmid DNA
124. The role of dioleoyl phosphatidylethanolamine in cationic liposome mediated gene transfer
125. The effects of cholesterol inclusion on the vesicular membranes of cationic lipids
126. Electrostatic and structural properties of complexes involving plasmid DNA and cationic lipids commonly used for gene delivery
127. Phases and phase transitions of the phosphatidylcholines
128. Lamellarity of cationic liposomes and mode of preparation of lipoplexes affect transfection efficiency
129. Serum as a modulator of lipoplex-mediated gene transfection: Dependence of amphiphile, cell type and complex stability
130. Distribution of DNA in cationic liposome complexes probed by Raman microscopy
131. Interplay in lipoplexes between type of pDNA promoter and lipid composition determines transfection efficiency of human growth hormone in NIH3T3 cells in culture
132. Chiral DNA packaging in DNA-cationic liposome assemblies
133. Interaction of oligonucleotides with cationic lipids: The relationship between electrostatics, hydration and state of aggregation
134. Gene transfer in vitro and in vivo by cationic lipids is not significantly affected by levels of supercoiling of a reporter plasmid
135. Probing DNA-cationic lipid interactions with the fluorophore trimethylammonium diphenyl-hexatriene (TMADPH)
136. Electrostatically mediated interactions between cationic lipid-DNA particles and an anionic surface
137. Efficient in vivo gene delivery by the negatively charged complexes of cationic liposomes and plasmid DNA
138. Analysis of self-assembled cationic lipid-DNA gene carrier complexes using flow field-flow fractionation and light scattering
139. Efficient encapsulation of antisense oligonucleotides in lipid vesicles using ionizable aminolipids: Formation of novel small multilamellar vesicle structures
140. Polymorphism of DNA/multi-cationic Lipid complexes driven by temperature and salts
141. Structure of inserum transfecting DNA-cationic lipid complexes
142. Solubilisation of DNA-cationic lipid complexes in hydrophobic solvents. A single molecule visualization by fluorescence microscopy
143. Mode of formation and structural features of DNA-cationic liposome complexes used for transfection
144. Structural and fusogenic properties of cationic liposomes in the presence of plasmid DNA
145. Formation of novel hydrophobic complexes between cationic lipids and plasmid DNA
146. Direct evidence for counterion release upon cationic lipid-DNA condensation
147. Differential scanning calorimetry - A tool to assess physical and chemical alterations in liposomes
148. Interaction of surfactants with DNA. Role of hydrophobicity and surface charge on intercalation and DNA melting
149. Characterization of DNA-lipid complexes commonly used for gene delivery
150. Structure of DNA-cationic liposome complexes: DNA intercalation in multilamellar membranes in distinct interhelical packing regimes
151. The structure of DNA-liposome complexes
152. An inverted hexagonal phase of cationic liposome-DNA complexes related to DNA release and delivery
153. Structure and interfacial aspects of self-assembled cationic lipid-DNA gene carrier complexes
154. DNA condensation in two dimensions
155. Atomic force microscopy of cationic liposomes
156. The structure of DNA complexes with cationic liposomes-cylindrical or flat bilayers?
157. Complexes between cationic liposomes and DNA visualized by cryo-TEM
158. Phosphatidylethanolamine liposomes: Drug delivery, gene transfer and immunodiagnostic applications
159. Structure and structure-function studies of lipid/plasmid DNA complexes
160. DNA transfection mediated by cationic liposomes containing lipopolylysine: Characterization and mechanism of action
161. Fusion of cationic liposomes with mammalian cells occurs after endocytosis
162. Comparison of different commercially available cationic lipid-based transfection kits
163. Overcoming the inhibitory effect of serum on lipofection by increasing the charge ratio of cationic liposome to DNA
164. Nonviral gene therapy: Promises and challenges
165. Kinetic analysis of the initial steps involved in lipoplex-cell interactions: Effect of various factors that influence transfection activity
166. Biodistribution and transgene expression with nonviral cationic vector/DNA complexes in the lungs
167. Development of gene drug delivery systems based on pharmacokinetic studies
168. Mechanism of lipoplex gene delivery in mouse lung: Binding and internalization of fluorescent lipid and DNA components
169. Gene therapy for peripheral arterial disease
170. Cationic lipid-mediated gene transfer to the growing murine and human airway
171. Efficacy of cationic liposome-mediated gene transfer to mesangial cells in vitro and in vivo
172. Targeting transgene expression for cystic fibrosis gene therapy
173. Induction of angiogenesis by cationic lipid-mediated VEGF165 gene transfer in the rabbit ischemic hindlimb model
174. Highly efficient cell-mediated gene transfer using non-viral vectors and FuGene6: In vitro and in vivo studies
175. Cationic liposome-mediated gene delivery to the liver and to hepatocellular carcinomas in mice
176. Gene therapy for lung disease
177. Studies of direct intratumoral gene transfer using cationic lipid-complexed plasmid DNA
178. Percutaneous in vivo gene transfer to the peripheral lungs using plasmid-liposome complexes
179. Nonviral gene delivery to the lateral ventricles in rat brain: Initial evidence for widespread distribution and expression in the central nervous system
180. Gene therapy in the CNS
181. Efficient lipid-mediated gene transfer to articular chondrocytes
182. Cutaneous gene transfer and therapy: The present and the future
183. Efficiency and toxicity of liposome-mediated gene transfer to corneal endothelial cells
184. Transferrin receptor-mediated gene transfer to the corneal endothelium
185. Aerosol gene delivery in vivo
186. Prospects for gene therapy for cystic fibrosis
187. A clinical inflammatory syndrome attributable to aerosolized lipid-DNA administration in cystic fibrosis
188. Cationic lipid-mediated gene transfer: Effect of serum on cellular uptake and intracellular fate of lipopolyamine/DNA complexes
189. Interaction between DNA-cationic liposome complexes and erythrocytes is an important factor in systemic gene transfer via the intravenous route in mice: The role of the neutral helper lipid
190. Dynamic changes in the characteristics of cationic lipidic vectors after exposure to mouse serum: Implications for intravenous lipofection
191. Pharmacokinetics and targeted delivery of proteins and genes
192. Membrane fusion with cationic liposomes: Effects of target membrane lipid composition
193. On the mechanism whereby cationic lipids promote intracellular delivery of polynucleic acids
194. Lipoplex size is a major determinant of in vitro lipofection efficiency
195. Evidence for the role of proteoglycans in cation-mediated gene transfer
196. An electron microscopy study into the mechanism of gene transfer with lipopolyamines
197. Proteoglycans mediate cationic liposome-DNA complex-based gene delivery in vitro and in vivo
198. Cellular and molecular barriers to gene transfer by a cationic lipid
199. Intracellular distribution and mechanism of delivery of oligonucleotides mediated by cationic lipids
200. Endocytosis and intracellular processing accompanying transfection mediated by cationic liposomes
201. The role of endosome destabilizing activity in the gene transfer process mediated by cationic lipids
202. Cell delivery, intracellular trafficking and expression of an integrin-mediated gene transfer vector in tracheal epithelial cells
203. Loss of binding and entry of liposome-DNA complexes decreases transfection efficiency in differentiated airway epithelial cells
204. Human serum albumin enhances DNA transfection by lipoplexes and confers resistance to inhibition by serum
205. Mannose receptor-mediated gene transfer into macrophages using novel mannosylated cationic liposomes
206. Parameters influencing the introduction of plasmid DNA into cells by the use of synthetic amphiphiles as a carrier system
207. Cationic lipids destabilize lysosomal membrane in vitro
208. Mechanism of DNA release from cationic liposome/DNA complexes used in cell transfection
209. Mechanism of oligonucleotide release from cationic liposomes
210. Structural and functional analysis of cationic transfection lipids: The hydrophobic domain
211. Tunable pH-sensitive liposomes composed of mixtures of cationic and anionic lipids
212. Liposomes: Conquering the nuclear barrier
213. Nuclear targeting of DNA
214. High efficiency transformation by direct microinjection of DNA into cultured mammalian cells
215. Metabolic instability of plasmid DNA in the cytosol: A potential barrier to gene transfer
216. Cationic lipids enhance cellular uptake and activity of phosphorothioate antisense oligonucleotides
217. Rapid nuclear accumulation of injected oligodeoxyribonucleotides
218. The nuclear pore complex: From molecular architecture to functional dinamics
219. Molecular mobility and nucleocytoplasmic flux in hepatoma cells
220. Transport pathways of macromolecules between the nucleus and the cytoplasm
221. Gene delivery: A single nuclear localisation signal peptide is sufficient to carry DNA to the cell nucleus
222. Increased expression of DNA cointroduced with nuclear protein in adult rat liver
223. Nuclear localisation signal peptides enhance cationic liposome-mediated gene therapy
224. Signalling and transport through the nuclear membrane
225. A peptide nucleic acid-nuclear localization signal fusion that mediates nuclear transport of DNA
226. Nuclear targeting peptide scaffolds for lipofection of nondividing mammalian cells
227. Complexes of non-cationic liposomes and histone H1 mediate efficient transfection of DNA without encapsulation
228. 2+ ions
229. Synthetic DNA-compacting peptides derived from human sequence enhance cationic lipid-mediated gene transfer in vitro and in vivo
230. Import of plasmid DNA into the nucleus is sequence specific
231. Cationic lipid-mediated transfection of cells in culture requires mitotic activity
232. Cell cycle dependence of gene transfer by lipoplex, polyplex and recombinant adenovirus
233. Human Gene Therapy Protocols
234. Cationic lipid-based delivery system for systemic cancer gene therapy
235. Optimization of cationic lipid/DNA complexes for systemic gene transfer to tumor lesions
236. Developing non-viral DNA delivery systems for cancer and infectious disease
237. Cystic fibrosis clinical trials
238. Prospects for gene therapy in lung disease
239. Effects of human tissue plasminogen gene transfer on allograft coronary atherosclerosis
240. Optimisation of non-viral gene transfer to human primary retinal pigment epithelial cells
241. Liposome application: Problems and prospects
242. Bacterial DNA is implicated in the inflammatory response to delivery of DNA/DOTAP to mouse lungs
243. Contribution of plasmid DNA to hepatotoxicity after systemic administration of lipoplexes
244. Toxicity of cationic liposome-DNA complex in lung isografts
245. Sequential injection of cationic liposome and plasmid DNA effectively transfects the lung with minimal inflammatory toxicity
246. Gene delivery systems: Bridging the gap between recombinant viruses and artificial vectors
247. Monomolecular collapse of plasmid DNA into stable virus-like particles
248. Potentiation of cationic liposome-mediated gene delivery by polycations
249. Protamine sulfate enhances lipid-mediated gene transfer
250. In vivo gene transfer via intravenous administration of cationic lipid-protamine-DNA (LPD) complexes
251. Characterization of cationic lipid-protamine-DNA (LPD) complexes for intravenous gene delivery
252. Aspartoacylase gene transfer to the mammalian central nervous system with therapeutic implications for Canavan disease
253. Enhanced cationic liposome-mediated transfection using the DNA-binding peptide mu (mu) from the adenovirus core