9780849319341

Polymeric Gene Delivery: Principles and Applications

by ;
  • ISBN13:

    9780849319341

  • ISBN10:

    084931934X

  • Format: Hardcover
  • Copyright: 2004-09-29
  • Publisher: CRC Press

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Summary

To treat disease or correct genetic disorders using gene therapy, the most suitable vehicle must be able to deliver genes to the appropriate tissues and cells in the body in a specific as well as safe and effective manner. While viruses are the most popular vehicles to date, their disadvantages include toxicity, limited size of genes they can carry, and limited scale of industrial production.Polymeric Gene Delivery: Principles and Applications is the first comprehensive book to specifically address polymeric gene delivery systems. Uniting the expertise of international academic and industrial scientists who are working in the area of polymeric vectors for gene delivery, it is written by prominent researchers directly involved in this field. The book is divided into five sections that deal with challenges and opportunities in gene delivery and the efficient delivery of genes into somatic cells using polymeric vectors. The authors discuss using biodegradable polymers, condensing and non-condensing polymeric systems, microspheres and nanospheres, and designing specialized delivery systems based on targeting strategies.Polymeric Gene Delivery: Principles and Applications accentuates the versatility of polymeric delivery systems, including the potential for biocompatibility, the ability to design their formulation and geometry for a specific purpose, and the ease of modification to the surface of polymeric carriers. This book is an up-to-date guide for researchers in the field and those interested in entering this dynamic field.

Table of Contents

Introduction
1(4)
Robert Langer
Part I Gene Delivery: Challenges and Opportunities
Tissue- and Cell-Specific Targeting for the Delivery of Genetic Information
5(24)
Randall J. Mrsny
Introduction
5(1)
Background Issues
6(2)
General Factors to Consider
8(5)
Viral Systems
8(2)
Nonviral Systems
10(2)
Immune Concerns
12(1)
Targeting Issues
12(1)
Targeting Cells and Tissues
13(1)
Topical Delivery Approaches
14(1)
Local Injection
15(1)
Vascular Targeting
16(1)
Cell-Based Targeting
17(2)
Conclusions
19(10)
References
20(9)
Biological Barriers to Gene Transfer
29(14)
Yasufumi Kaneda
Introduction
29(1)
Biological Barriers
30(7)
Step 1: Reaching Target Cells
30(1)
Recognition by Specific Target Tissues
30(1)
Avoidance of Nonspecific Uptake
31(1)
Resistance to Degradation in the Systemic Circulation
32(1)
Step 2: Crossing Over the Cell Membrane
32(1)
Step 3: Nuclear Targeting
33(1)
Step 4: Regulation of Gene Expression
34(1)
Stable Retention of a Transgene
35(1)
Regulation of Transcription
36(1)
Conclusion
37(6)
References
37(6)
Cellular Uptake and Trafficking
43(20)
Sujatha Dokka
Yon Rojanasakul
Introduction
44(1)
Viruses: Why Are They Such Efficient Delivery Systems?
44(1)
Cellular Barriers to DNA Delivery
45(7)
Stability in Extracellular Compartments
45(1)
Association of DNA with the Cell Membrane
45(1)
Cellular Internalization
46(1)
Proton Sponge
46(1)
pH-Sensitive Liposomes
46(1)
Fusogenic Peptides
47(1)
Cytosolic Transport of DNA
47(1)
Metabolic Instability of DNA in the Cytosol
47(1)
Nuclear Localization of Plasmid DNA
47(1)
Nucleocytoplasmic Transport
48(1)
Nuclear Import of DNA
49(1)
Attempts to Increase the Efficiency of Nuclear DNA Uptake
49(3)
Current Approaches for Enhancing Gene Delivery
52(3)
Viral Vectors
52(1)
Chemical/Synthetic Nonviral Vectors
53(1)
Liposomes
53(1)
DNA-Protein Conjugates
53(1)
Physical Methods of DNA Delivery
54(1)
Electroporation
54(1)
Direct DNA Injection and Microinjection
54(1)
Bombardment of DNA-Coated Particles
55(1)
Other Physical Approaches
55(1)
Conclusions
55(8)
References
56(7)
Pharmacokinetics of Polymer--Plasmid DNA Complex
63(16)
Makiya Nishikawa
Yoshinobu Takakura
Mitsuru Hashida
Introduction
63(1)
Pharmacokinetic Consideration of Macromolecules
64(3)
Basic Pharmacokinetic Properties of Naked Plasmid DNA
67(2)
Cellular Uptake Mechanism
67(1)
Tissue Distribution after Intravenous Injection
68(1)
Pharmacokinetics of Polymer--Plasmid DNA Complex
69(1)
Cellular Uptake
69(1)
Interaction with Biological Components
70(1)
Tissue Distribution
70(1)
Pharmacokinetics of Targeted Polymer--Plasmid DNA Complex
70(3)
Fundamentals of Targeted Delivery
70(1)
Sugar-Mediated Targeting
71(2)
Conclusion
73(6)
References
73(6)
Part II Condensing Polymeric Systems
Non-Degradable Polymers
Poly(L-Lysine) and Copolymers for Gene Delivery
79(18)
Minhyung Lee
Sung Wan Kim
Introduction
79(1)
Characteristics of PLL as a Gene Carrier
80(2)
PLL Conjugates
82(9)
PLL Conjugates for Low Cytotoxicity
82(1)
PLL--PEG Block Copolymer
82(1)
PEG-Grafted PLL
83(1)
PLL-Based Biodegradable Polymer
83(1)
PLL Conjugates for Receptor-Mediated Endocytosis
84(1)
PLL-Asialoorosomucoid
85(1)
PLL-Transferrin
85(1)
Galactose-PLL and Lactose-PLL
86(1)
PLL-Antibody
86(1)
PLL-Folate
87(1)
The Terplex System
87(1)
Artery-Wall-Binding Peptide PEG-Grafted-PLL
88(1)
PLL Conjugates for Efficient Intracellular Trafficking
89(1)
Histidylated PLL
89(1)
Histidylated Oligolysines
90(1)
Poly(histidine)-Grafted PLL
90(1)
Fusogenic Peptides
90(1)
PLL-NLS
90(1)
Conclusions
91(6)
References
91(6)
Gene Delivery Using Polyethylenimine and Copolymers
97(10)
Manfred Ogris
Introduction
97(1)
Polyethylenimine: Chemistry and Derivatives
98(1)
Coupling Strategies for PEI Conjugates
98(1)
Protein-PEI and Peptide-PEI Conjugates
98(1)
Biophysics of Nucleic Acid-PEI Interaction
98(1)
PEI Polyplexes for Nucleic Acid Delivery
99(1)
Ligand-PEI Conjugates for Gene Delivery
99(1)
Intracellular Aspects of PEI-Mediated Gene Delivery
100(1)
In Vivo Applications
101(1)
Local Delivery
101(1)
Systemic Application
102(1)
Therapeutic Gene Delivery with PEI
102(1)
Conclusions
103(4)
References
103(4)
Poly(2-(dimethylamino)ethyl methacrylate)-Based Polymers for the Delivery of Genes In Vitro and In Vivo
107(26)
F.J. Verbaan
D.J.A. Crommelin
W.E. Hennink
G. Storm
Introduction
107(1)
In Vitro Transfection Efficiency of pDMAEMA-Based Complexes: Critical Parameters
108(8)
Physicochemical Properties
108(2)
Design of pDMAEMA-Based Copolymers
110(2)
Cellular Interactions of pDMAEMA-Based Polyplexes
112(2)
Targeting of pDMAEMA-Based Polyplexes
114(2)
Pharmaceutical Aspects of pDMAEMA-Based Complexes
116(9)
Preparation of Highly Concentrated pDMAEMA-Polyplexes
116(2)
Structural Properties of Plasmid DNA
118(2)
Stability of pDMAEMA-Polyplexes
120(1)
Freeze-Drying and Freeze-Thawing of pDMAEMA-Polyplexes
120(2)
Long-Term Stability
122(3)
In Vivo Delivery of pDMAEMA-Based Complexes to Tumors
125(8)
Intraperitoneal Administration
125(2)
Intravenous Administration
127(3)
References
130(3)
Cationic Dendrimers as Gene Transfection Vectors: Dendri-Poly(amidoamines) and Dendri-Poly(propylenimines)
133(26)
Lori A. Kubasiak
Donald A. Tomalia
Introduction
134(3)
Dendritic Polymers: A Major New Architectural Class
134(1)
Dendrons and Dendrimers
135(2)
Poly(amidoamine) (PAMAM) Dendrimers
137(13)
Synthesis
137(1)
Structure
138(1)
Dendrimer Shape Changes: Nanoscale Container and Scaffolding Properties
138(1)
Nanoscale Container and Scaffolding Properties
139(1)
Nanoscale Dimensions and Shapes Mimicking Proteins
139(2)
Cationic (PAMAM) Dendrimer/DNA Interactions
141(1)
Cellular Entry
142(1)
In Vitro Transfection
142(1)
Plasmids
142(2)
Oligonucleotides
144(1)
Enhanced Dendrimer-Mediated Transfection
145(1)
In Vitro Toxicity
146(1)
Ex Vivo Gene Delivery
147(1)
In Vivo Gene Delivery
148(1)
Biodistribution
148(1)
In Vivo Toxicity
149(1)
Poly(propylenimine) (PPI) Dendrimers
150(2)
Synthesis
150(1)
Structure
150(1)
DNA--Dendrimer Interactions
151(1)
In Vitro Transfection
151(1)
In Vitro Toxicity
151(1)
Targeted Delivery
152(1)
Conclusion
152(7)
References
153(6)
Poly(ethylene glycol)-Conjugated Cationic Dendrimers
159(16)
Joon Sig Choi
Tae-il Kim
Jong-sang Park
Introduction
159(1)
Synthesis and Characterization of the Hybrid Block Copolymers
160(4)
MPEG-PLLD and PLLD-PEG-PLLD (Figures 10.2A and 10.2B)
160(1)
Liquid Phase Peptide Synthesis
160(1)
1H NMR and MALDI-TOF MS
161(1)
MPEG-PAMAM and PAMAM-PEG-PAMAM (Figures 10.2C and 10.2D)
161(1)
Synthesis of the Block Copolymers
161(2)
1H NMR and MALDI-TOF MS
163(1)
MPEG-PEI and PEG-PEI (Figures 10.2E and 10.2F)
163(1)
PEG Activation and Conjugation
163(1)
1H NMR
163(1)
Self-Assembly of Block Copolymers with Plasmid DNA
164(5)
Electrophoretic Mobility Shift Assay
164(1)
Atomic Force Microscopy (AFM)
164(3)
DNase I Protection Assay
167(1)
Zeta Potential Measurements
168(1)
Evaluation of Cytotoxicity In Vitro
169(1)
Application to In Vitro Transfection
169(3)
Conclusion
172(3)
Acknowledgments
172(1)
References
172(3)
Water Soluble Lipopolymers for Gene Delivery
175(12)
Ram I. Mahato
Sung Wan Kim
Introduction
175(1)
Basic Components Of Cationic Lipids
175(1)
Why Water-Soluble Lipopolymers?
176(1)
Synthesis and Characterization of Water-Soluble Lipopolymers
177(1)
Water-Soluble Lipopolymers for IL-12 Gene Delivery to Tumors
178(1)
Why Secondary Amines for Conjugation of Cholesterol to PEI?
179(2)
Characterization of T-Shaped Water-Soluble Lipopolymers
181(4)
Conclusion
185(2)
Acknowledgments
185(1)
References
185(2)
Cyclodextrin-Containing Polymers for Gene Delivery
187(26)
Suzie Hwang Pun
Mark E. Davis
Biodegradable Polymers
Introduction
187(1)
Linear, Cyclodextrin-Based Polymers
188(9)
Cyclodextrins
188(1)
Polymer Synthesis and Nanoparticle Formulation and Application
189(2)
Structure-Function Studies
191(4)
pH-Sensitive Polymers
195(2)
Polycations Containing Pendant Cyclodextrins
197(5)
Cyclodextrin-Dendrimer Conjugates
198(1)
Cyclodextrin-PEI Conjugates
199(3)
Formulations for In Vivo Applications
202(5)
Particle Modification by Adamantane Conjugates
202(1)
Particle Stabilization
202(1)
Surface Charge Tuning
203(2)
Targeting
205(2)
In Vivo Delivery
207(1)
Biocompatibility of Tf-Particles
208(1)
Systemic Plasmid Delivery to Tumors
208(1)
Summary
208(5)
References
209(4)
Gene Delivery Using Polyimidazoles and Related Polymers
213(14)
Sharon Wong
David Putnam
Introduction
213(1)
The ``Proton Sponge'' Hypothesis
214(1)
Histidine-Containing Peptides and Polymers
215(3)
Imidazole Acetic Acid-Containing Polymers
218(1)
Polyhistidine-Containing Polymers
219(2)
Vinyl Imidazole-Based Polymers or Polymethacrylates
221(1)
Future Trends
222(5)
Acknowledgments
223(1)
References
223(4)
Degradable Poly(β-amino ester)s for Gene Delivery
227(16)
David M. Lynn
Daniel G. Anderson
Akin Akinc
Robert Langer
Introduction
227(1)
Synthesis of Poly(β-amino ester)s
228(1)
Degradation and Cytotoxicity
229(2)
Self-Assembly with Plasmid DNA
231(2)
Parallel Synthesis and Screening of Poly(β-amino ester) Libraries
233(2)
Structure-Property Relationships and Biophysical Analysis
235(1)
Synthesis of Larger Polymer Libraries
236(1)
pH-Responsive Poly(β-amino ester) Microspheres
237(1)
Summary
238(5)
References
240(3)
Cationic Polyesters as Biodegradable Polymeric Gene Delivery Carriers
243(12)
Yong-Beom Lim
Yan Lee
Jong-Sang Park
Introduction
243(1)
Poly(4-hydroxy-l-proline ester) (PHP-ester)
244(2)
Polymer Synthesis
244(1)
Molecular Weight Distribution Determination and Degradation
244(1)
DNA Condensation and Gene Delivery
245(1)
Poly(α-[4-aminobutyl]-l-glycolic acid) (PAGA)
246(2)
Polymer Synthesis and PAGA Structure
246(1)
Degradation and DNA Condensation
247(1)
Cytotoxicity and Transfection Efficiency
247(1)
Hyperbranched poly(amino ester) (h-PAE)
248(2)
Structure and Synthesis
249(1)
Gene Delivery
249(1)
Network poly(amino ester) (n-PAE)
250(3)
Structure and Synthesis
250(1)
Stability of n-PAE-DNA Polyplex
250(1)
Gene Delivery
250(3)
Conclusion
253(2)
References
253(2)
Poly(amidoamine)s for Gene Delivery
255(24)
Paolo Ferruti
Jacopo Franchini
Introduction
256(1)
Synthetic Aspects
256(8)
Synthesis and Structure of PAAs
256(1)
Polymerization Kinetics
257(1)
Functionalization of PAAs
257(2)
Amphoteric PAAs
259(1)
Labeling of PAAs
260(1)
Radioactive Labeling
260(1)
PAAs with Amino Pendants and Fluorescent Labeling
260(3)
PAA Macromonomers
263(1)
Crosslinked PAAs
263(1)
PAA-Related Polymers
263(1)
Physico-Chemical Properties of PAAs
264(4)
Molecular Weight, Solubility, and Solution Properties
264(1)
Crystallinity
264(1)
Thermal Stability
264(1)
Degradation
264(3)
Acid--Base Properties of PAAs
267(1)
Biomedical Applications of PAAs
268(11)
Applications Mostly Related to Insoluble PAA Derivatives
268(1)
Neutralization of the Anticoagulant Activity of Heparin, Heparin-Absorbing Resins, and Heparinizable Materials
268(1)
Soft PAA Hydrogels as Scaffolds for Tissue Engineering
269(1)
PAAs in Polymer Therapeutics
270(1)
PAAs as Bioactive and Biocompatible Polymers
270(1)
Toxicity of PAAs
271(1)
Body Distribution of PAAs
271(1)
PAAs as Polymer Carriers for Anticancer Drugs
272(1)
pH-Mediated Membrane Activity, DNA Complexing, and Transfection Ability of PAAs
273(1)
PAAs as Promoters of Intracellular Trafficking of Proteins
274(1)
PAA-Grafted Proteins and Future Trends
275(1)
References
276(3)
Cationic Polysaccharides for Gene Delivery
279(22)
Tony Azzam
Abraham J. Domb
Introduction
279(2)
Cationic Lipids
280(1)
Cationic Polymers
280(1)
Chitosan
281(3)
Structure, Chemistry, Physico-Chemical Properties, and Application
281(1)
Chitosan Derivatives
282(1)
Deoxycholic Acid-Modified Chitosan
282(1)
Quaternized Chitosan
283(1)
Chitosan Modified with Hydrophilic Polymers
283(1)
Galactosylated Chitosan
283(1)
Chitosan Conjugated with Transferrin, KNOB, and Endosomolytic Proteins
284(1)
DEAE-Dextran Mediated Transfecting Agent
284(1)
Polysaccharide-Oligoamine Conjugates for Gene Delivery
285(7)
Synthesis of Polysaccharide-Oligoamine Conjugates
286(1)
Condensation Studies of Polysaccharide-Oligoamine Conjugates with DNA
287(2)
In Vitro Transfection of Polysaccharide-Oligoamine Conjugates
289(2)
In Vivo Transfection with Dextran-Spermine Vector as a Gene Carrier
291(1)
Intramuscular Injection of pSV-LacZ Gene Complex
291(1)
Intravenous Injection of pSV-LacZ Gene Complex
292(1)
Conclusions
292(9)
References
294(7)
Gene Delivery Using Chitosan and Chitosan Derivatives
301(12)
Gerrit Borchard
Maytal Bivas-Benita
Properties of Chitosan and Chitosan Derivatives
301(1)
Interactions of Chitosan with Cell Membranes
302(1)
Preparation of Chitosan-Based Gene Delivery Systems
303(1)
Polyplexes
303(1)
Nano- and Microparticles
304(1)
Transfection Studies
304(1)
DNA Vaccination
305(8)
References
307(6)
Part III Non-Condensing Polymeric Systems
Pluronic® Block Copolymers for Nonviral Gene Delivery
313(16)
Alexander V. Kabanov
Srikanth Sriadibhatla
Valery Yu. Alakhov
Introduction
313(3)
Structure and Synthesis of Pluronic® Block Copolymers
316(1)
Self-Assembly of Pluronic® Block Copolymers
317(1)
Pluronic® Block Copolymers as Functional Excipients
317(1)
Pluronics® Enhance Gene Transfer with Polyplexes
318(1)
Use of Pluronics® as Structural Components in Polyplexes
319(1)
Pluronics® Enhance Expression in Stably Transfected Cells
320(1)
Effect of Pluronics® on Expression of Naked DNA In Vivo
320(2)
Toxicological Considerations
322(1)
Conclusions
323(6)
Acknowledgments
323(1)
References
323(6)
Use of Poly(N-vinyl pyrrolidone) with Noncondensed Plasmid DNA Formulations for Gene Therapy and Vaccines
329(16)
Michael Nicolaou
Polly Chang
Mark J. Newman
Introduction
329(1)
Development of pDNA Vaccines
330(1)
Delivery Optimization for pDNA Vaccines and Immune-System-Based Therapeutics
331(1)
Development of Poly(N-vinyl pyrrolidone) (PVP) as a pDNA Delivery Agent
332(2)
Use of PVP in T-lymphocyte Epitope-Based pDNA Vaccines
334(2)
Safety Profile of PVP Used as a Drug and Gene Therapy Formulation Excipient
336(1)
Safety Testing of PVP Formulated EP HIV-1090 pDNA Vaccine for HIV Therapy and Prophylaxis
337(3)
Summary and Conclusions
340(5)
Acknowledgments
340(1)
References
340(5)
Use of HPMA Copolymers in Gene Delivery
345(12)
David Oupicky
Introduction
345(1)
Random Copolymers of HPMA with Cationic Comonomers
346(2)
Block and Graft Copolymers of HPMA with Polycations
348(1)
Semitelechelic HPMA Copolymers for Surface Modification of Polyplexes
349(1)
Multivalent HPMA Copolymers for Surface Modification of Polyplexes and Adenovirus
350(2)
Surface Modification of Polyplexes
350(2)
Surface Modification of Adenovirus
352(1)
Conclusions
352(5)
References
353(4)
Part IV Polymeric Nanospheres and Microspheres
Polymeric Nanospheres
Biodegradable Nanoparticles as a Gene Expression Vector
357(10)
Swayam Prabha
Wenxue Ma
Vinod Labhasetwar
Introduction
357(1)
Nanoparticles versus Microparticles: The Effect of Size
358(1)
DNA Incorporation and the Effect of Formulation Conditions
358(1)
Mechanism of Nanoparticle-Mediated Sustained Gene Transfection
359(2)
Factors Affecting Nanoparticle-Mediated Gene Transfection
361(3)
Intracellular Uptake and Endo-Lysosomal Escape
361(1)
DNA Release
362(2)
Applications of Nanoparticle-Mediated Gene Transfection
364(1)
Summary
364(3)
Acknowledgments
365(1)
References
365(2)
Nanoparticles Made of Poly(lactic acid) and Poly(ethylene oxide) as Carriers of Plasmid DNA
367(20)
Noemi Csaba
Celso Perez
Alejandro Sanchez
Maria Jose Alonso
Introduction
367(1)
Poly(ethylene oxide) and Derivatives: Physicochemical Properties and Biological Behavior
368(2)
Poly(ethylene oxide) and Derivatives: New Approaches for Improving Drug Delivery from Nanoparticulate Carriers
370(1)
Design of Nanoparticles Made of Polyesters, PEO, and Derivatives
371(3)
Poloxamer/Poloxamine-Coated PLGA Nanoparticles
371(1)
Poloxamer/Poloxamine-Containing PLGA Nanoparticles
371(1)
Design of Nanoparticles from Copolymers of PLA and PEG
372(2)
Nanoencapsulation and Delivery of Oligonucleotides and Plasmid DNA from Nanoparticles
374(6)
Nanoencapsulation of Oligonucleotides and Plasmid DNA into PLA-PEG Nanoparticles
374(2)
Nanoencapsulation of Plasmid DNA into PLGA Nanoparticles Containing Poloxamers and Poloxamines
376(1)
Controlled Release of Plasmid DNA from Nanoparticles of PLA-PLGA and PEO and Derivatives
377(1)
Stability of Plasmid DNA Released from Nanoparticles of PLA-PLGA and PEO and Derivatives
378(2)
In Vivo Administration of Plasmid-Containing PLA-PEG Nanoparticles
380(1)
Concluding Remarks
381(6)
Acknowledgments
381(1)
References
381(6)
Poly(alkylcyanoacrylate) Nanoparticles for Nucleic Acid Delivery
387(12)
Elias Fattal
Patrick Couvreur
Introduction
387(1)
Antisense Strategy and the Need for Particulate Formulations
388(3)
Poly(alkylcyanocrylate) Nanoparticles: Their Potential for ODN Delivery
391(1)
Delivery of ODN by Polyalkycyanoacrylate Nanospheres
391(3)
Association of ODN to Nanospheres
391(1)
In Vitro Stability of ODNs Adsorbed onto Nanospheres
392(1)
Cell Interactions with ODN-Loaded Nanospheres
393(1)
In Vitro Pharmacological Activity of Oligonucleotide-Loaded Nanospheres
394(1)
In Vivo Studies with Oligonucleotide Nanospheres
394(1)
Delivery of Oligonucleotides by Poly(cyanoacrylate) Nanocapsules
394(2)
Conclusion
396(3)
References
396(3)
Layer-by-Layer Nanoengineering with Polyelectrolytes for Delivery of Bioactive Materials
399(18)
Dinesh Shenoy
Alexei Antipov
Gleb Sukhorukov
Introduction
399(2)
LbL Engineering Principles in Design of Colloids with Tailored Architecture
401(6)
Extension of LbL Sequential Adsorption Technique from Flat Surfaces to Colloidal Particles
401(1)
Core-Shell Compositions for Drug Delivery Applications
402(2)
Fabrication of Hollow Capsules by the LbL Approach
404(1)
Scope and Methods for Loading of Bioactive Materials into Preformed Capsules
405(1)
Utilization of Donnan Equilibrium
405(1)
Capturing of Particulate Material, Polymeric Catalyst, or Enzyme
405(1)
Preparing the Double-Walled Capsules
406(1)
Extension of LbL Concept for Gene Delivery
407(2)
DNA Loading by Direct Surface Modification of the Compacted DNA Complex
407(1)
DNA Encapsulation as Shell Component
408(1)
Scope for Active Loading of DNA into Preformed Polyelectrolyte Hollow Capsules
409(1)
Conclusions and Outlook
409(8)
Acknowledgments
410(1)
References
410(7)
Ex Vivo and In Vivo Adenovirus-Mediated Gene Delivery into Refractory Cells via Nanoparticle Hydrogel Formulation
417(12)
Ales Prokop
Gianluca Carlesso
Jeffrey M. Davidson
Polymer-Driven Adenoviral Gene Delivery
417(2)
Gene Transfer into Dendritic Cells
419(3)
Gene Transfer into Pancreatic Cells
422(1)
In Vivo Gene Delivery
422(7)
References
423(6)
Protein Nanoparticles for Gene Delivery
429(22)
Goldie Kaul
Mansoor Amiji
Introduction
430(2)
Description of Gene Therapy
430(1)
Viral versus Non-Viral Vectors
430(1)
Nanoparticle Systems for Gene Delivery
431(1)
Protein Nanoparticles
431(1)
Collagen
432(4)
Collagen as a Biomaterial
432(1)
Collagen Nanoparticles for Gene Delivery
433(1)
Preparation of Nanoparticles
433(1)
Other Collagen-Based Gene Delivery Systems
434(1)
Collagen Films and Matrices
434(1)
Collagen Shields for Gene Delivery
434(1)
Collagen Sponges
435(1)
Collagen Hydrogels
435(1)
Collagen Pellets
435(1)
Gelatin
436(2)
Gelatin as a Biomaterial
436(1)
Gelatin Nanoparticles for Gene Delivery
436(1)
Gelatin-DNA Coacervates
436(1)
PEG-Modified Gelatin Nanoparticles
437(1)
Albumin
438(2)
Albumin as a Biomaterial
438(1)
Albumin Nanoparticles for Gene Delivery
439(1)
Preparation of Albumin Nanoparticles
439(1)
Additional Examples of Protein Nanoparticles
440(3)
Lipoproteins
440(1)
Epidermal Growth Factor
441(1)
Terplex System
442(1)
Conclusion
443(8)
References
443(8)
Polymeric Microspheres
Gene Delivery Using Poly(lactide-co-glycolide) Microspheres
451(16)
Mary Lynne Hedley
Introduction
451(1)
Advantages of PLG Microparticles for Gene Delivery
452(2)
Protection of DNA
452(1)
Biocompatibility and Biodegradability
452(1)
Cell Targeting
453(1)
Generation of DNA/PLG Microparticle Formulations
454(3)
Encapsulation of Gene Expression Vectors
454(1)
Double Emulsion Technique
454(1)
Spray Drying Technique
455(1)
DNA-Coated PLG Particles
456(1)
Particle Internalization and Gene Expression
457(2)
Immune Activation
459(4)
Immune Responses Elicited By PLG-Plasmid Formulations
460(1)
Delivery Systems to Elicit Immune Responses with PLG-Plasmid Particle Formulations
460(1)
Mucosal Delivery
460(1)
Parenteral Delivery
461(1)
Adjuvants for Microparticle-Based Immunization
461(1)
Nonhuman Primates and Clinical Studies
462(1)
Summary
463(4)
References
464(3)
Polyanhydride Microspheres for Gene Delivery
467(8)
Yong S. Jong
Camilla A. Santos
Edith Mathiowitz
Introduction
467(1)
Polyanhydrides
467(1)
Bioadhesion with Poly(fumaric-co-sebacic anhydride)
468(1)
Phase Inversion Nanoencapsulation
468(1)
Improving Particle Uptake for Gene Delivery
469(1)
Properties of Plasmid DNA
470(1)
In Vitro Transfection with P(FA:SA) PIN
470(1)
In Vivo Gene Delivery with P(FA:SA) PIN
471(1)
Summary
471(4)
References
472(3)
Microspheres Formulated from Native Hyaluronan for Applications in Gene Therapy
475(14)
Yang H. Yun
Weiliam Chen
Introduction
475(1)
Microspheres as Gene Delivery Vectors
475(1)
Microspheres Prepared from Hyaluronan
476(1)
Preparation and Characterization of Hyaluronan Microspheres
476(5)
Cross-linking of HA Microspheres
477(1)
HA Microspheres and Size Distribution
478(1)
Release Kinetics of DNA from HA-DNA Microspheres
478(2)
Gel Electrophoresis Analysis of DNA Released from HA-DNA Microspheres
480(1)
In Vitro Cellular Transfection
481(2)
In Vivo Transfection of Rat Hind Limb Muscles
483(1)
Conclusions
484(5)
Acknowledgments
485(1)
References
485(4)
Part V Specialized Delivery Systems
Genetically Engineered Protein-Based Polymers: Potential in Gene Delivery
489(20)
Zaki Megeed
Hamidreza Ghandehari
Introduction
489(1)
Genetically Engineered Protein-Based Polymers
490(2)
Synthesis and Characterization of Genetically Engineered Polymers
490(2)
Biomedical Applications of Genetically Engineered Polymers
492(2)
Biocompatibility and Biodegradation
492(1)
Applications of Genetically Engineered Polymers
493(1)
Gene Delivery Applications for Genetically Engineered Polymers
494(8)
Controlled Gene Delivery from Silk-Elastinlike Hydrogels
495(4)
Controlled Release of Adenovirus from Silk-Elastinlike Hydrogels
499(2)
Gene Delivery from Derivatized Recombinant Polymers
501(1)
Future Potential of Genetically Engineered Polymers in Gene Delivery
502(1)
Conclusion and Future Direction
503(6)
Acknowledgments
503(1)
References
503(6)
Glycopolymer Tools for Studying Targeted Nonviral Gene Delivery
509(14)
Kevin G. Rice
Ji-Seon Kim
Dijie Liu
Introduction
509(1)
Gene Targeting to C-Type Lectins
510(2)
Glycosylated Polylysine, PEI, and Peptides
512(4)
Glycosylated Lipoplexes
516(1)
Conclusions
516(7)
Acknowledgment
517(1)
References
517(6)
Targeted Gene Delivery via the Folate Receptor
523(14)
Shih-Jiuan Chiu
Robert J. Lee
Introduction
523(1)
Gene Therapy for Cancer
524(1)
The Folate Receptor as a Tumor Marker
524(1)
Targeted Drug Delivery via the FR
524(2)
Targeted Gene Delivery via the FR
526(6)
FR-Targeted Adenoviral Vectors
526(1)
FR-Targeted Polyplexes
527(1)
Folate-Poly(L-lysine) (PLL)
527(1)
Folate-Polyethylenimine (PEI)
528(1)
Folate-pDMAEMA
529(1)
FR-Targeted Lipoplexes
529(1)
FR-Targeted Lipopolyplexes
530(1)
LPDI
530(1)
LPDII
531(1)
Conclusions
532(5)
References
532(5)
Transferrin Receptor-Targeted Gene Delivery Systems
537(20)
Ralf Kircheis
Ernst Wagner
Introduction
538(1)
Intrinsic Properties of Polycations
538(4)
DNA Condensation and Electrostatic Interaction with Cell Membrane
538(1)
Endosomal Release
539(1)
Polylysine
539(1)
Polyethylenimine
540(1)
Migration through Cytoplasm, Nuclear Entry, and Complex Disassembly
541(1)
Receptor-Mediated Transfection
542(2)
Transferrin-PEI-DNA Complexes
543(1)
Specific Ligand-Receptor-Mediated Uptake versus Nonspecific Electrostatic Uptake
543(1)
Application In Vivo
544(6)
Systemic Application
546(1)
Systemically Delivered Polylysine-DNA Complexes
546(1)
Systemically Delivered PEI-DNA Complexes
547(1)
Shielding of Polycation-DNA Complexes
547(1)
PEG Shielding of Polyplexes
547(1)
Ligand Density
547(1)
Poly-N-(2-hydroxypropyl)methacrylamide (pHPMA) Shielding
548(1)
Poloxamer Shielding
548(1)
Targeted Gene Expression with Shielded Polyplexes
549(1)
Future Directions
550(1)
Conclusions
550(7)
References
551(6)
Gene Delivery to the Lungs
557(16)
Berma M. Kinsey
Charles L. Densmore
Frank M. Orson
Introduction
557(1)
Gene Delivery to the Lung
557(1)
Polycations
558(1)
Intravenous Gene Delivery
558(5)
Introduction
558(1)
Intravenous Gene Delivery by Polyethylenimine
559(1)
Ternary Complexes with Polyethylenimine
560(1)
Polyethylenimine Conjugated to Other Molecules
560(2)
Gene Delivery by Macroaggregated Albumin--PEI Particles
562(1)
Cancer Gene Therapy by Intravenous Gene Delivery
562(1)
Delivery of Genes to the Airways
563(5)
Introduction
563(1)
Fluid Instillation of Polyplexes into the Airway
563(2)
Fluid Phase Airway Gene Delivery with Polyethylenimine-Polyethylene Glycol
565(1)
Fluid Phase Airway Gene Delivery by Ternary PEI-TAT Peptide-DNA Complexes
565(1)
Cancer Gene Therapy by Fluid Phase Airway Gene Delivery
565(1)
Aerosol Gene Delivery to the Lungs by Polyethylenimine
565(2)
Cancer Gene Therapy by Aerosol Gene Delivery
567(1)
Genetic Immunization by Aerosol Gene Delivery
567(1)
Concluding Remarks
568(5)
References
568(5)
Cutaneous Gene Delivery
573(16)
James C. Birchall
Introduction
573(1)
Disease Targets for Cutaneous Gene Therapy
573(3)
The Skin as a Barrier to Gene Delivery
576(1)
Physical Methods for Enhancing Gene Delivery to Skin
577(4)
Formulation Strategies for Enhancing Gene Delivery to Skin
581(1)
Polymer-Mediated Gene Delivery to Skin
582(1)
Targeting Gene Expression in the Skin
583(1)
Summary
584(5)
Acknowledgments
584(1)
References
584(5)
Enhancement of Wound Repair by Sustained Gene Transfer via Hyaluronan Matrices
589(22)
Angela P. Kim
Daniel M. Checkla
Don Wen
Philip Dehazya
Weiliam Chen
Introduction
589(2)
Materials and Methods
591(3)
Plasmid DNA Gene Constructs
591(1)
DNA--HA Matrix Formulations
591(1)
Matrix Morphology Assessment
591(1)
Assessment of DNA Release Kinetics
592(1)
Gel Electrophoresis Analyses
592(1)
Transfections Using Release Samples with DNA Encoding β-Galactosidase
592(1)
Transfections Using Release Samples with DNA Encoding PDGF-ββ-FLAG
593(1)
In Vivo Wound Healing Studies
593(1)
Results and Discussion
594(13)
Matrix Morphology
594(1)
Assessment of DNA Release Kinetics
595(2)
Gel Electrophoresis Analyses
597(3)
β-Galactosidase Release Sample Transfections
600(3)
PDGF-ββ-FLAG Release Sample Transfections
603(4)
In Vivo Wound Healing
607(1)
Conclusions
607(4)
Acknowledgments
609(1)
References
609(2)
Gene Delivery from Tissue Engineering Matrices
611(14)
Zain Bengali
Christopher B. Rives
Lonnie D. Shea
Introduction
611(1)
Matrix-Based Release
612(4)
Nonviral Delivery
613(1)
Hydrogels
613(1)
Synthetic Polymers
614(1)
Viral Delivery
615(1)
Solid Phase Delivery
616(2)
Viral Vector Delivery
617(1)
Nonviral DNA Delivery
618(1)
Future Directions and Challenges
618(7)
References
619(6)
Gene Therapy Stents for In-Stent Restenosis
625(34)
Ilia Fishbein
Itay Perlstein
Robert Levy
Delivering Gene Therapy with a Stent
626(1)
In-Stent Restenosis: The Scope of the Problem
626(3)
ISR Mechanisms: Platelet Aggregation, Thrombosis, and Thrombus Organization
628(1)
ISR Mechanisms: SMC Proliferation, Migration, and Matrix Synthesis
628(1)
Treatment of In-Stent Restenosis
628(1)
Stent Composition and Design
629(1)
Coated Stents
629(1)
Drug-Eluting Stents
630(1)
Molecular Therapeutics for In-Stent Restenosis: Critical Issues
630(3)
Plasmid-Mediated Gene Transfer
631(1)
Replication-Defective Adenovirus-Mediated Gene Transfer
631(1)
Adeno-Associated-Virus-Mediated Gene Transfer
632(1)
Retrovirus- and Lentivirus-Mediated Gene Transfer
632(1)
Antisense-Based Strategies
632(1)
Cell-Mediated Gene Therapy
633(1)
Gene Therapy Molecular Targets in Restenosis
633(4)
Antiproliferative Gene Therapy
633(3)
Antimigration Gene Therapy
636(1)
Antithrombotic Gene Therapy
636(1)
Gene Therapy for Re-Endothelization
636(1)
Nitric-Oxide-Related Genes: Pleiotropic Therapeutic Effects
637(1)
Gene Delivery Stents
637(22)
Plasmid Delivery from a Stent: Bulk Immobilization
638(1)
Denatured Collagen-Coated Stents Demonstrate Facilitated Plasmid Transfection
639(3)
Other DNA Delivery Stents
642(1)
Adenovirus-Mediated Gene Transfer from Stents: The Tethering Strategy
643(1)
Stent-Tethered Ad Using Antiknob Antibodies
644(1)
Transduction Using Ad-Delivery Stents in the Pig Coronary Model
644(15)
Gene Delivery Using BioMEMS
659(12)
Krishnendu Roy
Introduction
659(1)
Drug and Gene Delivery Systems
660(1)
Barriers in Gene Delivery
661(1)
BioMEMS and Drug Delivery: Current Research
661(6)
Microfabricated Microneedles
661(2)
Integrated Delivery Systems
663(2)
Microchip-Based Delivery Systems
665(1)
Delivery from Immunoisolated Genetically Modified Cells
666(1)
Microfabricated Particles for Oral Drug Delivery
667(1)
Biocompatibility of MEMS Materials
667(1)
Potential of MEMS in Gene Delivery Applications
668(3)
References
668(3)
Index 671

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Reviews for Polymeric Gene Delivery: Principles and Applications (9780849319341)