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E-raamat: Guide to Human Gene Therapy [World Scientific e-raamat]

Edited by (Univ Of Florida, Usa), Edited by (Univ Of Florida, Usa)
  • Formaat: 416 pages, Illustrations (some col.)
  • Ilmumisaeg: 31-Jan-2010
  • Kirjastus: World Scientific Publishing Co Pte Ltd
  • ISBN-13: 9789814280914
Teised raamatud teemal:
  • World Scientific e-raamat
  • Hind: 155,23 €*
  • * hind, mis tagab piiramatu üheaegsete kasutajate arvuga ligipääsu piiramatuks ajaks
Guide to Human Gene TherapySuurem pilt
  • Formaat: 416 pages, Illustrations (some col.)
  • Ilmumisaeg: 31-Jan-2010
  • Kirjastus: World Scientific Publishing Co Pte Ltd
  • ISBN-13: 9789814280914
Teised raamatud teemal:
World Scientific e-raamatudRohkem infot World Scientific e-raamatute kohta
Raamatu kodulehekülg: http://www.worldscientific.com/worldscibooks/10.1142/7406#t=toc
Ever since the birth of molecular biology, the tantalizing possibility of treating disease at its genetic roots has become increasingly feasible Gene therapy---though still in its infancy---remains one of the hottest areas of research in medicine Its approach utilizes a gene transfer vehicle ("vector") to deliver therapeutic DNA or RNA to cells of the body in order to rectify the defect that is causing the disease Successful therapies have been reported in humans in recent years such as cures in boys with severe immune deficiencies Moreover, gene therapy strategies are being adapted in numerous biomedical laboratories to obtain novel treatments for a variety of diseases and to study basic biological aspects of disease Correction of disease in animal studies, is steadily gaining ground, highlighting the immense potential of gene therapy in the medical profession.

This book will cover topics that are at the forefront of biomedical research such as RNA interference, viral and non-viral gene transfer systems, treatment of hematological diseases and disorders of the central nervous system Leading experts on the respective vector or disease will contribute the individual chapters and explain cutting-edge technologies It also gives a broad overview of the most important gene transfer vectors and most extensively studied target diseases This comprehensive guide is therefore a must-read for anyone in the biotechnology, biomedical or medical industries seeking to further their knowledge in the area of human gene therapy.

Preface v
Contributors xxiii
1 Non-Viral Gene Therapy
1(20)
Sean M. Sullivan
1 Introduction
1(2)
2 Plasmid DNA
3(3)
2.1 Plasmid DNA Manufacture
5(1)
3 Plasmid DNA Gene Transfer Methods
6(11)
3.1 Plasmid DNA or "Naked DNA" as a Gene Delivery System
6(2)
3.1.1 Electroporation of Naked DNA
8(1)
3.1.2 Sonoporation of Naked DNA
9(1)
3.2 Plasmid DNA Formulations
9(1)
3.2.1 Cationic Lipids
9(1)
3.2.1.1 In vitro transfection
10(1)
3.2.1.2 Systemic in vivo gene transfer
11(1)
3.2.1.3 Local administration of cationic lipid/pDNA transfection complexes
12(2)
3.3 Polymer
14(1)
3.3.1 Cationic Polymers
14(1)
3.3.2 Neutral Polymer
15(2)
Conclusions
17(1)
References
17(4)
2 Adenoviral Vectors
21(16)
Stuart A. Nicklin
Andrew H. Baker
1 Introduction
21(1)
2 Adenoviral Capsid Structure
22(1)
3 Adenoviral Cell Entry
23(1)
4 Production of Adenoviral Vectors
24(2)
5 Production of Targeted Adenoviral Vectors
26(2)
6 Gene Therapy Applications
28(2)
7 Immune Responses to Ad Vectors
30(2)
8 Safety and Regulatory Issues
32(1)
9 Conclusions
33(1)
References
33(4)
3 Retroviral Vectors and Integration Analysis
37(16)
Cynthia C. Bartholomae
Romy Kirsten
Hanno Glimm
Manfred Schmidt
Christofvon Kalle
1 Introduction
37(1)
2 Design, Production and Mechanism of Transduction
38(3)
3 In vivo Application
41(1)
4 Side Effects in Retroviral Gene Therapy
42(5)
4.1 Distribution of Retroviral Integration Sites in the Cellular Genome
42(3)
4.2 Side Effects in Clinical and Preclinical Gene Therapy Studies
45(2)
5 New Strategies for Vector Biosafety in Gene Therapy
47(2)
References
49(4)
4 Lentiviral Vectors
53(16)
Janka Matrai
Marinee K. L. Chuah
Thierry Vanden Driessche
1 Basic Viral Biology
53(3)
2 Vector Design and Production
56(3)
2.1 Vector Development
56(2)
2.2 Vector Production
58(1)
3 Gene Transfer Concepts and Potential Applications
59(3)
3.1 Target Cells and Diseases
59(1)
3.2 Pseudotyping
59(1)
3.3 Cell Type Specific Targeting
60(1)
3.4 Integration-Defective Lentiviral Vectors
60(2)
4 Immune Consequences
62(1)
5 Safety Issues
63(1)
6 Conclusions and Perspectives
64(1)
References
64(5)
5 Herpes Simplex Virus Vectors
69(18)
William F. Goins
David M. Krisky
James B. Wechuck
Darren Wolfe
Justus B. Cohen
Joseph C. Glorioso
1 Introduction
69(5)
2 HSV Biology in the Design of Replication Defective Vectors
74(3)
3 HSV Vector Design Technology
77(2)
4 Gene Transfer/Therapy Applications
79(1)
5 Immunology
80(1)
6 Safety and Regulatory Issues
81(1)
7 Summary
81(1)
References
82(5)
6 Adeno-Associated Viral (AAV) Vectors
87(16)
Nicholas Muzyczka
1 Introduction
87(1)
2 Biology of AAV
88(5)
3 Vector Technology
93(3)
4 Vector Characteristics In Vivo
96(2)
5 Next Generation Vectors
98(1)
6 Conclusions and Outlook
99(1)
References
99(4)
7 Regulatory RNA in Gene Therapy
103(20)
Alfred S. Lewin
1 Introduction
103(3)
2 Delivery of Therapeutic RNAs
106(3)
3 Ribozymes
109(2)
4 RNAi for Gene Therapy
111(3)
5 Gene Therapy Using miRNA
114(1)
6 Aptamers, Decoys and Bi-Functional RNAs
115(1)
7 Modification of Cis-Acting Regulatory RNA Sequences
116(3)
8 Conclusions
119(1)
References
120(3)
8 DNA Integrating Vectors (Transposon, Integrase)
123(16)
Lauren E. Woodard
Michele P. Calos
1 Basic Vector Biology
123(5)
1.1 Transposon Systems
124(2)
1.2 Integrase Systems
126(2)
2 Vector Design and Production
128(2)
2.1 Design of Transposon Systems
128(1)
2.2 Design of Integrase Systems
128(1)
2.3 Production of Plasmid DNA
129(1)
3 Gene Transfer Protocols and Potential Applications
130(2)
3.1 Hepatocyte Transfection via Hydrodynamic Injection
130(1)
3.2 Lipophilic Complexes to Transfect Endothelial Cells and Glioblastoma
131(1)
3.3 Direct DNA Injection and Electroporation to Target Muscle, Retina, and Joints
131(1)
3.4 Integration into Cultured Cells for Ex vivo Gene Therapy
131(1)
4 Immunology
132(1)
5 Safety and Regulatory Issues
133(2)
5.1 Integration Profiles and Associated Hazards
133(1)
5.2 Efforts to Enhance Integration Specificity
133(1)
5.3 Effects on Tumor Latency in Mouse Models of Cancer
134(1)
References
135(4)
9 Homologous Recombination and Targeted Gene Modification for Gene Therapy
139(24)
Matthew Porteus
1 Introduction
139(1)
2 Problems with Using Gene Targeting by Homologous Recombination
140(1)
3 Homologous Recombination in Embryonic Stem Cells
141(3)
4 Homologous Recombination using Adeno-Associated Virus
144(1)
5 Site-Specific Modification of the Genome using Double-Strand Breaks
144(1)
6 Double-Strand Break Repair
144(2)
7 Double-Strand Break Induced Homologous Recombination
146(1)
8 Re-design of Homing Endonucleases to Recognize New Target Sites
146(1)
9 Development of Zinc Finger Nucleases
147(1)
10 Using Zinc Finger Nucleases to Stimulate Gene Targeting
147(2)
11 Using Zinc Finger Nucleases to Site-Specifically Modify Genes by Mutagenic Non-Homologous End-Joining
149(2)
12 Strategies of Zinc Finger Nuclease Design
151(2)
13 Aspects of Zinc Finger Binding Sites and Structure of Zinc Finger Nucleases
153(1)
14 Zinc Finger Nuclease Toxicity: Measuring and Minimizing
154(2)
15 The Challenge of Delivery
156(1)
16 Future Directions and Promise of Homologous Recombination as a Gene Correction Approach to Gene Therapy
157(1)
References
157(6)
10 Gene Switches for Pre-Clinical Studies in Gene Therapy
163(18)
Caroline Le Guiner
Knut Stieger
Alice Toromanoff
Fabienne Rolling
Philippe Moullier
Oumeya Adjali
1 Introduction
163(2)
2 Rapamycin-Dependent Regulatable System
165(3)
2.1 Molecular Mechanisms Involved in Transgene Regulation
165(1)
2.2 Pharmacology of Rapamycin
166(1)
2.3 Translation Development of the Rapamycin Dependent Regulation System
166(2)
3 Tetracycline-Dependent Regulatable Systems
168(7)
3.1 Molecular Mechanisms Involved in Transgene Regulation
168(3)
3.2 Pharmacology of Doxycycline (Dox)
171(1)
3.3 Translational Development of Tet-dependant Regulation Systems
171(4)
4 Other Regulatable Systems
175(2)
5 General Conclusions
177(1)
References
177(4)
11 Gene Therapy for Central Nervous System Disorders
181(16)
Deborah Young
Patricia A. Lawlor
1 Introduction
181(1)
2 Gene Therapy for Parkinson's Disease
182(4)
3 Gene Therapy for Temporal Lobe Epilepsy
186(1)
4 Huntington's Disease Gene Therapy
187(2)
5 Amyotrophic Lateral Sclerosis (ALS)
189(1)
6 Gene Therapy for Canavan Disease
190(1)
7 Gene Therapy for Alzheimer's Disease
191(2)
8 Conclusions and Outlook
193(1)
References
194(3)
12 Gene Therapy of Hemoglobinopathies
197(16)
Angela E. Rivers
Arun Srivastava
1 Introduction
198(1)
2 β-Thalassemia
198(1)
3 Sickle Cell Disease
199(1)
4 Gene Therapy
200(8)
4.1 Oncoretroviral Vector-Mediated Globin Gene Transfer
202(1)
4.2 Lentiviral Vector-Mediated Globin Gene Transfer
203(1)
4.3 Adeno-Associaied Viral Vector-Mediated Globin Gene Transfer
204(4)
References
208(5)
13 Gene Therapy for Primary Immunodeficiencies
213(20)
Aisha Sauer
Barbara Cassani
Alessandro Aiuti
1 Introduction
214(1)
2 Adenosine Deaminase (ADA)-deficient SCID
215(3)
3 X-linked Severe Combined Immunodeficiency (SCID XI)
218(2)
4 Gene Therapy for Other SCIDs
220(4)
4.1 V(D)J Recombination Defects
220(2)
4.2 Purine Nucleoside Phosphorylase (PNP) Deficiency
222(1)
4.3 Janus Kinase 3 (Jak3) Deficiency
222(1)
4.4 IL-7R Deficiency
223(1)
4.5 Zeta Associated 70 kDa Phosphoprotein (ZAP-70) Deficiency
223(1)
5 Wiskott-Aldrich-Syndrome (WAS)
224(1)
6 Chronic Granulomatous Disease
225(2)
7 Conclusions and Outlook
227(1)
References
228(5)
14 Gene Therapy for Hemophilia
233(18)
David Markusic
Babak Moghimi
Roland Herzog
1 Introduction
233(2)
2 Limitations of Hemophilia Treatment With Coagulation Factor Concentrates or Recombinant Coagulation Factors
235(1)
3 Gene Transfer for Correction of Hemophilia
236(2)
3.1 Ex Vivo Gene Transfer of F. VIII and F. IX
236(1)
3.2 In Vivo Gene Transfer of F. VIII and F. IX
237(1)
4 AAV is a Preferred Gene Therapy Vector for In Vivo Gene Transfer to Correct of Hemophilia
238(1)
5 Immunological Considerations for Efficient F. IX Gene Transfer
239(3)
6 Advancements from Small and Large Animal Models of Hemophilia
242(2)
6.1 Murine Hemophilia Models
242(1)
6.2 Canine Hemophilia Models
242(2)
7 Gene Therapy Trials for Hemophilia Past, Present, and Future
244(1)
8 Conclusions
245(1)
References
246(5)
15 Gene Therapy for Obesity and Diabetes
251(10)
Sergei Zolotukhin
Clive H. Wasserfall
1 Introduction
251(1)
2 Understanding Obesity: Why We Get Fat
252(1)
2.1 Genetic Factors: Human Obesity Gene Map
252(1)
2.2 Environmental Factors: The Big Two and Other Causal Contributors
253(1)
3 General Strategies in Gene Therapy for Obesity
253(2)
4 Gene Delivery Vehicles
255(1)
5 Gene Targets for Obesity
255(5)
5.1 Leptin
255(1)
5.2 Neurocytokines
256(1)
5.3 AMP-Activated Protein Kinase (AMPK)
256(1)
5.4 Adiponectin
257(1)
5.5 Wnt-10b
257(1)
5.6 Obesity Gene Menu a la Carte
258(1)
5.7 Obesity and Diabetes
259(1)
References
260(1)
16 Gene Therapy for Duchenne Muscular Dystrophy
261(18)
Takashi Okada
Shin'ichi Takeda
1 Introduction
261(1)
1.1 Background of Duchenne Muscular Dystrophy
261(1)
2 Gene-replacement Strategies using Virus Vectors
262(4)
2.1 Choice of Vector
262(2)
2.2 Modification of the Dystrophin Gene and Promoter
264(2)
2.3 Use of Surrogate Genes
266(1)
3 AAV-Mediated Transduction of Animal Models
266(4)
3.1 Vector Production
266(1)
3.2 Animal Models for the Gene Transduction Study
267(1)
3.3 Immunological Issues of rAAV
268(1)
3.4 Intravascular Vector Administration by Limb Perfusion
269(1)
3.5 Global Muscle Therapies
269(1)
4 Safety and Potential Impact of Clinical Trials
270(1)
5 Development of Alternative Strategies
271(2)
5.1 Design of Read-through Drugs
271(1)
5.2 Modification of mRNA Splicing
272(1)
5.3 Ex Vivo Gene Therapy
272(1)
6 Future Perspectives
273(1)
6.1 Pharmacological Intervention
273(1)
6.2 Capsid Modification
273(1)
7 Conclusions and Outlook
273(1)
References
274(5)
17 Cancer Gene Therapy
279(16)
Kirsten A.K. Weigel-Van Aken
1 Introduction
280(1)
2 Targeting the Tumor Cell
280(6)
2.1 DNA Electroporation
280(1)
2.2 Non-Oncolytic Viral Vectors
281(1)
2.2.1 Retrovirus
281(1)
2.2.2 Lentivirus
282(1)
2.3 Oncolytic Viruses
282(1)
2.3.1 Herpesvirus
283(1)
2.3.2 Adenovirus
283(1)
2.3.3 Poxvirus
284(1)
2.3.4 Measles virus
285(1)
2.3.5 Vesicular stomatitis virus
285(1)
3 Targeting the Immune System
286(3)
3.1 Cancer Vaccines
287(1)
3.1.1 Vaccinia virus
287(1)
3.1.2 Lentivirus
287(1)
3.1.3 Adenovirus
288(1)
3.1.4 Parvoviruses
288(1)
3.2 Mesenchymal Stem Cells (MSC) as Delivery Vehicles
288(1)
3.3 Adoptive T Cell Transfer
288(1)
4 Targeting the Tumor Microenvironment
289(1)
5 Challenges and Risks of Cancer Gene Therapy
289(1)
6 Novel Strategies
290(1)
6.1 Prime/Boost Regimens
290(1)
6.2 Immune Cells as Carriers for Viruses
290(1)
7 Conclusions
291(1)
References
291(4)
18 Gene Therapy for Autoimmune Disorders
295(16)
Daniel F. Gaddy
Melanie A. Ruffner
Paul D. Robbins
1 Introduction
295(1)
2 Rheumatoid Arthritis
296(5)
2.1 Background
296(1)
2.2 Existing Therapies
296(1)
2.3 Target Tissues and Routes of Delivery
297(1)
2.3.1 Local RA Gene Therapy
297(1)
2.3.2 Systemic RA Gene Therapy
297(1)
2.4 Immunomodulation
298(1)
2.5 Overview of Preclinical Gene Therapy Studies
299(2)
2.6 Overview of Clinical Gene Therapy Studies
301(1)
3 Type I Diabetes Mellitus
301(6)
3.1 Background
301(1)
3.2 Existing Therapies
302(1)
3.3 Target Tissues and Routes of Delivery
303(1)
3.4 Immunomodulation
303(2)
3.5 Overview of Preclinical Gene Therapy Studies
305(1)
3.6 Overview of Clinical Gene Therapy Studies
306(1)
4 Conclusions and Outlook
307(1)
References
308(3)
19 Gene Therapy for Inherited Metabolic Storage Diseases
311(16)
Cathryn Mah
1 Introduction
311(1)
2 Lysosomal Storage Diseases
312(2)
3 Glycogen Storage Diseases
314(1)
4 Animal Models
315(4)
5 Cross-Correction Strategies
319(2)
6 Direct Correction of Target Tissues
321(3)
7 Conclusions and Outlook
324(1)
References
324(3)
20 Retinal Diseases
327(18)
Shannon E. Boye
Sanford L. Boye
William W. Hauswirth
1 Introduction
327(3)
2 Rod and Cone Photoreceptors
330(3)
3 Cone Photoreceptors
333(2)
4 Retinal Ganglion Cells
335(2)
5 Retinal Pigment Epithelium
337(2)
6 LCA2 Gene Therapy, a Perspective on Translational Research
339(3)
References
342(3)
21 A Brief Guide to Gene Therapy Treatments for Pulmonary Diseases
345(16)
Ashley T. Martino
Christian Mueller
Terence R. Flotte
1 Introduction
345(1)
2 Common Disorders
346(2)
2.1 Cystic Fibrosis
346(2)
2.2 Alpha-1 Antitrypsin (A1AT)
348(1)
3 Development of Viral Vectors for Lung Disease
348(2)
3.1 Adenoviral Vectors
349(1)
3.2 Adeno-Associated Viral Vectors
349(1)
3.3 Early Conclusions
349(1)
4 Enhancing Efficiency
350(2)
4.1 Alternative AAV Viral Vector Serotypes
350(1)
4.1.1 Addition of Expression Enhancing Elements
351(1)
4.2 Adenoviral Vectors
351(1)
4.3 Physiological Hurdles in the Lung Environment
352(1)
5 Non-Viral Vectors
352(1)
5.1 Cationic Liposomes
352(1)
5.2 Compacted DNA Nanoparticles
353(1)
6 Gene Therapy Development for Alpha-1 Anti-trypsin
353(1)
7 Lung Cancer Gene Therapy Development
354(1)
8 Cystic Fibrosis Animal Models
355(1)
9 Cell-Based Therapy for Cystic Fibrosis
356(1)
10 Conclusion and Outlooks
357(1)
References
358(3)
22 Cardiovascular Disease
361(18)
Darin J. Falk
Cathryn S. Mah
Barry J. Byrne
1 Introduction
361(1)
2 Therapeutic Targets
362(5)
2.1 Congenital Heart Disease
362(3)
2.2 Coronary Artery Disease and Ischemia/ Reperfusion Injury
365(1)
2.3 Oxidative Stress
365(1)
2.4 Antioxidants
366(1)
2.5 Cardiac Contractility
367(1)
3 Animal Models
367(1)
4 Vector Delivery
368(6)
5 Conclusions and Outlook
374(1)
Acknowledgments
374(1)
References
374(5)
Index 379
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