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Guide to Human Gene Therapy > 생명과학

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Guide to Human Gene Therapy
판매가격 15,000원
저자 Sergei
도서종류 외국도서
출판사 World Scientific
발행언어 영어
발행일 2010-6
페이지수 416
ISBN 9789814280907
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    1. Non-Viral Gene Therapy 1
    Sean M. Sullivan
    1. Introduction . . . . . . . . . . . . . . . . . . . . . . . . 1
    2. Plasmid DNA . . . . . . . . . . . . . . . . . . . . . . . 3
    2.1 Plasmid DNA Manufacture . . . . . . . . . . . . . 5
    3. Plasmid DNA Gene Transfer Methods . . . . . . . . . . 6
    3.1 Plasmid DNA or “Naked DNA” as a Gene Delivery
    System . . . . . . . . . . . . . . . . . . . . . . . 6
    3.1.1 Electroporation of Naked DNA . . . . . . . 8
    3.1.2 Sonoporation of Naked DNA . . . . . . . . 9
    3.2 Plasmid DNA Formulations . . . . . . . . . . . . 9
    3.2.1 Cationic Lipids . . . . . . . . . . . . . . . 9
    3.2.1.1 In vitro transfection . . . . . . . . 10
    3.2.1.2 Systemic in vivo gene transfer . . . 11
    3.2.1.3 Local administration of cationic
    lipid/pDNA transfection
    complexes . . . . . . . . . . . . . 12
    3.3 Polymer . . . . . . . . . . . . . . . . . . . . . . . 14
    3.3.1 Cationic Polymers . . . . . . . . . . . . . . 14
    3.3.2 Neutral Polymer . . . . . . . . . . . . . . . 15
    Conclusions . . . . . . . . . . . . . . . . . . . . . . . . 17
    References . . . . . . . . . . . . . . . . . . . . . . . . . . . 17

    2. Adenoviral Vectors 21
    Stuart A. Nicklin and Andrew H. Baker
    1. Introduction . . . . . . . . . . . . . . . . . . . . . . . . 21
    2. Adenoviral Capsid Structure . . . . . . . . . . . . . . . 22
    3. Adenoviral Cell Entry . . . . . . . . . . . . . . . . . . . 23
    4. Production of Adenoviral Vectors . . . . . . . . . . . . . 24
    5. Production of Targeted Adenoviral Vectors . . . . . . . . 26
    6. Gene Therapy Applications . . . . . . . . . . . . . . . . 28
    7. Immune Responses to Ad Vectors . . . . . . . . . . . . 30
    8. Safety and Regulatory Issues . . . . . . . . . . . . . . . 32
    9. Conclusions . . . . . . . . . . . . . . . . . . . . . . . . 33
    References . . . . . . . . . . . . . . . . . . . . . . . . . . . 33

    3. Retroviral Vectors and Integration Analysis 37
    Cynthia C. Bartholomae, Romy Kirsten, Hanno Glimm,
    Manfred Schmidt and Christof von Kalle
    1. Introduction . . . . . . . . . . . . . . . . . . . . . . . . 37
    2. Design, Production and Mechanism of Transduction . . . 38
    3. In vivo Application . . . . . . . . . . . . . . . . . . . . 41
    4. Side Effects in Retroviral Gene Therapy . . . . . . . . . 42
    4.1 Distribution of Retroviral Integration Sites
    in the Cellular Genome . . . . . . . . . . . . . . . 42
    4.2 Side Effects in Clinical and Preclinical Gene
    Therapy Studies . . . . . . . . . . . . . . . . . . . 45
    5. New Strategies for Vector Biosafety in Gene Therapy . . 47
    References . . . . . . . . . . . . . . . . . . . . . . . . . . . 49
    4. Lentiviral Vectors 53
    Janka Matrai, Marinee K. L. Chuah
    and Thierry VandenDriessche
    1. Basic Viral Biology . . . . . . . . . . . . . . . . . . . . 53
    2. Vector Design and Production . . . . . . . . . . . . . . 56
    2.1 Vector Development . . . . . . . . . . . . . . . . 56
    2.2 Vector Production . . . . . . . . . . . . . . . . . . 58
    3. Gene Transfer Concepts and Potential Applications . . . 59
    3.1 Target Cells and Diseases . . . . . . . . . . . . . . 59
    3.2 Pseudotyping . . . . . . . . . . . . . . . . . . . . 59
    3.3 Cell Type Specific Targeting . . . . . . . . . . . . 60
    3.4 Integration-Defective Lentiviral Vectors . . . . . . 60
    4. Immune Consequences . . . . . . . . . . . . . . . . . . 62
    5. Safety Issues . . . . . . . . . . . . . . . . . . . . . . . 63
    6. Conclusions and Perspectives . . . . . . . . . . . . . . . 64
    References . . . . . . . . . . . . . . . . . . . . . . . . . . . 64

    5. Herpes Simplex Virus Vectors 69
    William F. Goins, David M. Krisky, James B. Wechuck,
    Darren Wolfe, Justus B. Cohen and Joseph C. Glorioso
    1. Introduction . . . . . . . . . . . . . . . . . . . . . . . . 69
    2. HSVBiology in the Design of Replication DefectiveVectors 74
    3. HSV Vector Design Technology . . . . . . . . . . . . . 77
    4. Gene Transfer/Therapy Applications . . . . . . . . . . . 79
    5. Immunology . . . . . . . . . . . . . . . . . . . . . . . 80
    6. Safety and Regulatory Issues . . . . . . . . . . . . . . . 81
    7. Summary . . . . . . . . . . . . . . . . . . . . . . . . . 81
    References . . . . . . . . . . . . . . . . . . . . . . . . . . . 82

    6. Adeno-Associated Viral (AAV) Vectors 87
    Nicholas Muzyczka
    1. Introduction . . . . . . . . . . . . . . . . . . . . . . . . 87
    2. Biology of AAV . . . . . . . . . . . . . . . . . . . . . . 88
    3. Vector Technology . . . . . . . . . . . . . . . . . . . . 93
    4. Vector Characteristics In Vivo . . . . . . . . . . . . . . . 96
    5. Next Generation Vectors . . . . . . . . . . . . . . . . . 98
    6. Conclusions and Outlook . . . . . . . . . . . . . . . . . 99
    References . . . . . . . . . . . . . . . . . . . . . . . . . . . 99

    7. Regulatory RNA in Gene Therapy 103
    Alfred. S. Lewin
    1. Introduction . . . . . . . . . . . . . . . . . . . . . . . . 103
    2. Delivery of Therapeutic RNAs . . . . . . . . . . . . . . 106
    3. Ribozymes . . . . . . . . . . . . . . . . . . . . . . . . 109
    4. RNAi for Gene Therapy . . . . . . . . . . . . . . . . . 111
    5. Gene Therapy Using miRNA . . . . . . . . . . . . . . . 114
    6. Aptamers, Decoys and Bi-Functional RNAs . . . . . . . 115
    7. Modification of Cis-Acting Regulatory
    RNA Sequences . . . . . . . . . . . . . . . . . . . . . . 116
    8. Conclusions . . . . . . . . . . . . . . . . . . . . . . . . 119
    References . . . . . . . . . . . . . . . . . . . . . . . . . . . 120

    8. DNA Integrating Vectors (Transposon, Integrase) 123
    Lauren E. Woodard and Michele P. Calos
    1. Basic Vector Biology . . . . . . . . . . . . . . . . . . . 123
    1.1 Transposon Systems . . . . . . . . . . . . . . . . 124
    1.2 Integrase Systems . . . . . . . . . . . . . . . . . . 126
    2. Vector Design and Production . . . . . . . . . . . . . . 128
    2.1 Design of Transposon Systems . . . . . . . . . . . 128
    2.2 Design of Integrase Systems . . . . . . . . . . . . 128
    2.3 Production of Plasmid DNA . . . . . . . . . . . . 129
    3. Gene Transfer Protocols and Potential Applications . . . 130
    3.1 Hepatocyte Transfection via Hydrodynamic
    Injection . . . . . . . . . . . . . . . . . . . . . . . 130
    3.2 Lipophilic Complexes to Transfect Endothelial
    Cells and Glioblastoma . . . . . . . . . . . . . . . 131
    3.3 Direct DNA Injection and Electroporation
    to Target Muscle, Retina, and Joints . . . . . . . . 131
    3.4 Integration into Cultured Cells for
    Ex vivo Gene Therapy . . . . . . . . . . . . . . . 131
    4. Immunology . . . . . . . . . . . . . . . . . . . . . . . 132
    5. Safety and Regulatory Issues . . . . . . . . . . . . . . . 133
    5.1 Integration Profiles and Associated Hazards . . . . 133
    5.2 Efforts to Enhance Integration Specificity . . . . . 133
    5.3 Effects on Tumor Latency in Mouse Models
    of Cancer . . . . . . . . . . . . . . . . . . . . . . 134
    References . . . . . . . . . . . . . . . . . . . . . . . . . . . 135

    9. Homologous Recombination and Targeted Gene
    Modification for Gene Therapy 139
    Matthew Porteus
    1. Introduction . . . . . . . . . . . . . . . . . . . . . . . . 139
    2. Problems with Using Gene Targeting by Homologous
    Recombination . . . . . . . . . . . . . . . . . . . . . . 140
    3. Homologous Recombination in Embryonic
    Stem Cells . . . . . . . . . . . . . . . . . . . . . . . . . 141
    4. Homologous Recombination using
    Adeno-Associated Virus . . . . . . . . . . . . . . . . . 144
    5. Site-Specific Modification of the Genome using
    Double-Strand Breaks . . . . . . . . . . . . . . . . . . 144
    6. Double-Strand Break Repair . . . . . . . . . . . . . . . 144
    7. Double-Strand Break Induced Homologous
    Recombination . . . . . . . . . . . . . . . . . . . . . . 146
    8. Re-design of Homing Endonucleases to Recognize
    New Target Sites . . . . . . . . . . . . . . . . . . . . . 146
    9. Development of Zinc Finger Nucleases . . . . . . . . . 147
    10. Using Zinc Finger Nucleases to Stimulate
    Gene Targeting . . . . . . . . . . . . . . . . . . . . . . 147
    11. Using Zinc Finger Nucleases to Site-Specifically Modify
    Genes by Mutagenic Non-Homologous
    End-Joining . . . . . . . . . . . . . . . . . . . . . . . . 149
    12. Strategies of Zinc Finger Nuclease Design . . . . . . . . 151
    13. Aspects of Zinc Finger Binding Sites and Structure
    of Zinc Finger Nucleases . . . . . . . . . . . . . . . . . 153
    14. Zinc Finger Nuclease Toxicity: Measuring
    and Minimizing . . . . . . . . . . . . . . . . . . . . . . 154
    15. The Challenge of Delivery . . . . . . . . . . . . . . . . 156
    16. Future Directions and Promise of Homologous
    Recombination as a Gene Correction Approach
    to Gene Therapy . . . . . . . . . . . . . . . . . . . . . 157
    References . . . . . . . . . . . . . . . . . . . . . . . . . . . 157
    10. Gene Switches for Pre-Clinical Studies in Gene Therapy 163
    Caroline Le Guiner, Knut Stieger, Alice Toromanoff,
    Fabienne Rolling, Philippe Moullier and Oumeya Adjali
    1. Introduction . . . . . . . . . . . . . . . . . . . . . . . . 163
    2. Rapamycin-Dependent Regulatable System . . . . . . . 165
    2.1 Molecular Mechanisms Involved in Transgene
    Regulation . . . . . . . . . . . . . . . . . . . . . 165
    2.2 Pharmacology of Rapamycin . . . . . . . . . . . . 166
    2.3 Translation Development of the Rapamycin
    Dependent Regulation System . . . . . . . . . . . 166
    3. Tetracycline-Dependent Regulatable Systems . . . . . . 168
    3.1 Molecular Mechanisms Involved in Transgene
    Regulation . . . . . . . . . . . . . . . . . . . . . 168
    3.2 Pharmacology of Doxycycline (Dox) . . . . . . . . 171
    3.3 Translational Development of Tet-dependant
    Regulation Systems . . . . . . . . . . . . . . . . . 171
    4. Other Regulatable Systems . . . . . . . . . . . . . . . . 175
    5. General Conclusions . . . . . . . . . . . . . . . . . . . 177
    References . . . . . . . . . . . . . . . . . . . . . . . . . . . 177

    11. Gene Therapy for Central Nervous System Disorders 181
    Deborah Young and Patricia A. Lawlor
    1. Introduction . . . . . . . . . . . . . . . . . . . . . . . . 181
    2. Gene Therapy for Parkinson’s Disease . . . . . . . . . . 182
    3. Gene Therapy for Temporal Lobe Epilepsy . . . . . . . 186
    4. Huntington’s Disease Gene Therapy . . . . . . . . . . . 187
    5. Amyotrophic Lateral Sclerosis (ALS) . . . . . . . . . . 189
    6. Gene Therapy for Canavan Disease . . . . . . . . . . . . 190
    7. Gene Therapy for Alzheimer’s Disease . . . . . . . . . . 191
    8. Conclusions and Outlook . . . . . . . . . . . . . . . . . 193
    References . . . . . . . . . . . . . . . . . . . . . . . . . . . 194

    12. Gene Therapy of Hemoglobinopathies 197
    Angela E. Rivers and Arun Srivastava
    1. Introduction . . . . . . . . . . . . . . . . . . . . . . . . 198
    2. β-Thalassemia . . . . . . . . . . . . . . . . . . . . . . 198
    3. Sickle Cell Disease . . . . . . . . . . . . . . . . . . . . 199
    4. Gene Therapy . . . . . . . . . . . . . . . . . . . . . . . 200
    4.1 Oncoretroviral Vector-Mediated Globin
    Gene Transfer . . . . . . . . . . . . . . . . . . . . 202
    4.2 Lentiviral Vector-Mediated Globin
    Gene Transfer . . . . . . . . . . . . . . . . . . . . 203
    4.3 Adeno-Associated Viral Vector-Mediated Globin
    Gene Transfer . . . . . . . . . . . . . . . . . . . . 204
    References . . . . . . . . . . . . . . . . . . . . . . . . . . . 208

    13. Gene Therapy for Primary Immunodeficiencies 213
    Aisha Sauer, Barbara Cassani and Alessandro Aiuti
    1. Introduction . . . . . . . . . . . . . . . . . . . . . . . . 214
    2. Adenosine Deaminase (ADA)-deficient SCID . . . . . . 215
    3. X-linked Severe Combined Immunodeficiency
    (SCID-X1) . . . . . . . . . . . . . . . . . . . . . . . . 218
    4. Gene Therapy for Other SCIDs . . . . . . . . . . . . . . 220
    4.1 V(D)J Recombination Defects . . . . . . . . . . . 220
    4.2 Purine Nucleoside Phosphorylase (PNP)
    Deficiency . . . . . . . . . . . . . . . . . . . . . . 222
    4.3 Janus Kinase 3 (Jak3) Deficiency . . . . . . . . . . 222
    4.4 IL-7R Deficiency . . . . . . . . . . . . . . . . . . 223
    4.5 Zeta Associated 70 kDa Phosphoprotein
    (ZAP-70) Deficiency . . . . . . . . . . . . . . . . 223
    5. Wiskott-Aldrich-Syndrome (WAS) . . . . . . . . . . . . 224
    6. Chronic Granulomatous Disease . . . . . . . . . . . . . 225
    7. Conclusions and Outlook . . . . . . . . . . . . . . . . . 227
    References . . . . . . . . . . . . . . . . . . . . . . . . . . . 228

    14. Gene Therapy for Hemophilia 233
    David Markusic, Babak Moghimi and Roland Herzog
    1. Introduction . . . . . . . . . . . . . . . . . . . . . . . . 233
    2. Limitations of Hemophilia TreatmentWith
    Coagulation Factor Concentrates or Recombinant
    Coagulation Factors . . . . . . . . . . . . . . . . . . . . 235
    3. Gene Transfer for Correction of Hemophilia . . . . . . . 236
    3.1 Ex Vivo Gene Transfer of F.VIII and F.IX . . . . . 236
    3.2 In Vivo Gene Transfer of F.VIII and F.IX . . . . . . 237
    4. AAV is a Preferred Gene Therapy Vector for In Vivo
    Gene Transfer to Correct of Hemophilia . . . . . . . . . 238
    5. Immunological Considerations for Efficient F.IX
    Gene Transfer . . . . . . . . . . . . . . . . . . . . . . . 239
    6. Advancements from Small and Large Animal Models
    of Hemophilia . . . . . . . . . . . . . . . . . . . . . . . 242
    6.1 Murine Hemophilia Models . . . . . . . . . . . . 242
    6.2 Canine Hemophilia Models . . . . . . . . . . . . . 242
    7. Gene Therapy Trials for Hemophilia Past, Present, and
    Future . . . . . . . . . . . . . . . . . . . . . . . . . . . 244
    8. Conclusions . . . . . . . . . . . . . . . . . . . . . . . . 245
    References . . . . . . . . . . . . . . . . . . . . . . . . . . . 246

    15. Gene Therapy for Obesity and Diabetes 251
    Sergei Zolotukhin and Clive H. Wasserfall
    1. Introduction . . . . . . . . . . . . . . . . . . . . . . . . 251
    2. Understanding Obesity: WhyWe Get Fat . . . . . . . . 252
    2.1 Genetic Factors: Human Obesity Gene Map . . . . 252
    2.2 Environmental Factors: The Big Two and Other
    Causal Contributors . . . . . . . . . . . . . . . . . 253
    3. General Strategies in Gene Therapy for Obesity . . . . . 253
    4. Gene Delivery Vehicles . . . . . . . . . . . . . . . . . . 255
    5. Gene Targets for Obesity . . . . . . . . . . . . . . . . . 255
    5.1 Leptin . . . . . . . . . . . . . . . . . . . . . . . . 255
    5.2 Neurocytokines . . . . . . . . . . . . . . . . . . . 256
    5.3 AMP-Activated Protein Kinase (AMPK) . . . . . . 256
    5.4 Adiponectin . . . . . . . . . . . . . . . . . . . . . 257
    5.5 Wnt-10b . . . . . . . . . . . . . . . . . . . . . . . 257
    5.6 Obesity Gene Menu a la Carte . . . . . . . . . . . 258
    5.7 Obesity and Diabetes . . . . . . . . . . . . . . . . 259
    References . . . . . . . . . . . . . . . . . . . . . . . . . . . 260

    16. Gene Therapy for Duchenne Muscular
    Dystrophy 261
    Takashi Okada and Shin’ichi Takeda
    1. Introduction . . . . . . . . . . . . . . . . . . . . . . . . 261
    1.1 Background of Duchenne Muscular Dystrophy . . 261
    2. Gene-replacement Strategies using Virus Vectors . . . . 262
    2.1 Choice of Vector . . . . . . . . . . . . . . . . . . 262
    2.2 Modification of the Dystrophin Gene
    and Promoter . . . . . . . . . . . . . . . . . . . . 264
    2.3 Use of Surrogate Genes . . . . . . . . . . . . . . . 266
    3. AAV-Mediated Transduction of Animal Models . . . . . 266
    3.1 Vector Production . . . . . . . . . . . . . . . . . . 266
    3.2 Animal Models for the Gene
    Transduction Study . . . . . . . . . . . . . . . . . 267
    3.3 Immunological Issues of rAAV . . . . . . . . . . . 268
    3.4 Intravascular Vector Administration
    by Limb Perfusion . . . . . . . . . . . . . . . . . 269
    3.5 Global Muscle Therapies . . . . . . . . . . . . . . 269
    4. Safety and Potential Impact of Clinical Trials . . . . . . 270
    5. Development of Alternative Strategies . . . . . . . . . . 271
    5.1 Design of Read-through Drugs . . . . . . . . . . . 271
    5.2 Modification of mRNA Splicing . . . . . . . . . . 272
    5.3 Ex Vivo Gene Therapy . . . . . . . . . . . . . . . 272
    6. Future Perspectives . . . . . . . . . . . . . . . . . . . . 273
    6.1 Pharmacological Intervention . . . . . . . . . . . . 273
    6.2 Capsid Modification . . . . . . . . . . . . . . . . 273
    7. Conclusions and Outlook . . . . . . . . . . . . . . . . . 273
    References . . . . . . . . . . . . . . . . . . . . . . . . . . . 274

    17. Cancer Gene Therapy 279
    Kirsten A.K. Weigel-Van Aken
    1. Introduction . . . . . . . . . . . . . . . . . . . . . . . . 280
    2. Targeting the Tumor Cell . . . . . . . . . . . . . . . . . 280
    2.1 DNA Electroporation . . . . . . . . . . . . . . . . 280
    2.2 Non-Oncolytic Viral Vectors . . . . . . . . . . . . 281
    2.2.1 Retrovirus . . . . . . . . . . . . . . . . . . 281
    2.2.2 Lentivirus . . . . . . . . . . . . . . . . . . 282
    2.3 Oncolytic Viruses . . . . . . . . . . . . . . . . . . 282
    2.3.1 Herpesvirus . . . . . . . . . . . . . . . . . 283
    2.3.2 Adenovirus . . . . . . . . . . . . . . . . . . 283
    2.3.3 Poxvirus . . . . . . . . . . . . . . . . . . . 284
    2.3.4 Measles virus . . . . . . . . . . . . . . . . 285
    2.3.5 Vesicular stomatitis virus . . . . . . . . . . 285
    3. Targeting the Immune System . . . . . . . . . . . . . . 286
    3.1 Cancer Vaccines . . . . . . . . . . . . . . . . . . . 287
    3.1.1 Vaccinia virus . . . . . . . . . . . . . . . . 287
    3.1.2 Lentivirus . . . . . . . . . . . . . . . . . . 287
    3.1.3 Adenovirus . . . . . . . . . . . . . . . . . . 288
    3.1.4 Parvoviruses . . . . . . . . . . . . . . . . . 288
    3.2 Mesenchymal Stem Cells (MSC)
    as Delivery Vehicles . . . . . . . . . . . . . . . . 288
    3.3 Adoptive T Cell Transfer . . . . . . . . . . . . . . 288
    4. Targeting the Tumor Microenvironment . . . . . . . . . 289
    5. Challenges and Risks of Cancer Gene Therapy . . . . . 289
    6. Novel Strategies . . . . . . . . . . . . . . . . . . . . . . 290
    6.1 Prime/Boost Regimens . . . . . . . . . . . . . . . 290
    6.2 Immune Cells as Carriers for Viruses . . . . . . . . 290
    7. Conclusions . . . . . . . . . . . . . . . . . . . . . . . . 291
    References . . . . . . . . . . . . . . . . . . . . . . . . . . . 291

    18. Gene Therapy for Autoimmune Disorders 295
    Daniel F. Gaddy, Melanie A. Ruffner and Paul D. Robbins
    1. Introduction . . . . . . . . . . . . . . . . . . . . . . . . 295
    2. Rheumatoid Arthritis . . . . . . . . . . . . . . . . . . . 296
    2.1 Background . . . . . . . . . . . . . . . . . . . . . 296
    2.2 Existing Therapies . . . . . . . . . . . . . . . . . 296
    2.3 Target Tissues and Routes of Delivery . . . . . . . 297
    2.3.1 Local RA Gene Therapy . . . . . . . . . . . 297
    2.3.2 Systemic RA Gene Therapy . . . . . . . . . 297
    2.4 Immunomodulation . . . . . . . . . . . . . . . . . 298
    2.5 Overview of Preclinical Gene Therapy Studies . . 299
    2.6 Overview of Clinical Gene Therapy Studies . . . . 301
    3. Type I Diabetes Mellitus . . . . . . . . . . . . . . . . . 301
    3.1 Background . . . . . . . . . . . . . . . . . . . . . 301
    3.2 Existing Therapies . . . . . . . . . . . . . . . . . 302
    3.3 Target Tissues and Routes of Delivery . . . . . . . 303
    3.4 Immunomodulation . . . . . . . . . . . . . . . . . 303
    3.5 Overview of Preclinical Gene Therapy Studies . . 305
    3.6 Overview of Clinical Gene Therapy Studies . . . . 306
    4. Conclusions and Outlook . . . . . . . . . . . . . . . . . 307
    References . . . . . . . . . . . . . . . . . . . . . . . . . . . 308

    19. Gene Therapy for Inherited Metabolic Storage Diseases 311
    Cathryn Mah
    1. Introduction . . . . . . . . . . . . . . . . . . . . . . . . 311
    2. Lysosomal Storage Diseases . . . . . . . . . . . . . . . 312
    3. Glycogen Storage Diseases . . . . . . . . . . . . . . . . 314
    4. Animal Models . . . . . . . . . . . . . . . . . . . . . . 315
    5. Cross-Correction Strategies . . . . . . . . . . . . . . . . 319
    6. Direct Correction of Target Tissues . . . . . . . . . . . . 321
    7. Conclusions and Outlook . . . . . . . . . . . . . . . . . 324
    References . . . . . . . . . . . . . . . . . . . . . . . . . . . 324

    20. Retinal Diseases 327
    Shannon E. Boye, Sanford L. Boye and William W. Hauswirth
    1. Introduction . . . . . . . . . . . . . . . . . . . . . . . . 327
    2. Rod and Cone Photoreceptors . . . . . . . . . . . . . . 330
    3. Cone Photoreceptors . . . . . . . . . . . . . . . . . . . 333
    4. Retinal Ganglion Cells . . . . . . . . . . . . . . . . . . 335
    5. Retinal Pigment Epithelium . . . . . . . . . . . . . . . 337
    6. LCA2 Gene Therapy, a Perspective on Translational
    Research . . . . . . . . . . . . . . . . . . . . . . . . . 339
    References . . . . . . . . . . . . . . . . . . . . . . . . . . . 342

    21. A Brief Guide to Gene Therapy Treatments
    for Pulmonary Diseases 345
    Ashley T. Martino, Christian Mueller
    and Terence R. Flotte
    1. Introduction . . . . . . . . . . . . . . . . . . . . . . . . 345
    2. Common Disorders . . . . . . . . . . . . . . . . . . . . 346
    2.1 Cystic Fibrosis . . . . . . . . . . . . . . . . . . . 346
    2.2 Alpha-1 Antitrypsin (A1AT) . . . . . . . . . . . . 348
    3. Development of Viral Vectors for Lung Disease . . . . . 348
    3.1 Adenoviral Vectors . . . . . . . . . . . . . . . . . 349
    3.2 Adeno-Associated Viral Vectors . . . . . . . . . . 349
    3.3 Early Conclusions . . . . . . . . . . . . . . . . . 349
    4. Enhancing Efficiency . . . . . . . . . . . . . . . . . . . 350
    4.1 Alternative AAV Viral Vector Serotypes . . . . . . 350
    4.1.1 Addition of Expression Enhancing
    Elements . . . . . . . . . . . . . . . . . . . 351
    4.2 Adenoviral Vectors . . . . . . . . . . . . . . . . . 351
    4.3 Physiological Hurdles in the Lung
    Environment . . . . . . . . . . . . . . . . . . . . 352
    5. Non-Viral Vectors . . . . . . . . . . . . . . . . . . . . . 352
    5.1 Cationic Liposomes . . . . . . . . . . . . . . . . . 352
    5.2 Compacted DNA Nanoparticles . . . . . . . . . . 353
    6. Gene Therapy Development for Alpha-1
    Anti-trypsin . . . . . . . . . . . . . . . . . . . . . . . . 353
    7. Lung Cancer Gene Therapy Development . . . . . . . . 354
    8. Cystic Fibrosis Animal Models . . . . . . . . . . . . . . 355
    9. Cell-Based Therapy for Cystic Fibrosis . . . . . . . . . 356
    10. Conclusion and Outlooks . . . . . . . . . . . . . . . . . 357
    References . . . . . . . . . . . . . . . . . . . . . . . . . . . 358

    22. Cardiovascular Disease 361
    Darin J. Falk, Cathryn S. Mah and Barry J. Byrne
    1. Introduction . . . . . . . . . . . . . . . . . . . . . . . . 361
    2. Therapeutic Targets . . . . . . . . . . . . . . . . . . . . 362
    2.1 Congenital Heart Disease . . . . . . . . . . . . . . 362
    2.2 Coronary Artery Disease and Ischemia/
    Reperfusion Injury . . . . . . . . . . . . . . . . . 365
    2.3 Oxidative Stress . . . . . . . . . . . . . . . . . . . 365
    2.4 Antioxidants . . . . . . . . . . . . . . . . . . . . 366
    2.5 Cardiac Contractility . . . . . . . . . . . . . . . . 367
    3. Animal Models . . . . . . . . . . . . . . . . . . . . . . 367
    4. Vector Delivery . . . . . . . . . . . . . . . . . . . . . . 368
    5. Conclusions and Outlook . . . . . . . . . . . . . . . . . 374
    Acknowledgments . . . . . . . . . . . . . . . . . . . . . . . 374
    References . . . . . . . . . . . . . . . . . . . . . . . . . . . 374

    Index 379
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