Title:
Adeno-associated virus vectors for gene therapy
Series:
Laboratory techniques in biochemistry and molecular biology ; 31
Publication Information:
Amsterdam, The Netherlands : Elsevier Science Publishing, 2005
ISBN:
9780444519498
Available:*
Library | Item Barcode | Call Number | Material Type | Item Category 1 | Status |
---|---|---|---|---|---|
Searching... | 30000010107633 | QZ50 A33 2005 | Open Access Book | Book | Searching... |
On Order
Summary
Summary
Written by well-known experts in the field, this is a timely update on Adeno-associated virus used in gene therapy. The 10 chapters cover gene therapy in specific diseases such as hemophilia, kidney diseases, and disordes of the central nervous system.
Table of Contents
Preface | p. v |
Chapter 1 Adeno-associated viral vectors for gene therapy | p. 1 |
1.1 Biological properties of adeno-associated virus | p. 1 |
1.2 AAV-based gene therapy vectors | p. 2 |
1.3 In vivo applications of rAAV | p. 4 |
1.4 Clinical experience with rAAV | p. 7 |
1.5 Persistence of rAAV vectors | p. 8 |
1.6 Safety of rAAV vector delivery | p. 11 |
1.7 Host range, alternate serotypes, and capsid modifications | p. 12 |
1.8 Remaining questions | p. 13 |
References | p. 13 |
Chapter 2 Production of research and clinical-grade recombinant adeno-associated virus vectors | p. 19 |
2.1 Adeno-associated virus biology | p. 20 |
2.2 rAAV preparation | p. 24 |
2.3 Protocols | p. 27 |
2.4 Small-scale rAAV 1, 2, and 5 vector purification | p. 28 |
2.5 Large-scale rAAV purification | p. 30 |
2.6 rAAV vector characterization | p. 31 |
2.7 Safety testing | p. 34 |
2.8 Pre-clinical regulatory compliance activities | p. 35 |
2.9 Manufacture of clinical-grade rAAV vectors | p. 38 |
2.10 Clinical manufacturing regulatory compliance activities | p. 44 |
References | p. 49 |
Chapter 3 Gene therapy for hemophilia | p. 57 |
3.1 Non-viral DNA vectors | p. 59 |
3.2 Adenovirus vectors | p. 60 |
3.3 Retrovirus vectors | p. 62 |
3.4 Adeno-associated virus vectors | p. 64 |
3.5 Immunological considerations | p. 69 |
3.6 Laboratory protocols | p. 72 |
References | p. 73 |
Chapter 4 Recombinant AAV vectors for gene transfer to the lung: a compartmental approach | p. 83 |
4.1 Introduction | p. 83 |
4.2 Genes, targets and vectors for the lung | p. 84 |
4.3 Therapies targeting the alveoli | p. 85 |
4.4 Therapies targeting the airways | p. 87 |
4.5 Therapies targeting the pulmonary vasculature and pleura | p. 92 |
4.6 Future directions | p. 93 |
References | p. 94 |
Chapter 5 Adeno-associated virus mediated gene therapy for vascular retinopathies | p. 103 |
5.1 Introduction | p. 103 |
5.2 New strategies for treating NV | p. 106 |
5.3 Protocols | p. 114 |
5.4 Discussion | p. 118 |
References | p. 119 |
Chapter 6 Gene therapy for prevention and treatment of type 1 diabetes | p. 125 |
6.1 The clinical problem diabetes | p. 125 |
6.2 Transplantation | p. 126 |
6.3 Allograft rejection: mechanisms for increasing graft acceptance | p. 127 |
6.4 Recurrent autoimmunity as a mechanism of [beta] cell allograft failure | p. 129 |
6.5 Gene transfer into islet cells | p. 131 |
6.6 Potential utility of rAAV-mediated gene therapy for islet transplantation and prevention of autoimmunity recurrence in type 1 diabetes | p. 136 |
6.7 Progress in insulin replacement strategies utilizing gene therapy | p. 142 |
6.8 Summary and future directions | p. 146 |
References | p. 147 |
Chapter 7 Gene therapy for kidney diseases | p. 161 |
7.1 Structure-function correlations | p. 162 |
7.2 Vector systems for gene delivery | p. 163 |
7.3 Methods of gene delivery | p. 170 |
7.4 Targeting specific cells in the kidney | p. 172 |
7.5 Application of gene therapy for specific kidney diseases | p. 176 |
References | p. 185 |
Chapter 8 AAV for disorders of the CNS | p. 193 |
8.1 Introduction | p. 193 |
8.2 Parkinson disease (PD) | p. 198 |
8.3 Alzheimer's disease (AD) | p. 204 |
8.4 Epilepsy | p. 206 |
8.5 Lysosomal storage disorders (LSD) | p. 208 |
8.6 Conclusion | p. 212 |
References | p. 213 |
Chapter 9 Gene therapy for cardiovascular applications | p. 225 |
9.1 Viral gene delivery systems | p. 227 |
9.2 Non-viral gene delivery systems | p. 232 |
9.3 Gene delivery route | p. 233 |
9.4 Cellular and gene therapy combinations | p. 234 |
9.5 Conclusions | p. 234 |
9.6 Methods | p. 235 |
References | p. 239 |
Chapter 10 Gene therapy for lysosomal storage disorders | p. 243 |
10.1 The lysosome | p. 243 |
10.2 Lysosomal storage diseases | p. 244 |
10.3 Current therapies | p. 244 |
10.4 Gene therapy | p. 245 |
10.5 Glycogen storage disease type II | p. 248 |
10.6 Gene therapy for GSD II: Proof of concept studies | p. 254 |
10.7 Recombinant adeno-associated virus vector studies | p. 256 |
10.8 Recombinant AAV-mediated treatment of GSDII | p. 257 |
10.9 Gene therapy for CNS pathologies in LSDs | p. 262 |
10.10 Conclusion | p. 265 |
References | p. 266 |
Index | p. 277 |