Adeno-associated viral vectors for gene therapy:
Saved in:
| Format: | Book |
|---|---|
| Language: | English |
| Published: |
Amsterdam [u.a.]
Elsevier
2005
|
| Edition: | 1. ed. |
| Series: | Laboratory techniques in biochemistry and molecular biology
31 |
| Subjects: | |
| Online Access: | Inhaltsverzeichnis |
| Physical Description: | X, 283 S. Ill., graph. Darst. |
| ISBN: | 9780444519498 0444519491 |
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MARC
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| 245 | 1 | 0 | |a Adeno-associated viral vectors for gene therapy |c ed. by T. R. Flotte ... |
| 250 | |a 1. ed. | ||
| 264 | 1 | |a Amsterdam [u.a.] |b Elsevier |c 2005 | |
| 300 | |a X, 283 S. |b Ill., graph. Darst. | ||
| 336 | |b txt |2 rdacontent | ||
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| 490 | 1 | |a Laboratory techniques in biochemistry and molecular biology |v 31 | |
| 650 | 7 | |a Gene Therapy |2 cabt | |
| 650 | 7 | |a Gene Transfer |2 cabt | |
| 650 | 7 | |a Vectors |2 cabt | |
| 650 | 7 | |a Viruses |2 cabt | |
| 650 | 4 | |a Thérapie génique | |
| 650 | 4 | |a Vecteurs de clonage | |
| 650 | 4 | |a Adenoviridae |x genetics | |
| 650 | 4 | |a Gene Therapy |x methods | |
| 650 | 4 | |a Gene therapy | |
| 650 | 4 | |a Genetic Vectors | |
| 650 | 4 | |a Genetic vectors | |
| 700 | 1 | |a Flotte, Terrence R. |e Sonstige |4 oth | |
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Record in the Search Index
| _version_ | 1804134160471687168 |
|---|---|
| adam_text | Contents
Preface v
Chapter 1. Adeno associated viral vectors for
gene therapy 1
T. R. Flotte and K. I. Berns
1.1. Biological properties of adeno associated virus 1
1.2. AAV based gene therapy vectors 2
1.3. In vivo applications of rAAV 4
1.4. Clinical experience with rAAV 7
1.5. Persistence of rAAV vectors 8
1.6. Safety of rAAV vector delivery 11
1.7. Host range, alternate serotypes, and capsid modifications 12
1.8. Remaining questions 13
References 13
Chapter 2. Production of research and clinical grade
recombinant adeno associated virus vectors 19
J. D. Francis and R. O. Snyder
2.1. Adeno associated virus biology 20
2.2. rAAV preparation 24
2.3. Protocols 27
2.4. Small scale rAAV 1, 2, and 5 vector purification 28
2.5. Large scale rAAV purification 30
2.6. rAAV vector characterization 31
2.7. Safety testing 34
2.8. Pre clinical regulatory compliance activities 35
2.9. Manufacture of clinical grade rAAV vectors 38
2.10. Clinical manufacturing regulatory compliance activities 44
References 49
Viii CONTENTS
Chapter 3. Gene therapy for hemophilia 57
C. Mah
j
3.1. Non viral DNA vectors 59 j
3.2. Adenovirus vectors 60 }
3.3. Retrovirus vectors 62 ¦
3.4. Adeno associated virus vectors 64 •
3.5. Immunological considerations 69
3.6. Laboratory protocols 72
References 73
Chapter 4. Recombinant AAV vectors for gene transfer
to the lung: a compartmental approach 83
T. R. Flotte
4.1. Introduction 83
4.2. Genes, targets and vectors for the lung 84
4.3. Therapies targeting the alveoli 85
4.4. Therapies targeting the airways 87
4.5. Therapies targeting the pulmonary vasculature and pleura 92
4.6. Future directions 93
References 94
Chapter 5. Adeno associated virus mediated gene therapy
for vascular retinopathies 103
B. J. Raisler, W. T. Deng, K. I. Berns and W. W. Hauswirth
5.1. Introduction 103
5.2. New strategies for treating NV 106
5.3. Protocols 114
5.4. Discussion 118
References 119
CONTENTS IX
Chapter 6. Gene therapy for prevention and treatment of
type 1 diabetes 125
M. H. Kapturczak, B. R. Burkhardt and M. A. Atkinson
6.1. The clinical problem diabetes 125
6.2. Transplantation 126
6.3. Allograft rejection: mechanisms for increasing
graft acceptance 127
6.4. Recurrent autoimmunity as a mechanism of fi cell
allograft failure 129
6.5. Gene transfer into islet cells 131
6.6. Potential utility of rAAV mediated gene therapy for islet
transplantation and prevention of autoimmunity
recurrence in type 1 diabetes 136
6.7. Progress in insulin replacement strategies utilizing
gene therapy 142
6.8. Summary and future directions 146
References 147
Chapter 7. Gene therapy for kidney diseases 161
S. Chen, K. M. Madsen, C. C. Tisher and A. Agarwal
7.1. Structure function correlations 162
7.2. Vector systems for gene delivery 163
7.3. Methods of gene delivery 170
7.4. Targeting specific cells in the kidney 172
7.5. Application of gene therapy for specific
kidney diseases 176
References 185
Chapter 8. A A V for disorders of the CNS 193
C. Burger, R. J. Mandel and N. Muzyczka
8.1. Introduction 193
j 8.2. Parkinson disease (PD) 198
I 8.3. Alzheimer s disease (AD) 204
I 8.4. Epilepsy 206
X CONTENTS
8.5. Lysosomal storage disorders (LSD) 208
8.6. Conclusion 212
References 213
Chapter 9. Gene therapy for cardiovascular applications .. 225
C. A. Pacak, C. Mah and B. J. Byrne
9.1. Viral gene delivery systems 227
9.2. Non viral gene delivery systems ¦ . . 232
9.3. Gene delivery route 233
9.4. Cellular and gene therapy combinations 234
9.5. Conclusions 234
9.6. Methods 235
References 239
Chapter 10. Gene therapy for lysosomal storage disorders. 243
K. O. Cresawn and B. J. Byrne
10.1. The lysosome . 243
10.2. Lysosomal storage diseases . 244
10.3. Current therapies . 244
10.4. Gene therapy . 245
10.5. Glycogen storage disease type II . 248
10.6. Gene therapy for GSD II: Proof of concept studies . 254
10.7. Recombinant adeno associated virus vector studies . 256
10.8. Recombinant AAV mediated treatment of GSDII . 257
10.9. Gene therapy for CNS pathologies in LSDs . 262
10.10. Conclusion . 265
References . 266
Index 277
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| indexdate | 2024-07-09T20:20:12Z |
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| isbn | 9780444519498 0444519491 |
| language | English |
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| spelling | Adeno-associated viral vectors for gene therapy ed. by T. R. Flotte ... 1. ed. Amsterdam [u.a.] Elsevier 2005 X, 283 S. Ill., graph. Darst. txt rdacontent n rdamedia nc rdacarrier Laboratory techniques in biochemistry and molecular biology 31 Gene Therapy cabt Gene Transfer cabt Vectors cabt Viruses cabt Thérapie génique Vecteurs de clonage Adenoviridae genetics Gene Therapy methods Gene therapy Genetic Vectors Genetic vectors Flotte, Terrence R. Sonstige oth Laboratory techniques in biochemistry and molecular biology 31 (DE-604)BV005871299 31 HBZ Datenaustausch application/pdf http://bvbr.bib-bvb.de:8991/F?func=service&doc_library=BVB01&local_base=BVB01&doc_number=013834654&sequence=000002&line_number=0001&func_code=DB_RECORDS&service_type=MEDIA Inhaltsverzeichnis |
| spellingShingle | Adeno-associated viral vectors for gene therapy Laboratory techniques in biochemistry and molecular biology Gene Therapy cabt Gene Transfer cabt Vectors cabt Viruses cabt Thérapie génique Vecteurs de clonage Adenoviridae genetics Gene Therapy methods Gene therapy Genetic Vectors Genetic vectors |
| title | Adeno-associated viral vectors for gene therapy |
| title_auth | Adeno-associated viral vectors for gene therapy |
| title_exact_search | Adeno-associated viral vectors for gene therapy |
| title_full | Adeno-associated viral vectors for gene therapy ed. by T. R. Flotte ... |
| title_fullStr | Adeno-associated viral vectors for gene therapy ed. by T. R. Flotte ... |
| title_full_unstemmed | Adeno-associated viral vectors for gene therapy ed. by T. R. Flotte ... |
| title_short | Adeno-associated viral vectors for gene therapy |
| title_sort | adeno associated viral vectors for gene therapy |
| topic | Gene Therapy cabt Gene Transfer cabt Vectors cabt Viruses cabt Thérapie génique Vecteurs de clonage Adenoviridae genetics Gene Therapy methods Gene therapy Genetic Vectors Genetic vectors |
| topic_facet | Gene Therapy Gene Transfer Vectors Viruses Thérapie génique Vecteurs de clonage Adenoviridae genetics Gene Therapy methods Gene therapy Genetic Vectors Genetic vectors |
| url | http://bvbr.bib-bvb.de:8991/F?func=service&doc_library=BVB01&local_base=BVB01&doc_number=013834654&sequence=000002&line_number=0001&func_code=DB_RECORDS&service_type=MEDIA |
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