Adeno-associated viral vectors for gene therapy [electronic resource] /
edited by T.R. Flotte, K.I. Berns.
1st ed.
Amsterdam ; Boston : Elsevier, 2005.
x, 283 p. : ill.
0444519491 (alk. paper), 9780444519498 (alk. paper)
More Details
Amsterdam ; Boston : Elsevier, 2005.
0444519491 (alk. paper)
9780444519498 (alk. paper)
Licensed for access by U. of T. users.
catalogue key
Includes bibliographical references and index.
A Look Inside
Main Description
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. *Presents a timely update of gene therapy using the adeno-associated virus *Addresses gene therapy used for hemophilia, kidney diseases, and disorders of the central nervous system *Provides practical advance on vector design *Includes balanced treatment of theoretical risks of gene therapy
Table of Contents
Prefacep. v
Adeno-associated viral vectors for gene therapyp. 1
Biological properties of adeno-associated virusp. 1
AAV-based gene therapy vectorsp. 2
In vivo applications of rAAVp. 4
Clinical experience with rAAVp. 7
Persistence of rAAV vectorsp. 8
Safety of rAAV vector deliveryp. 11
Host range, alternate serotypes, and capsid modificationsp. 12
Remaining questionsp. 13
Referencesp. 13
Production of research and clinical-grade recombinant adeno-associated virus vectorsp. 19
Adeno-associated virus biologyp. 20
rAAV preparationp. 24
Protocolsp. 27
Small-scale rAAV 1, 2, and 5 vector purificationp. 28
Large-scale rAAV purificationp. 30
rAAV vector characterizationp. 31
Safety testingp. 34
Pre-clinical regulatory compliance activitiesp. 35
Manufacture of clinical-grade rAAV vectorsp. 38
Clinical manufacturing regulatory compliance activitiesp. 44
Referencesp. 49
Gene therapy for hemophiliap. 57
Non-viral DNA vectorsp. 59
Adenovirus vectorsp. 60
Retrovirus vectorsp. 62
Adeno-associated virus vectorsp. 64
Immunological considerationsp. 69
Laboratory protocolsp. 72
Referencesp. 73
Recombinant AAV vectors for gene transfer to the lung: a compartmental approachp. 83
Introductionp. 83
Genes, targets and vectors for the lungp. 84
Therapies targeting the alveolip. 85
Therapies targeting the airwaysp. 87
Therapies targeting the pulmonary vasculature and pleurap. 92
Future directionsp. 93
Referencesp. 94
Adeno-associated virus mediated gene therapy for vascular retinopathiesp. 103
Introductionp. 103
New strategies for treating NVp. 106
Protocolsp. 114
Discussionp. 118
Referencesp. 119
Gene therapy for prevention and treatment of type 1 diabetesp. 125
The clinical problem diabetesp. 125
Transplantationp. 126
Allograft rejection: mechanisms for increasing graft acceptancep. 127
Recurrent autoimmunity as a mechanism of [beta] cell allograft failurep. 129
Gene transfer into islet cellsp. 131
Potential utility of rAAV-mediated gene therapy for islet transplantation and prevention of autoimmunity recurrence in type 1 diabetesp. 136
Progress in insulin replacement strategies utilizing gene therapyp. 142
Summary and future directionsp. 146
Referencesp. 147
Gene therapy for kidney diseasesp. 161
Structure-function correlationsp. 162
Vector systems for gene deliveryp. 163
Methods of gene deliveryp. 170
Targeting specific cells in the kidneyp. 172
Application of gene therapy for specific kidney diseasesp. 176
Referencesp. 185
AAV for disorders of the CNSp. 193
Introductionp. 193
Parkinson disease (PD)p. 198
Alzheimer's disease (AD)p. 204
Epilepsyp. 206
Lysosomal storage disorders (LSD)p. 208
Conclusionp. 212
Referencesp. 213
Gene therapy for cardiovascular applicationsp. 225
Viral gene delivery systemsp. 227
Non-viral gene delivery systemsp. 232
Gene delivery routep. 233
Cellular and gene therapy combinationsp. 234
Conclusionsp. 234
Methodsp. 235
Referencesp. 239
Gene therapy for lysosomal storage disordersp. 243
The lysosomep. 243
Lysosomal storage diseasesp. 244
Current therapiesp. 244
Gene therapyp. 245
Glycogen storage disease type IIp. 248
Gene therapy for GSD II: Proof of concept studiesp. 254
Recombinant adeno-associated virus vector studiesp. 256
Recombinant AAV-mediated treatment of GSDIIp. 257
Gene therapy for CNS pathologies in LSDsp. 262
Conclusionp. 265
Referencesp. 266
Indexp. 277
Table of Contents provided by Ingram. All Rights Reserved.

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