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Repair of dystrophin gene 211
Routes of administration of gene therapy in DMD 211
Types of dystrophin constructs 211
Viral vectors for DMD 212
Conclusions and future prospects of gene therapy of DMD 213
Limb-girdle muscular dystrophy 214
Myotonic dystrophy 214
Spinal muscular atrophy 215
Antisense gene therapy of SMA 215
Hereditary neuropathies 215
Charcot-Marie-Tooth disease 215
Hereditary axonal neuropathies of the peripheral nerves 216
Gene therapy of mitochondrial disorders 216
Companies involved in gene therapy of genetic disorders 217
5. Gene Therapy of Cancer 219
Strategies for cancer gene therapy 219
Direct gene delivery to the tumor 220
Injection into tumor 220
Direct injection of adenoviral vectors 220
Direct injection of a plasmid DNA-liposome complex 221
A polymer approach to local gene therapy for cancer 221
Electroporation for cancer gene therapy 221
Control of gene expression in tumor by local heat 222
Radiation-guided gene therapy of cancer 222
Nanoparticles to facilitate combination of hyperthermia and gene therapy 223
Cell-based cancer gene therapy 223
Adoptive cell therapy 223
Cytokine gene therapy 224
Genetic modification of human hematopoietic stem cells 227
Immunogene therapy 227
Cancer vaccines 228
Genetically modified cancer cell vaccines 228
GVAX cancer vaccines 228
Genetically modified dendritic cells 229
Nucleic acid-based cancer vaccines 229
DNA cancer vaccines 230
RNA vaccines 230
Viral vector-based cancer vaccines 230
Intradermal delivery of cancer vaccines by Ad vectors 231
Future prospects of cancer vaccines 231
Companies involved in nucleic acid-based cancer vaccines 231
Monoclonal antibody gene transfer for cancer 232
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