Gene Therapy - Technologies, Markets and Companies
NEW YORK, Feb. 1, 2012 /PRNewswire/ -- Reportlinker.com announces that a new market research report is available in its catalogue:
Gene Therapy - technologies, markets and companieshttp://www.reportlinker.com/p0203543/Gene-Therapy---technologies-markets-and-companies.html#utm_source=prnewswire&utm_medium=pr&utm_campaign=Biological_Therapy
Gene therapy can be broadly defined as the transfer of defined genetic material to specific target cells of a patient for the ultimate purpose of preventing or altering a particular disease state. Genes and DNA are now being introduced without the use of vectors and various techniques are being used to modify the function of genes in vivo without gene transfer. If one adds to this the cell therapy particularly with use of genetically modified cells, the scope of gene therapy becomes much broader. Gene therapy can now combined with antisense techniques such as RNA interference (RNAi), further increasing the therapeutic applications. This report takes broad overview of gene therapy and is the most up-to-date presentation from the author on this topic built-up from a series of gene therapy report written by him during the past decade including a textbook of gene therapy and a book on gene therapy companies. This report describes the setbacks of gene therapy and renewed interest in the topic
Gene therapy technologies are described in detail including viral vectors, nonviral vectors and cell therapy with genetically modified vectors. Gene therapy is an excellent method of drug delivery and various routes of administration as well as targeted gene therapy are described. There is an introduction to technologies for gene suppression as well as molecular diagnostics to detect and monitor gene expression.
Clinical applications of gene therapy are extensive and cover most systems and their disorders. Full chapters are devoted to genetic syndromes, cancer, cardiovascular diseases, neurological disorders and viral infections with emphasis on AIDS. Applications of gene therapy in veterinary medicine, particularly for treating cats and dogs, are included.
Research and development is in progress in both the academic and the industrial sectors. The National Institutes of Health (NIH) of the US is playing an important part. As of 2011, over 2030 clinical trials have been completed, are ongoing or have been approved worldwide.A breakdown of these trials is shown according to the areas of application.
Since the death of Jesse Gelsinger in the US following a gene therapy treatment, the FDA has further tightened the regulatory control on gene therapy. A further setback was the reports of leukemia following use of retroviral vectors in successful gene therapy for adenosine deaminase deficiency. Several clinical trials were put on hold and many have resumed now. The report also discusses the adverse effects of various vectors, safety regulations and ethical aspects of gene therapy including germline gene therapy.
The markets for gene therapy are difficult to estimate as there is only one approved gene therapy product and it is marketed in China since 2004. Gene therapy markets are estimated for the years 2011-2021. The estimates are based on epidemiology of diseases to be treated with gene therapy, the portion of those who will be eligible for these treatments, competing technologies and the technical developments anticipated in the next decades. In spite of some setbacks, the future for gene therapy is bright.The markets for DNA vaccines are calculated separately as only genetically modified vaccines and those using viral vectors are included in the gene therapy markets
The voluminous literature on gene therapy was reviewed and selected 700 references are appended in the bibliography.The references are constantly updated. The text is supplemented with 72 tables and 13 figures.
Profiles of 186 companies involved in developing gene therapy are presented along with 188 collaborations. There were only 44 companies involved in this area in 1995. In spite of some failures and mergers, the number of companies has increased more than 4-fold within a decade. These companies have been followed up since they were the topic of a book on gene therapy companies by the author of this report. John Wiley & Sons published the book in 2000 and from 2001 to 2003, updated versions of these companies (approximately 160 at mid-2003) were available on Wiley's web site. Since that free service was discontinued and the rights reverted to the author, this report remains the only authorized continuously updated version on gene therapy companies.
TABLE OF CONTENTS1. Introduction 21
Definitions 21
Historical evolution of gene therapy 21
Relation of gene therapy to other biotechnologies 23
Molecular biological basics for gene therapy 23
Genome 23
DNA 24
RNA 24
Alternative RNA splicing 25
Genes 26
Gene regulation 26
Gene expression 28
Chromosomes 28
Telomeres 29
Mitochondrial DNA 29
Proteins 30
2. Gene Therapy Technologies 31
Classification of gene therapy techniques 31
Ex vivo and in vivo gene therapy 32
Ex vivo gene therapy 32
In vivo gene therapy 33
Physical methods of gene transfer 33
Electroporation 33
Applications of electroporation 34
Clinical applications of electroporation 35
Advantages of electroporation 35
Limitations of electroporation 36
Hydrodynamic 36
Microinjection 36
Particle bombardment 37
Ultrasound-mediated transfection 39
Molecular vibration 39
Application of pulsed magnetic field and superparamagnetic nanoparticles 39
Gene transfection using laser irradiation 40
Photochemical transfection 40
Chemical methods of gene transfer 41
Gene repair and replacement 41
Gene repair by single-stranded oligonucleotides 41
History and current status of chimeraplasty 42
mRNA gene therapy 42
Spliceosome mediated RNA trans-splicing 42
Vectors for gene therapy 43
Basic considerations 43
Use of genes as pharmaceuticals 44
The ideal vector for gene therapy 44
Viral vectors 45
Adenovirus vectors 46
Adeno-associated virus vectors 48
Alphavirus vectors 50
Baculovirus vectors 50
Foamy virus vectors 51
Herpes simplex virus vectors 51
Lentiviral vectors 53
Multicistronic retroviral vectors 54
Retroviral vectors 55
Oncogenic potential of retroviral vectors 56
Future prospects of viral vectors 57
Companies using viral vectors 57
Nonviral vectors for gene therapy 59
Anionic lipid-DNA complexes 59
Cationic lipid-DNA complexes 60
Effects of shape of DNA molecules on delivery with nonviral vectors 60
Electrostatic modifications of surface to improve gene delivery 60
Liposomes for gene therapy 61
Liposome-nucleic acid complexes 62
Liposome-HVJ complex 63
Transposons DNA vectors 63
Polycation-DNA complexes (polyplexes) 64
Plasmid DNA vector for treatment of chronic inflammatory disease 65
Polymer molecules 65
Synthetic biology and DNA vectors 65
Synthetic peptide complexes 66
Future prospects of nonviral vs viral vectors 66
Nanobiotechnology for gene therapy 66
Antisense nanoparticles for gene regulation 67
Biological nanoparticle technology 67
Dendrimers 67
Cochleates 67
Calcium phosphate nanoparticles as nonviral vectors 68
Gelatin nanoparticles for gene delivery 68
Lipid nanoparticles for nucleic acid delivery 69
Nanoparticles as nonviral vectors for gene therapy 69
Nanoparticles with virus-like function as gene therapy vectors 70
Nanobiolistics for nucleic acid delivery 70
Nonionic polymeric micelles for oral gene delivery 70
Silica nanoparticles as a nonviral vector for gene delivery 71
Receptor-mediated endocytosis 71
Artificial viral vectors 72
Directed evolution of AAV to create efficient gene delivery vectors 73
Bacterial ghosts as DNA delivery systems 73
Bacteria plus nanoparticles for gene delivery into cells 73
Chromosome-based vectors for gene therapy 74
Mammalian artificial chromosomes (MACs) 75
Artificial Chromosome Expression (ACE) 75
Human artificial chromosomes (HACs) 75
?C31 integrase system 76
Companies using nonviral vectors 76
Concluding remarks about vectors 77
Cell-mediated gene therapy 78
Fibroblasts 79
Skeletal muscle cells 79
Vascular smooth muscle cells 80
Keratinocytes 80
Hepatocytes 80
Lymphocytes 81
Regulating protein delivery by genetically encoded lymphocytes 81
Implantation of microencapulated genetically modified cells 81
Stem cell gene therapy 82
Therapeutic applications for hematopoietic stem cell gene transfer 82
Improving delivery of genes to stem cells 83
Lentiviral vectors for gene transfer to marrow stem cells 83
Use of mesenchymal stem cells for gene therapy 83
Microporation for transfection of MSCs 83
In utero gene therapy using stem cells 84
Gene delivery to stem cells by artificial chromosome expression 84
Linker based sperm-mediated gene transfer technology 84
Combination of gene therapy with therapeutic cloning 84
Expansion of transduced HSCs in vivo 85
The future of hematopoietic stem cell gene therapy 85
Routes of administration for gene therapy 85
Direct injection of naked DNA 86
Intramuscular injection 86
Intravenous DNA injection 86
Intraarterial delivery 87
Companies with gene delivery devices/ technologies 87
Targeted gene therapy 88
Targeted integration 88
Bacteriophage integrase system for site-specific gene delivery 89
Controlled-release delivery of DNA 89
Controlled gene therapy 90
Controlled delivery of genetic material 90
Controlled induction of gene expression 90
Drug-inducible systems for control of gene expression 91
Timed activation of gene therapy by a circuit based on signaling network 91
Small molecules for post-transcriptional regulation of gene expression 91
Engineered zinc finger DNA binding proteins for gene correction 92
Light Activated Gene Therapy 92
Spatial control of gene expression via local hyperthermia 92
Companies with regulated /targeted gene therapy 93
Gene marking 94
Germline gene therapy 94
Potential applications of human germline genome modification 94
Pros and cons of human germline genome modification 95
Role of gene transfer in antibody therapy 96
Genetically engineered vaccines 96
DNA vaccines 97
DNA inoculation technology 97
Methods for enhancing the potency of DNA vaccines 98
Advantages of DNA vaccines 98
Vaccine vectors 98
Challenges and limitations of genetically engineered vaccines 99
Vaccines based on reverse genetics 100
Technologies for gene suppression 100
Antisense oligonucleotides 100
Transcription factor decoys 101
Aptamers 102
Ribozymes 102
Peptide nucleic acid 102
Intracellular delivery of PNAs 102
Locked nucleic acid 103
Zorro-LNA 103
Gene silencing 103
Post-transcriptional gene silencing 104
Definitions and terminology of RNAi 104
RNAi mechanisms 104
Inhibition of gene expression by antigene RNA 105
RNAi gene therapy 106
microRNA gene therapy 106
Application of molecular diagnostic methods in gene therapy 106
Use of PCR to study biodistribution of gene therapy vector 107
PCR for verification of the transcription of DNA 107
In situ PCR for direct quantification of gene transfer into cells 107
Detection of retroviruses by reverse transcriptase (RT)-PCR 108
Confirmation of viral vector integration 108
Monitoring of gene expression 108
Monitoring of gene expression by green fluorescent protein 108
Monitoring in vivo gene expression by molecular imaging 109
Advantages of gene therapy compared with protein therapy 109
3. Clinical Applications of Gene Therapy 111
Introduction 111
Bone and joint disorders 111
Bone fractures 111
Gene therapy for intervertebral disc degeneration 112
Spinal fusion 112
Osteogenesis imperfecta 113
Rheumatoid arthritis 113
Local or systemic treatment 114
In vivo or ex vivo gene therapy of RA 114
Clinical trials 115
Gene therapy for osteoarthritis 116
Sports injuries 117
Repair of articular cartilage defects 117
Regeneration and replacement of bone by gene therapy 118
Bacterial infections 119
Antisense approach to bacterial infections 119
Dentistry 119
Tissue engineering in dental implant defects 119
Endocrine and metabolic disorders 120
Introduction 120
Gene therapy of obesity 120
Ad viral vector-mediated transfer of leptin gene 120
AAV vector-mediated delivery of GDNF for obesity 121
Diabetes mellitus 121
Methods of gene therapy of diabetes mellitus 122
Viral vector-mediated gene transfer in diabetes 122
Gene delivery with ultrasonic microbubble destruction technology 123
Genetically engineered cells for diabetes mellitus 123
Genetically altered liver cells 124
Genetically modified stem cells 124
Genetically engineered dendritic cells 124
Insertion of gene encoding for IL-4 124
Leptin gene therapy 125
Concluding remarks about cell and gene therapy of diabetes 125
Gene therapy of growth-hormone deficiency 126
Gastrointestinal disorders 126
Introduction 126
Methods of gene transfer to the gastrointestinal tract 127
Direct delivery of genes 127
Naked plasmid DNA into the submucosa 127
Viral vectors 127
Receptor-mediated endocytosis 128
Indications for gastrointestinal gene therapy 128
Gene therapy for inflammatory disorders of the bowel 128
Gene transfer to the salivary glands 129
Potential clinical applications of salivary gene therapy 130
Hematology 130
Hemophilias 130
Gene therapy of hemophilia 131
Hemophilia A 131
Hemophilia B 132
Concluding remarks about gene therapy of hemophilias 133
Hemoglobinopathies 133
Stem cell-based gene therapy and RNAi for sickle cell disease 134
Gene therapy for ?-thalassemia 135
Gene therapy of Fanconi's anemia 136
Acquired hematopoietic disorders 136
Chronic acquired anemias 137
Neutropenia 137
Thrombocytopenia 138
Concluding remarks about gene therapy of hemoglobinopathies 139
Companies involved in gene thery of hematological disorders 139
In utero/fetal gene therapy 139
Fetal gene transfer techniques 140
Animal models of fetal gene therapy 141
Potential applications of fetal gene therapy 141
Fetal gene therapy for cystic fibrosis 141
Fetal intestinal gene therapy 142
Hearing disorders 142
Potential of gene therapy 142
Vectors for gene therapy of hearing disorders 143
Auditory hair cell replacement and hearing improvement by gene therapy 143
Kidney diseases 144
End-stage renal disease 144
Methods of gene delivery to the kidney 144
Gene transfer into kidney by adenoviral vectors 145
Non-viral gene transfer to the kidneys 145
Gene transfer into the glomerulus by HVJ-liposome 145
Bone marrow stem cells for renal disease 145
Mesangial cell therapy 146
Liposome-mediated gene transfer into the tubules 146
Gene transfer to tubules with cationic polymer polyethylenimine 146
Gene therapy in animal experimental models of renal disease 147
Genetic manipulations of the embryonic kidney 147
Antisense intervention in glomerulonephritis 147
Gene therapy for renal fibrosis 148
Use of genetically engineered cells for uremia due to renal failure 148
Concluding remarks 149
Liver disorders 149
Techniques of gene delivery to liver 150
Direct injection of DNA into liver 150
Local gene delivery by isolated organ perfusion 150
Liposome-mediated direct gene transfer 150
Retroviral vector for gene transfer to liver 151
Adenoviral vectors for gene transfer to liver 151
Receptor-mediated approach 151
Cell therapy for liver disorders 151
Transplantation of genetically modified hepatocytes 152
Genetically modified hematopoietic stem cells 152
Gene therapy by ex vivo transduced liver progenitor cells 152
Gene therapy of genetic diseases affecting the liver 153
Crigler-Najjar syndrome 153
Hereditary tyrosinemia type I (HT1) 153
Hereditary tyrosinemia type 3 153
Gene therapy of acquired diseases affecting the liver 154
Cirrhosis of liver 154
Ophthalmic disorders 154
Introduction to gene therapy of ophthalmic disorders 154
Degenerative retinal disorders 155
Age-related macular degeneration 155
Inherited retinal degenerations 157
Inherited disorders affecting vision 157
Gene therapy for color blindness 157
Leber congenital amaurosis 158
Retinitis pigmentosa 159
Stargardt disease 160
Usher syndrome 160
X-linked juvenile retinoschisis 160
Proliferative retinopathies 161
Methods of gene transfer to retinal cells 161
DNA nanoparticles for nonviral gene transfer to the eye 162
Prevention of complications associated with eye surgery 162
Prevention of proliferative retinopathy by gene therapy 162
DNA nanoparticles for gene therapy of retinal degenerative disorders 163
Posterior capsule opacification after cataract surgery 163
Autoimmune uveitis 163
Retinal ischemic injury 164
Corneal disorders 164
Glaucoma 165
Disorders of hearing 165
Gene therapy for hearing loss 165
Organ transplantation 166
Introduction 166
DNA vaccines for transplantation 166
Gene therapy for prolonging allograft survival 166
Gene therapy in lung transplantation 167
Role of gene therapy in liver transplantation 167
Gene therapy in kidney transplantation 167
Veto cells and transplant tolerance 168
Pulmonary disorders 168
Techniques of gene delivery to the lungs 169
Adenoviral vectors 169
Non-viral vectors 170
Aerosolization as an aid to gene transfer to lungs. 170
Cystic fibrosis 170
Genetics and clinical features 170
Gene therapy for CF 171
CFTR gene transfer in CF 171
Concluding remarks about gene therapy of CF 172
Miscellaneous pulmonary disorders 173
Gene therapy for pulmonary arterial hypertension 173
Gene therapy for bleomycin-induced pulmonary fibrosis 174
Pulmonary complications of a1-antitrypsin deficiency 174
Gene therapy for asthma 175
Gene therapy for adult respiratory distress syndrome 176
Gene therapy for lung injury 176
Gene therapy for bronchopulmonary dysplasia 176
Concluding remarks about gene therapy of lungs 177
Companies involved in pulmonary gene therapy 177
Skin and soft tissue disorders 178
Gene transfer to the skin 178
Electroporation for transdermal delivery of plasmid DNA 178
Electroporation for transdermal delivery of DNA vaccines 178
Liposomes for transdermal gene delivery 179
Ultrasound and topical gene therapy 179
Gene therapy in skin disorders 179
Gene therapy of hair loss 179
Gene therapy for xeroderma pigmentosa 180
Gene therapy for lamellar ichthyosis 180
Gene therapy for epidermolysis bullosa 180
Gene transfer techniques for wound healing 181
Urogenital disorders 182
Gene therapy for urinary tract dysfunction 182
Gene therapy for erectile dysfunction 182
NOS gene transfer for erectile dysfunction 182
Clinical trial of hMaxi-K Gene transfer in erectile dysfunction 182
Gene therapy for erectile dysfunction due to nerve injury 183
Concluding remarks on gene therapy for erectile dysfunction 183
Veterinary gene therapy 183
Gene therapy for mucopolysaccharidosis VII in dogs 184
Gene therapy to increase disease resistance 184
Gene therapy for infections 184
Gene therapy for chronic anemia 185
Gene therapy for endocrine disorders 185
Gene therapy for arthritis 185
Cancer gene therapy 186
Brain tumors in cats and dogs 186
Breast cancer in dogs 187
Canine hemangiosarcoma 187
Canine melanoma 188
Canine soft tissue sarcoma 188
Melanoma in horses 188
4. Gene Therapy of Genetic Disorders 189
Introduction 189
Primary immunodeficiency disorders 190
Severe combined immune deficiency 191
Chronic granulomatous disease 193
Wiskott-Aldrich syndrome 193
Purine nucleoside phosphorylase deficiency 194
Major histocompatibility class II deficiency 194
Future prospects of gene therapy of inherited immunodeficiencies 195
Metabolic disorders 195
Adrenoleukodystrophy 196
Canavan disease 196
Lesch-Nyhan syndrome 197
LPL deficiency 197
Ornithine transcarbamylase deficiency 198
Phenylketonuria 198
Porphyrias 199
Tetrahydrobiopterin deficiency 199
Lysosomal storage disorders. 200
Batten disease 201
Fabry's disease 201
Farber's disease 202
Gaucher disease 202
Animals models of Gaucher's disease 202
Gene therapy of Gaucher's disease 203
Hunter syndrome 204
Combination of cell and gene therapy for Krabbe's disease 204
Metachromatic leukodystrophy 205
Mucopolysaccharidosis type 1 (Hurler syndrome) 205
Niemann-Pick type A disease 206
Pompe disease 206
Sanfilippo A syndrome 207
Sly syndrome 207
Tay-Sachs disease 207
Future prospects of gene therapy of lysosomal storage disorders 208
Trinucleotide repeat disorders 208
Muscular dystrophies 208
Duchenne muscular dystrophy (DMD) 208
Animal models for gene therapy of DMD 209
Antisense approach to DMD 209
Exon-skipping technology for DMD 210
Liposome-mediated gene transfer 210
Myoblast-based gene transfer in DMD 211
Plasmid-mediated gene therapy 211
Post-transcriptional modulation of gene expression in DMD 211
Repair of dystrophin gene 212
Routes of administration of gene therapy in DMD 212
Types of dystrophin constructs 212
Viral vectors for DMD 213
Conclusions and future prospects of gene therapy of DMD 214
Limb-girdle muscular dystrophy 215
Myotonic dystrophy 215
Spinal muscular atrophy 216
Antisense gene therapy of SMA 216
Hereditary neuropathies 216
Charcot-Marie-Tooth disease 216
Hereditary axonal neuropathies of the peripheral nerves 217
Gene therapy of mitochondrial disorders 217
Companies involved in gene therapy of genetic disorders 218
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
Radioprotective gene therapy 223
Nanoparticles to facilitate combination of hyperthermia and gene therapy 223
Cell-based cancer gene therapy 223
Adoptive cell therapy 224
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 230
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
Transfer and expression of intracellular adhesion-1 molecules 233
Other gene-based techniques of immunotherapy of cancer 233
Fas (Apo-1) 233
Chemokines 233
Major Histocompatibility Complex (MHC) Class I 234
IGF (Insulin-Like Growth Factor) 234
Inhibition of immunosuppressive function in cancer 234
Delivery of toxic genes to tumor cells for eradication 235
Gene-directed enzyme prodrug therapy 235
Combination of gene therapy with radiotherapy 235
Correction of genetic defects in cancer cells 236
Targeted gene therapy for cancer 236
Antiangiogenic therapy for cancer 236
Bacteria as novel anticancer gene vectors 237
Cancer-specific gene expression 238
Cancer-specific transcription 238
Delivery of retroviral particles hitchhiking on T cells 238
Electrogene and electrochemotherapy 239
Epidermal growth factor-mediated DNA delivery 239
Gene-based targeted drug delivery to tumors 239
Gene expression in hypoxic tumor cells 240
Genetically modified T cells for targeting tumors 240
Genetically engineered stem cells for targeting tumors 241
Hematopoietic stem cells for targeted cancer gene therapy 242
Immunolipoplex for delivery of p53 gene 243
Nanomagnets for targeted cell-based cancer gene therapy 243
Nanoparticles for targeted site-specific delivery of anticancer genes 243
Targeted cancer therapy using a dendrimer-based synthetic vector 244
Tumor-targeted gene therapy by receptor-mediated endocytosis 244
Virus-mediated oncolysis 244
Cancer terminator virus 244
Cytokine-induced killer cells for delivery of an oncolytic virus 245
Monitoring of viral-mediated oncolysis by PET 246
Oncolytic HSV 246
Oncolytic adenoviruses 246
Oncolytic vesicular stomatitis virus 248
Oncolytic paramyxovirus 248
Oncolytic vaccinia virus 248
Targeted cancer treatments based on oncolytic viruses 248
Concluding remarks on oncolytic gene therapy 249
Companies developing oncolytic viruses 249
Apoptotic approach to improve cancer gene therapy 250
Tumor suppressor gene therapy 250
P53 gene therapy 250
BRIT1 gene therapy 251
Nitric oxide-based cancer gene therapy 251
Nitric oxide synthase II DNA injection 251
Gene therapy for radiosensitization of cancer 251
Gene therapy of cancer of selected organs 252
Gene therapy for bladder cancer 252
Gene therapy for glioblastoma multiforme. 252
Adenoviral vectors for treatment of brain tumors 254
Antiangiogenic gene therapy 254
Autophagy induced by conditionally replicating adenoviruses 255
Baculovirus vector for diphtheria toxin gene therapy 255
Cerepro® (sitimagene ceradenovec) 255
Gene therapy targeting hepatocyte growth factor 256
Genetically engineered MSCs for gene delivery to intracranial gliomas 256
Intravenous gene delivery with nanoparticles into brain tumors 256
Ligand-directed delivery of dsRNA molecules targeted to EGFR 256
RNAi gene therapy of brain cancer 257
Targeting normal brain cells with an AAV vector encoding interferon-? 257
Viral oncolysis of brain tumors 258
Gene therapy for breast cancer 258
Gene vaccine for breast cancer 259
Recombinant adenoviral ErbB-2/neu vaccine 259
Gene Therapy for ovarian cancer 260
Gene therapy for malignant melanoma 261
Gene therapy of lung cancer 263
Intravenous nanoparticle formulation for delivery of FUS1 gene 263
Aerosol gene delivery for lung cancer 263
Gene therapy for cancer of prostate 264
Experimental studies 264
Nanoparticle-based gene therapy for prostate cancer 264
Tumor suppressor gene therapy in prostate cancer 264
Vaccines for prostate cancer 265
Clinical trials 265
Gene therapy of head and neck cancer 266
Adenoviral vector based P53 gene therapy 266
Gene therapy of pancreatic cancer 266
Rexin-G? for targeted gene delivery in cancer 267
Targeted Expression of BikDD gene 267
Concluding remarks on gene therapy of pancreatic cancer 267
Cancer gene therapy companies 267
6. Gene Therapy of Neurological Disorders 271
Indications 271
Gene transfer techniques for the nervous system 272
Methods of gene transfer to the nervous system 272
Ideal vector for gene therapy of neurological disorders 272
Promoters of gene transfer 272
Lentivirus-mediated gene transfer to the CNS 273
AAV vector mediated gene therapy for neurogenetic disorders 273
Gene transfer to the CNS using recombinant SV40-derived vectors 274
Routes of delivery of genes to the CNS 274
Direct injection into CNS 274
Introduction of the genes into cerebral circulation 275
Introduction of genes into cerebrospinal fluid 275
Intravenous administration of vectors 275
Delivery of gene therapy to the peripheral nervous system 276
Cell-mediated gene therapy of neurological disorders 276
Neuronal cells 276
Neural stem cells and progenitor cells 276
Astrocytes 277
Cerebral endothelial cells 277
Implantation of genetically modified encapsulated cells into the brain 277
Gene therapy of neurodegenerative disorders 277
Gene therapy for Parkinson disease 277
Rationale 278
Techniques of gene therapy for PD 279
Delivery of neurotrophic factors by gene therapy 282
Delivery of parkin gene 283
Introduction of functional genes into the brain of patients with PD 283
Nanoparticle-based gene therapy for PD 283
Mitochondrial gene therapy for PD 283
RNAi approach to PD 284
Prospects of gene therapy for PD 284
Companies developing gene therapy for PD 285
Gene therapy for Alzheimer disease 286
Rationale 286
NGF gene therapy for AD 286
FGF2 gene transfer in AD 287
Neprilysin gene therapy 288
Targeting plasminogen activator inhibitor type-1 gene 288
Gene vaccination 288
Combination of gene therapy with other treatments for AD 289
Gene therapy of Huntington disease 289
Encapsulated genetically engineered cellular implants 289
Viral vector mediated administration of neurotrophic factors 289
RNAi gene therapy 290
Gene therapy of amyotrophic lateral sclerosis 290
Rationale 290
Technique of gene therapy of ALS 290
Gene therapy of cerebrovascular diseases 291
Preclinical research in gene therapy for cerebrovascular disease 291
Animal models of stroke relevant to gene therapy 292
Transgenic mice as models for stroke 292
Animal models for gene therapy of arteriovenous malformations 292
Gene transfer to cerebral blood vessels 293
Gene therapy for vasospasm following subarachnoid hemorrhage 294
NOS gene therapy for cerebral vasospasm 294
Gene therapy for stroke 295
Gene therapy for stroke using neurotrophic factors 296
Gene therapy of strokes with a genetic component 296
Gene therapy for intracranial aneurysms 297
RNAi-based gene silencing for neuroprotection in cerebral ischemia 297
Concluding remarks about gene therapy for stroke 297
Gene therapy of injuries to the nervous system 298
Traumatic brain injury 298
Spinal cord injury 29
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Industry Analysis and InsightsNicolas Bombourg
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