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 companies

http://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 CONTENTS 0. Executive Summary 19

1. 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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