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Gene Therapy - Technologies, Markets and Companies

NEW YORK, Nov. 30, 2011 /PRNewswire/ -- announces that a new market research report is available in its catalogue:

Gene Therapy - technologies, markets and companies


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 January 2010, over 2024 gene therapy 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 2010-2020. 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.


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 75

Mammalian artificial chromosomes (MACs) 76

Artificial Chromosome Expression (ACE) 76

Human artificial chromosomes (HACs) 76

?C31 integrase system 77

Companies using nonviral vectors 77

Concluding remarks about vectors 78

Cell-mediated gene therapy 79

Fibroblasts 79

Skeletal muscle cells 80

Vascular smooth muscle cells 81

Keratinocytes 81

Hepatocytes 81

Lymphocytes 82

Regulating protein delivery by genetically encoded lymphocytes 82

Implantation of microencapulated genetically modified cells 82

Stem cell gene therapy 83

Therapeutic applications for hematopoietic stem cell gene transfer 83

Improving delivery of genes to stem cells 83

Lentiviral vectors for gene transfer to marrow stem cells 84

Use of mesenchymal stem cells for gene therapy 84

Microporation for transfection of MSCs 84

In utero gene therapy using stem cells 84

Gene delivery to stem cells by artificial chromosome expression 85

Linker based sperm-mediated gene transfer technology 85

Combination of gene therapy with therapeutic cloning 85

Expansion of transduced HSCs in vivo 86

The future of hematopoietic stem cell gene therapy 86

Routes of administration for gene therapy 86

Direct injection of naked DNA 87

Intramuscular injection 87

Intravenous DNA injection 87

Intraarterial delivery 87

Companies with gene delivery devices/ technologies 88

Targeted gene therapy 89

Targeted integration 89

Bacteriophage integrase system for site-specific gene delivery 90

Controlled-release delivery of DNA 90

Controlled gene therapy 91

Controlled delivery of genetic material 91

Controlled induction of gene expression 91

Drug-inducible systems for control of gene expression 92

Timed activation of gene therapy by a circuit based on signaling network 92

Small molecules for post-transcriptional regulation of gene expression 92

Engineered zinc finger DNA binding proteins for gene correction 93

Light Activated Gene Therapy 93

Spatial control of gene expression via local hyperthermia 93

Companies with regulated /targeted gene therapy 94

Gene marking 95

Germline gene therapy 95

Potential applications of human germline genome modification 95

Pros and cons of human germline genome modification 96

Role of gene transfer in antibody therapy 97

Genetically engineered vaccines 97

DNA vaccines 98

DNA inoculation technology 98

Methods for enhancing the potency of DNA vaccines 99

Advantages of DNA vaccines 99

Vaccine vectors 99

Challenges and limitations of genetically engineered vaccines 100

Vaccines based on reverse genetics 101

Technologies for gene suppression 101

Antisense oligonucleotides 101

Transcription factor decoys 102

Aptamers 103

Ribozymes 103

Peptide nucleic acid 103

Intracellular delivery of PNAs 103

Locked nucleic acid 104

Zorro-LNA 104

Gene silencing 104

Post-transcriptional gene silencing 105

Definitions and terminology of RNAi 105

RNAi mechanisms 105

Inhibition of gene expression by antigene RNA 106

RNAi gene therapy 107

microRNA gene therapy 107

Application of molecular diagnostic methods in gene therapy 107

Use of PCR to study biodistribution of gene therapy vector 108

PCR for verification of the transcription of DNA 108

In situ PCR for direct quantification of gene transfer into cells 108

Detection of retroviruses by reverse transcriptase (RT)-PCR 109

Confirmation of viral vector integration 109

Monitoring of gene expression 109

Monitoring of gene expression by green fluorescent protein 109

Monitoring in vivo gene expression by molecular imaging 110

Advantages of gene therapy compared with protein therapy 110

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 133

Gene therapy for ?-thalassemia 134

Gene therapy of Fanconi's anemia 135

Acquired hematopoietic disorders 136

Chronic acquired anemias 136

Neutropenia 137

Thrombocytopenia 138

Concluding remarks about gene therapy of hemoglobinopathies 138

Companies involved in gene thery of hematological disorders 139

In utero/fetal gene therapy 139

Fetal gene transfer techniques 139

Animal models of fetal gene therapy 140

Potential applications of fetal gene therapy 140

Fetal gene therapy for cystic fibrosis 141

Fetal intestinal gene therapy 141

Hearing disorders 141

Potential of gene therapy 142

Vectors for gene therapy of hearing disorders 142

Auditory hair cell replacement and hearing improvement by gene therapy 143

Kidney diseases 143

End-stage renal disease 143

Methods of gene delivery to the kidney 144

Gene transfer into kidney by adenoviral vectors 144

Non-viral gene transfer to the kidneys 144

Gene transfer into the glomerulus by HVJ-liposome 145

Bone marrow stem cells for renal disease 145

Mesangial cell therapy 145

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 146

Genetic manipulations of the embryonic kidney 147

Antisense intervention in glomerulonephritis 147

Gene therapy for renal fibrosis 147

Use of genetically engineered cells for uremia due to renal failure 148

Concluding remarks 148

Liver disorders 148

Techniques of gene delivery to liver 149

Direct injection of DNA into liver 149

Local gene delivery by isolated organ perfusion 150

Liposome-mediated direct gene transfer 150

Retroviral vector for gene transfer to liver 150

Adenoviral vectors for gene transfer to liver 150

Receptor-mediated approach 151

Cell therapy for liver disorders 151

Transplantation of genetically modified hepatocytes 151

Genetically modified hematopoietic stem cells 152

Gene therapy by ex vivo transduced liver progenitor cells 152

Gene therapy of genetic diseases affecting the liver 152

Crigler-Najjar syndrome 152

Hereditary tyrosinemia type I (HT1) 153

Hereditary tyrosinemia type 3 153

Gene therapy of acquired diseases affecting the liver 153

Cirrhosis of liver 153

Ophthalmic disorders 154

Introduction to gene therapy of ophthalmic disorders 154

Degenerative retinal disorders 155

Age-related macular degeneration 155

Inherited retinal degenerations 156

Inherited disorders affecting vision 157

Gene therapy for color blindness 157

Leber congenital amaurosis 157

Retinitis pigmentosa 158

Stargardt disease 159

Usher syndrome 159

X-linked juvenile retinoschisis 160

Proliferative retinopathies 160

Methods of gene transfer to retinal cells 160

DNA nanoparticles for nonviral gene transfer to the eye 161

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 162

Posterior capsule opacification after cataract surgery 162

Autoimmune uveitis 162

Retinal ischemic injury 163

Corneal disorders 163

Glaucoma 164

Disorders of hearing 164

Gene therapy for hearing loss 164

Organ transplantation 165

Introduction 165

DNA vaccines for transplantation 165

Gene therapy for prolonging allograft survival 165

Gene therapy in lung transplantation 166

Role of gene therapy in liver transplantation 166

Gene therapy in kidney transplantation 166

Veto cells and transplant tolerance 167

Pulmonary disorders 167

Techniques of gene delivery to the lungs 168

Adenoviral vectors 168

Non-viral vectors 169

Aerosolization as an aid to gene transfer to lungs. 169

Cystic fibrosis 170

Genetics and clinical features 170

Gene therapy for CF 170

CFTR gene transfer in CF 170

Concluding remarks about gene therapy of CF 172

Miscellaneous pulmonary disorders 172

Gene therapy for pulmonary arterial hypertension 172

Gene therapy for bleomycin-induced pulmonary fibrosis 173

Pulmonary complications of a1-antitrypsin deficiency 173

Gene therapy for asthma 174

Gene therapy for adult respiratory distress syndrome 175

Gene therapy for lung injury 175

Gene therapy for bronchopulmonary dysplasia 175

Concluding remarks about gene therapy of lungs 176

Companies involved in pulmonary gene therapy 176

Skin and soft tissue disorders 177

Gene transfer to the skin 177

Electroporation for transdermal delivery of plasmid DNA 177

Electroporation for transdermal delivery of DNA vaccines 178

Liposomes for transdermal gene delivery 178

Ultrasound and topical gene therapy 178

Gene therapy in skin disorders 178

Gene therapy of hair loss 179

Gene therapy for xeroderma pigmentosa 179

Gene therapy for lamellar ichthyosis 179

Gene therapy for epidermolysis bullosa 180

Gene transfer techniques for wound healing 180

Urogenital disorders 181

Gene therapy for urinary tract dysfunction 181

Gene therapy for erectile dysfunction 181

NOS gene transfer for erectile dysfunction 181

Clinical trial of hMaxi-K Gene transfer in erectile dysfunction 182

Gene therapy for erectile dysfunction due to nerve injury 182

Concluding remarks on gene therapy for erectile dysfunction 182

Veterinary gene therapy 183

Gene therapy for mucopolysaccharidosis VII in dogs 183

Gene therapy to increase disease resistance 183

Gene therapy for infections 184

Gene therapy for chronic anemia 184

Gene therapy for endocrine disorders 185

Gene therapy for arthritis 185

Cancer gene therapy 185

Brain tumors in cats and dogs 185

Breast cancer in dogs 186

Canine hemangiosarcoma 187

Canine melanoma 187

Canine soft tissue sarcoma 187

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 298

Gene therapy of epilepsy 299

Gene therapy for control of seizures 299

Gene therapy for neuroprotection in epilepsy 300

Gene therapy for genetic forms of epilepsy 301

Gene therapy for multiple sclerosis 301

Gene therapy for relief of pain 302

Rationale of gene therapy for pain 302

Vectors for gene therapy of pain 302

Methods of gene delivery for pain 302

Endogenous analgesic production for cranial neuralgias 303

Gene delivery by intrathecal route 303

Gene transfer for delivery of analgesics to the spinal nerve roots 304

Gene therapy of peripheral neuropathic pain 305

Gene transfer by injections into the brain substance 305

Targets for gene therapy of pain 306

Zinc finger DNA-binding protein therapeutic for chronic pain 306

Gene therapy for producing enkephalin to block pain signals 306

Targeting nuclear factor-?B 306

Gene therapy targeted to neuroimmune component of chronic pain 306

Potential applications of gene therapy for management of pain 307

Concluding remarks on gene therapy for pain 307

Gene therapy for psychiatric disorders 308

Gene therapy for depression 309

Gene therapy for enhancing cognition after stress 309

Gene therapy against fear disorders 309

Companies involved in gene therapy of neurological disorders 310

7. Gene Therapy of Cardiovascular Disorders 311

Introduction 311

Techniques of gene transfer to the cardiovascular system 311

Direct plasmid injection into the myocardium 312

Catheter-based systems for vector delivery 312

Ultrasound microbubbles for cardiovascular gene delivery 313

Vectors for cardiovascular gene therapy 313

Adenoviral vectors for cardiovascular diseases 313

Plasmid DNA-based delivery in cardiovascular disorders 313

Intravenous rAAV vectors for targeted delivery to the heart 314

Hypoxia-regulated gene therapy for myocardial ischemia 314

Angiogenesis and gene therapy of ischemic disorders 314

Therapeutic angiogenesis vs vascular growth factor therapy 315

Gene painting for delivery of targeted gene therapy to the heart 315

Gene delivery to vascular endothelium 316

Targeted plasmid DNA delivery to the cardiovascular system with nanoparticles 316

Vascular stents for gene delivery 316

Gene therapy for genetic cardiovascular disorders 317

Genetic disorders predisposing to atherosclerosis 317

Familial hypercholesterolemia (FH) 317

Apolipoprotein E (apoE) deficiency 319

Hypertension 319

Genetic factors for myocardial infarction 320

Acquired cardiovascular diseases 320

Coronary artery disease with angina pectoris 320

Ad5FGF-4 320

Ischemic heart disease with myocardial infarction 321

Myocardial repair with IGF-1 therapy 322

Metalloproteinase-2 inhibitor gene therapy 323

miRNA gene therapy for ischemic heart disease 323

Congestive heart failure 323

Rationale of gene therapy in CHF 323

?-ARKct gene therapy 324

Intracoronary adenovirus-mediated gene therapy for CHF 324

AAV-mediated gene transfer for CHF 325

AngioCell gene therapy for CHF 325

nNOS gene transfer in CHF 326

Cardiomyopathies 326

Cardiac conduction disturbances 326

Gene transfer approaches for biological pacemakers 326

Genetically engineered biological pacemakers 327

Gene therapy and heart transplantation 327

Peripheral arterial disease 328

Incidence and clinical features 328

Current management 328

Gene therapy for peripheral arterial disease 329

Angiogenesis by gene therapy 329

HIF-1? gene therapy for peripheral arterial disease 329

HGF gene therapy for peripheral arterial disease 330

Ischemic neuropathy secondary to peripheral arterial disease 330

Prevention of restenosis after angioplasty 330

Antisense approaches 331

Gene therapy to prevent restenosis after angioplasty 331

Techniques of gene therapy for restenosis 332

NOS gene therapy for restenosis 333

hTIMP-1 gene therapy to prevent intimal hyperplasia 334

Maintaining vascular patency after surgery 334

Companies involved in gene therapy of cardiovascular diseases 334

Future prospects of gene therapy of cardiovascular disorders 335

8. Gene therapy of viral infections 338

Introduction 338

Acquired Immunodeficiency Syndrome (AIDS) 338

Current management of AIDS 338

Gene therapy strategies in HIV/AIDS 339

HIV/AIDS vaccines 339

Insertion of protective genes into target cells. 340

Cell/gene therapies for HIV/AIDS 341

Transplantation of genetically modified T-cells 341

Transplantation of genetically modified hematopoietic cells 341

Anti-HIV ribozyme delivered in hematopoietic progenitor cells 342

Inhibition of HIV-1 replication by lentiviral vectors 342

VRX496 342

Intracellular immunization 343

Engineered cellular proteins such as soluble CD4s 343

Intracellular antibodies 343

Anti-rev single chain antibody fragment 343

Use of genes to chemosensitize HIV-1 infected cells 344

Autocrine interferon (INF)-? production by somatic cell gene therapy 344

Antisense approaches to AIDS 344

RNA decoys 344

Antisense oligodeoxynucleotides 344

RNA decoys 345

Ribozymes 345

RNAi applications in HIV/AIDS 346

siRNA-directed inhibition of HIV-1 infection 346

Role of the nef gene during HIV-1 infection and RNAi 346

Bispecific siRNA constructs 347

Targeting CXCR4 with siRNAs 347

Targeting CCR5 with siRNAs 347

Companies involved in developing gene therapy for HIV/AIDS 348

Conclusions regarding gene therapy of HIV/AIDS 349

Genetic vaccines for other viral infections 349

Cytomegalic virus infections 349

Viral hepatitis 350

Vaccine for hepatitis B virus 350

Vaccine for hepatitis C virus 351

Vaccine for herpes simplex virus 351

DNA vaccine against rabies 351

DNA vaccine for Ebola 352

Vaccines for avian influenza 352

Future prospects of DNA vaccines for avian influenza 353

Human trial of a DNA vaccine for avian influenza 354

Companies developing genetic vaccines for infections other than AIDS 354

9. Research, Development and Future of Gene Therapy 356

Basic research in gene therapy 356

R & D in gene therapy 356

Animal models of human diseases for gene therapy research 357

Lentiviral transgenesis 357

Financing research and development 357

Role of the NIH in gene therapy research 357

National Gene Vector Laboratories 357

Financing by the industry 358

Clinical trials in gene therapy 358

Clinical trials worldwide 359

Clinical trials in cancer gene therapy 359

Clinical trials in cardiovascular gene therapy 360

Clinical trials in inherited monogenic diseases 360

Clinical trials for other indications 360

Clinical trials i

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Biological Therapy Industry: Gene Therapy - technologies, markets and companies

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Nicolas Bombourg
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