Reportlinker Adds Gene Therapy - technologies, markets and companies
NEW YORK, Dec. 21, 2010 /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
Benefits of this report
- Up-to-date on-stop information on gene therapy with 72 tables and 13 figures
- Evaluation of gene therapy technologies
- 700 selected references from the literature
- Estimates of gene therapy markets from 2009-2019
- Profiles of 189 companies involved and collaborations in this area
Who should read this report?
- Biotechnology companies developing gene therapy
- Academic institutions doing research in gene therapy
- Drug delivery companies
- Pharmaceutical companies interested in gene therapy
- Gene therapy companies
- Venture capital and investment companies
Summary
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 2009-2019. 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 189 companies involved in developing gene therapy are presented along with 210 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 40
Application of pulsed magnetic field and superparamagnetic nanoparticles 40
Gene transfection using laser irradiation 40
Photochemical transfection 41
Chemical methods of gene transfer 41
Gene repair and replacement 41
Gene repair by single-stranded oligonucleotides 42
History and current status of chimeraplasty 42
mRNA gene therapy 42
Spliceosome mediated RNA trans-splicing 43
Vectors for gene therapy 44
Basic considerations 44
Use of genes as pharmaceuticals 44
The ideal vector for gene therapy 45
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
Nonionic polymeric micelles for oral gene delivery 70
Silica nanoparticles as a nonviral vector for gene delivery 70
Receptor-mediated endocytosis 71
Artificial viral vectors 72
Directed evolution of AAV to create efficient gene delivery vectors 72
Bacterial ghosts as DNA delivery systems 73
Bacteria plus nanoparticles for gene delivery into cells 73
Chromosome-based vectors for gene therapy 74
Companies using nonviral vectors 76
Concluding remarks about vectors 77
Cell-mediated gene therapy 78
Fibroblasts 78
Skeletal muscle cells 79
Vascular smooth muscle cells 79
Keratinocytes 80
Hepatocytes 80
Lymphocytes 80
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 82
Lentiviral vectors for gene transfer to marrow stem cells 82
Use of mesenchymal stem cells for gene therapy 83
Microporation for transfection of MSCs 83
In utero gene therapy using stem cells 83
Gene delivery to stem cells by artificial chromosome expression 83
Linker based sperm-mediated gene transfer technology 84
Combination of gene therapy with therapeutic cloning 84
Expansion of transduced HSCs in vivo 84
The future of hematopoietic stem cell gene therapy 85
Routes of administration for gene therapy 85
Direct injection of naked DNA 85
Intramuscular injection 86
Intravenous DNA injection 86
Intraarterial delivery 86
Companies with gene delivery devices/ technologies 86
Targeted gene therapy 88
Targeted integration 88
Bacteriophage integrase system for site-specific gene delivery 88
Controlled-release delivery of DNA 89
Controlled gene therapy 89
Controlled delivery of genetic material 89
Controlled induction of gene expression 90
Drug-inducible systems for control of gene expression 90
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 92
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 96
DNA inoculation technology 97
Methods for enhancing the potency of DNA vaccines 97
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 101
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 103
Definitions and terminology of RNAi 104
RNAi mechanisms 104
Inhibition of gene expression by antigene RNA 105
RNAi gene therapy 106
Application of molecular diagnostic methods in gene therapy 106
Use of PCR to study biodistribution of gene therapy vector 106
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 107
Confirmation of viral vector integration 107
Monitoring of gene expression 108
Monitoring of gene expression by green fluorescent protein 108
Monitoring in vivo gene expression by molecular imaging 108
Advantages of gene therapy compared with protein therapy 108
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 121
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 123
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 125
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 127
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 129
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 b-thalassemia 134
Gene therapy of Fanconi's anemia 135
Acquired hematopoietic disorders 136
Chronic acquired anemias 136
Neutropenia 137
Thrombocytopenia 137
Concluding remarks about gene therapy of hemoglobinopathies 138
Companies involved in gene therapy of hematological disorders 139
In utero 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
Ornithine transcarbamylase deficiency 197
Phenylketonuria 198
Porphyrias 198
Tetrahydrobiopterin deficiency 199
Lysosomal storage disorders. 199
Batten disease 200
Fabry's disease 201
Farber's disease 201
Gaucher disease 202
Animals models of Gaucher's disease 202
Gene therapy of Gaucher's disease 202
Hunter syndrome 203
Combination of cell and gene therapy for Krabbe's disease 203
Metachromatic leukodystrophy 204
Mucopolysaccharidosis type 1 (Hurler syndrome) 205
Niemann-Pick type A disease 205
Pompe disease 205
Sanfilippo A syndrome 206
Sly syndrome 206
Tay-Sachs disease 207
Future prospects of gene therapy of lysosomal storage disorders 207
Trinucleotide repeat disorders 207
Muscular dystrophies 208
Duchenne muscular dystrophy (DMD) 208
Animal models for gene therapy of DMD 208
Antisense approach to DMD 208
Liposome-mediated gene transfer 209
Myoblast-based gene transfer in DMD 210
Plasmid-mediated gene therapy 210
Post-transcriptional modulation of gene expression in DMD 210
Repair of dystrophin gene 211
Routes of administration of gene therapy in DMD 211
Types of dystrophin constructs 211
Viral vectors for DMD 212
Conclusions and future prospects of gene therapy of DMD 213
Limb-girdle muscular dystrophy 214
Myotonic dystrophy 214
Spinal muscular atrophy 215
Antisense gene therapy of SMA 215
Hereditary neuropathies 215
Charcot-Marie-Tooth disease 215
Hereditary axonal neuropathies of the peripheral nerves 216
Gene therapy of mitochondrial disorders 216
Companies involved in gene therapy of genetic disorders 217
5. Gene Therapy of Cancer 219
Strategies for cancer gene therapy 219
Direct gene delivery to the tumor 220
Injection into tumor 220
Direct injection of adenoviral vectors 220
Direct injection of a plasmid DNA-liposome complex 221
A polymer approach to local gene therapy for cancer 221
Electroporation for cancer gene therapy 221
Control of gene expression in tumor by local heat 222
Radiation-guided gene therapy of cancer 222
Nanoparticles to facilitate combination of hyperthermia and gene therapy 223
Cell-based cancer gene therapy 223
Adoptive cell therapy 223
Cytokine gene therapy 224
Genetic modification of human hematopoietic stem cells 227
Immunogene therapy 227
Cancer vaccines 228
Genetically modified cancer cell vaccines 228
GVAX cancer vaccines 228
Genetically modified dendritic cells 229
Nucleic acid-based cancer vaccines 229
DNA cancer vaccines 230
RNA vaccines 230
Viral vector-based cancer vaccines 230
Intradermal delivery of cancer vaccines by Ad vectors 231
Future prospects of cancer vaccines 231
Companies involved in nucleic acid-based cancer vaccines 231
Monoclonal antibody gene transfer for cancer 232
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 242
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 243
Tumor-targeted gene therapy by receptor-mediated endocytosis 244
Virus-mediated oncolysis 244
Targeted cancer treatments based on oncolytic viruses 244
Oncolytic HSV 244
Oncolytic adenoviruses 245
Oncolytic vesicular stomatitis virus 246
Oncolytic paramyxovirus 246
Oncolytic vaccinia virus 247
Cancer terminator virus 247
Cytokine-induced killer cells for delivery of an oncolytic virus 248
Monitoring of viral-mediated oncolysis by PET 248
Oncolytic gene therapy 248
Companies developing oncolytic viruses 249
Apoptotic approach to improve cancer gene therapy 249
Tumor suppressor gene therapy 250
P53 gene therapy 250
BRIT1 gene therapy 250
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 251
Gene therapy for bladder cancer 251
Gene therapy for glioblastoma multiforme. 252
Targeted adenoviral vectors 253
Genetically engineered MSCs for gene delivery to intracranial gliomas 254
Targeting normal brain cells with an AAV vector encoding interferon-b 254
Viral oncolysis of brain tumors 254
Autophagy induced by conditionally replicating adenoviruses 255
Oncolytic virus targeted to brain tumor stem cells 255
Antiangiogenic gene therapy 256
Baculovirus vector for diphtheria toxin gene therapy 256
Intravenous gene delivery with nanoparticles into brain tumors 256
Gene therapy targeting hepatocyte growth factor 257
RNAi gene therapy of brain cancer 257
Ligand-directed delivery of dsRNA molecules targeted to EGFR 257
Gene therapy for breast cancer 258
Gene vaccine for breast cancer 258
Recombinant adenoviral ErbB-2/neu vaccine 259
Gene Therapy for ovarian cancer 259
Gene therapy for malignant melanoma 260
Gene therapy of lung cancer 262
Intravenous nanoparticle formulation for delivery of FUS1 gene 262
Aerosol gene delivery for lung cancer 263
Gene therapy for cancer of prostate 263
Experimental studies 263
Nanoparticle-based gene therapy for prostate cancer 263
Tumor suppressor gene therapy in prostate cancer 264
Vaccines for prostate cancer 264
Clinical trials 264
Gene therapy of head and neck cancer 265
Adenoviral vector based P53 gene therapy 265
Gene therapy of pancreatic cancer 265
Rexin-GO for targeted gene delivery in cancer 266
Targeted Expression of BikDD gene 266
Concluding remarks on gene therapy of pancreatic cancer 266
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 276
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
Neprilysin gene therapy 287
Targeting plasminogen activator inhibitor type-1 gene 288
Gene vaccination 288
Combination of gene therapy with other treatments for AD 288
Gene therapy of Huntington disease 289
Encapsulated genetically engineered cellular implants 289
Viral vector mediated administration of neurotrophic factors 289
RNAi gene therapy 289
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 291
Transgenic mice as models for stroke 292
Animal models for gene therapy of arteriovenous malformations 292
Gene transfer to cerebral blood vessels 292
Gene therapy for vasospasm following subarachnoid hemorrhage 293
NOS gene therapy for cerebral vasospasm 294
Gene therapy for stroke 294
Gene therapy for stroke using neurotrophic factors 296
Gene therapy of strokes with a genetic component 296
Gene therapy for intracranial aneurysms 296
Concluding remarks about gene therapy for stroke 296
Gene therapy of injuries to the nervous system 297
Traumatic brain injury 297
Spinal cord injury 297
Gene therapy of epilepsy 298
Gene therapy for control of seizures 298
Gene therapy for neuroprotection in epilepsy 299
Gene therapy for genetic forms of epilepsy 300
Gene therapy for multiple sclerosis 300
Gene therapy for relief of pain 301
Rationale of gene therapy for pain 301
Vectors for gene therapy of pain 301
Methods of gene delivery for pain 302
Endogenous analgesic production for cranial neuralgias 302
Gene delivery by intrathecal route 303
Gene transfer for delivery of analgesics to the spinal nerve roots 303
Gene therapy of peripheral neuropathic pain 304
Gene transfer by injections into the brain substance 305
Targets for gene therapy of pain 305
Zinc finger DNA-binding protein therapeutic for chronic pain 305
Gene therapy for producing enkephalin to block pain signals 305
Targeting nuclear factor-kB 306
Gene therapy targeted to neuroimmune component of chronic pain 306
Potential applications of gene therapy for management of pain 306
Concluding remarks on gene therapy for pain 307
Gene therapy for psychiatric disorders 308
Gene therapy for depression 308
Gene therapy for enhancing cognition after stress 308
Gene therapy against fear disorders 309
Companies involved in gene therapy of neurological disorders 309
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 322
Congestive heart failure 323
Rationale of gene therapy in CHF 323
Beta-ARKct gene therapy 323
Intracoronary adenovirus-mediated gene therapy for CHF 324
AAV-mediated gene transfer for CHF 324
AngioCell gene therapy for CHF 325
nNOS gene transfer in CHF 325
Cardiomyopathies 325
Cardiac conduction disturbances 326
Gene transfer approaches for biological pacemakers 326
Genetically engineered biological pacemakers 326
Gene therapy and heart transplantation 327
Peripheral arterial disease 327
Incidence and clinical features 327
Current management 328
Gene therapy for peripheral arterial disease 328
Angiogenesis by gene therapy 328
HIF-1a gene therapy for peripheral arterial disease 328
HGF gene therapy for peripheral arterial disease 329
Ischemic neuropathy secondary to peripheral arterial disease 329
Prevention of restenosis after angioplasty 330
Antisense approaches 330
Gene therapy to prevent restenosis after angioplasty 330
Techniques of gene therapy for restenosis 332
NOS gene therapy for restenosis 332
hTIMP-1 gene therapy to prevent intimal hyperplasia 333
Maintaining vascular patency after surgery 333
Companies involved in gene therapy of cardiovascular diseases 334
Future prospects of gene therapy of cardiovascular disorders 334
8. Gene therapy of viral infections 337
Introduction 337
Acquired Immunodeficiency Syndrome (AIDS) 337
Current management of AIDS 337
Gene therapy strategies in HIV/AIDS 338
HIV/AIDS vaccines 338
Insertion of protective genes into target cells. 339
Cell/gene therapies for HIV/AIDS 340
Transplantation of genetically modified T-cells 340
Transplantation of genetically modified hematopoietic cells 340
Anti-HIV ribozyme delivered in hematopoietic progenitor cells 341
Inhibition of HIV-1 replication by lentiviral vectors 341
VRX496 341
Intracellular immunization 342
Engineered cellular proteins such as soluble CD4s 342
Intracellular antibodies 342
Anti-rev single chain antibody fragment 342
Use of genes to chemosensitize HIV-1 infected cells 343
Autocrine interferon (INF)-b production by somatic cell gene therapy 343
Antisense approaches to AIDS 343
RNA decoys 343
Antisense oligodeoxynucleotides 343
RNA decoys 344
Ribozymes 344
RNAi applications in HIV/AIDS 345
siRNA-directed inhibition of HIV-1 infection 345
Role of the nef gene during HIV-1 infection and RNAi 345
Bispecific siRNA constructs 346
Targeting CXCR4 with siRNAs 346
Targeting CCR5 with siRNAs 346
Companies involved in developing gene therapy for HIV/AIDS 347
Conclusions regarding gene therapy of HIV/AIDS 348
Genetic vaccines for other viral infections 348
Cytomegalic virus infections 348
Viral hepatitis 349
Vaccine for hepatitis B virus 349
Vaccine for hepatitis C virus 350
Vaccine for herpes simplex virus 350
DNA vaccine against rabies 350
DNA vaccine for Ebola 351
Vaccines for avian influenza 351
Future prospects of DNA vaccines for avian influenza 352
Human trial of a DNA vaccine for avian influenza 353
Companies developing genetic vaccines for infections other than AIDS 353
9. Research, Development and Future of Gene Therapy 355
Basic research in gene therapy 355
R & D in gene therapy 355
Animal models of human diseases for gene therapy research 356
Lentiviral transgenesis 356
Financing research and development 356
Role of the NIH in gene therapy research 356
National Gene Vector Laboratories 356
Financing by the industry 357
Clinical trials in gene therapy 357
Clinical trials worldwide 357
Clinical
To order this report:
Biotechnology Industry: Gene Therapy - technologies, markets and companies
Check our Company Profile, SWOT and Revenue Analysis!
CONTACT: |
|
Nicolas Bombourg |
|
Reportlinker |
|
Email: [email protected] |
|
US: (805)652-2626 |
|
Intl: +1 805-652-2626 |
|
SOURCE Reportlinker
Share this article