Reportlinker Adds Gene Therapy - Technologies, Markets and Companies

Jun 03, 2010, 11:08 ET from Reportlinker

NEW YORK, June 3 /PRNewswire/ -- announces that a new market research report is available in its catalogue:

Gene Therapy - technologies, markets and companies

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 188 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


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.


0.Executive Summary19



Historical evolution of gene therapy21

Relation of gene therapy to other biotechnologies23

Molecular biological basics for gene therapy23




Alternative RNA splicing25


Gene regulation26

Gene expression28



Mitochondrial DNA29


2.Gene Therapy Technologies31

Classification of gene therapy techniques31

Ex vivo and in vivo gene therapy32

Ex vivo gene therapy32

In vivo gene therapy33

Physical methods of gene transfer33


Applications of electroporation34

Clinical applications of electroporation35

Advantages of electroporation35

Limitations of electroporation36



Particle bombardment37

Ultrasound-mediated transfection39

Molecular vibration40

Application of pulsed magnetic field and superparamagnetic nanoparticles40

Gene transfection using laser irradiation40

Photochemical transfection41

Chemical methods of gene transfer41

Gene repair and replacement41

Gene repair by single-stranded oligonucleotides42

History and current status of chimeraplasty42

mRNA gene therapy42

Spliceosome mediated RNA trans-splicing43

Vectors for gene therapy44

Basic considerations44

Use of genes as pharmaceuticals44

The ideal vector for gene therapy44

Viral vectors45

Adenovirus vectors46

Adeno-associated virus vectors48

Alphavirus vectors50

Baculovirus vectors50

Foamy virus vectors51

Herpes simplex virus vectors51

Lentiviral vectors53

Multicistronic retroviral vectors54

Retroviral vectors55

Oncognic potential of retroviral vectors56

Future prospects of viral vectors57

Companies using viral vectors57

Nonviral vectors for gene therapy59

Anionic lipid-DNA complexes59

Cationic lipid-DNA complexes60

Effects of shape of DNA molecules on delivery with nonviral vectors60

Electrostatic modifications of surface to improve gene delivery60

Liposomes for gene therapy61

Liposome-nucleic acid complexes62

Liposome-HVJ complex63

Transposons DNA vectors63

Polycation-DNA complexes (polyplexes)64

Plasmid DNA vector for treatment of chronic inflammatory disease65

Polymer molecules65

Synthetic peptide complexes65

Future prospects of nonviral vs viral vectors65

Nanobiotechnology for gene transfer66

Nanoparticles as nonviral vectors for gene therapy66



Calcium phosphate nanoparticles as non-viral vectors67

Lipid nanoparticles for nucleic acid delivery68

Silica nanoparticles as a nonviral vector for gene delivery68

Gelatin nanoparticles for gene delivery69

Nonionic polymeric micelles for oral gene delivery69

Biological nanoparticle technology69

Nanoparticles with virus-like function as gene therapy vectors69

Receptor-mediated endocytosis70

Artificial viral vectors71

Directed evolution of AAV to create efficient gene delivery vectors71

Bacterial ghosts as DNA delivery systems72

Bacteria plus nanoparticles for gene delivery into cells72

Chromosome-based vectors for gene therapy73

Companies using nonviral vectors74

Concluding remarks about vectors75

Cell-mediated gene therapy76


Skeletal muscle cells78

Vascular smooth muscle cells78




Regulating protein delivery by genetically encoded lymphocytes79

Implantation of microencapulated genetically modified cells79

Stem cell gene therapy80

Therapeutic applications for hematopoietic stem cell gene transfer81

Improving delivery of genes to stem cells81

Lentiviral vectors for gene transfer to marrow stem cells81

Use of mesenchymal stem cells for gene therapy81

Microporation for transfection of MSCs81

In utero gene therapy using stem cells82

Gene delivery to stem cells by artificial chromosome expression82

Linker based sperm-mediated gene transfer technology82

Combination of gene therapy with therapeutic cloning83

Expansion of transduced HSCs in vivo83

The future of hematopoietic stem cell gene therapy83

Routes of administration for gene therapy83

Direct injection of naked DNA84

Intramuscular injection84

Intravenous DNA injection85

Intraarterial delivery85

Companies with gene delivery devices/ technologies85

Targeted gene therapy86

Targeted integration87

Bacteriophage integrase system for site-specific gene delivery87

Controlled-release delivery of DNA87

Controlled gene therapy88

Controlled delivery of genetic material88

Controlled induction of gene expression88

Drug-inducible systems for control of gene expression89

Timed activation of gene therapy by a circuit based on signaling network89

Small molecules for post-transcriptional regulation of gene expression90

Engineered zinc finger DNA binding proteins for gene correction90

Light Activated Gene Therapy90

Spatial control of gene expression via local hyperthermia91

Companies with regulated /targeted gene therapy91

Gene marking92

Germline gene therapy92

Potential applications of human germline genome modification92

Pros and cons of human germline genome modification93

Role of gene transfer in antibody therapy94

Genetically engineered vaccines95

DNA vaccines95

DNA inoculation technology95

Methods for enhancing the potency of DNA vaccines96

Advantages of DNA vaccines96

Vaccine vectors97

Challenges and limitations of genetically engineered vaccines98

Vaccines based on reverse genetics98

Technologies for gene suppression98

Antisense oligonucleotides99

Transcription factor decoys99



Peptide nucleic acid100

Intracellular delivery of PNAs101

Locked nucleic acid101


Gene silencing102

Post-transcriptional gene silencing102

Definitions and terminology of RNAi102

RNAi mechanisms102

Inhibition of gene expression by antigene RNA104

RNAi gene therapy105

Application of molecular diagnostic methods in gene therapy105

Use of PCR to study biodistribution of gene therapy vector105

PCR for verification of the transcription of DNA106

In situ PCR for direct quantification of gene transfer into cells106

Detection of retroviruses by reverse transcriptase (RT)-PCR106

Confirmation of viral vector integration106

Monitoring of gene expression107

Monitoring of gene expression by green fluorescent protein107

Monitoring in vivo gene expression by molecular imaging107

Advantages of gene therapy compared with protein therapy107

3.Clinical Applications of Gene Therapy109


Bone and joint disorders109

Bone fractures109

Gene therapy for intervertebral disc degeneration110

Spinal fusion110

Osteogenesis imperfecta111

Rheumatoid arthritis111

Local or systemic treatment112

In vivo or ex vivo gene therapy of RA112

Clinical trials113

Gene therapy for osteoarthritis114

Sports injuries115

Repair of articular cartilage defects115

Regeneration and replacement of bone by gene therapy116

Bacterial infections117

Antisense approach to bacterial infections117


Tissue engineering in dental implant defects117

Endocrine disorders118


Diabetes mellitus118

Methods of gene therapy of diabetes mellitus118

Viral vector-mediated gene transfer in diabetes119

Gene delivery with ultrasonic microbubble destruction technology120

Genetically engineered cells for diabetes mellitus120

Genetically altered liver cells120

Genetically modified stem cells121

Genetically engineered dendritic cells121

Insertion of gene encoding for IL-4121

Concluding remarks about cell and gene therapy of diabetes121

Gene therapy of growth-hormone deficiency122

Gene therapy of obesity123

Ad viral vector-mediated transfer of leptin gene123

AAV vector-mediated delivery of GDNF for obesity124

Gastrointestinal disorders124


Methods of gene transfer to the gastrointestinal tract125

Direct delivery of genes125

Naked plasmid DNA into the submucosa125

Viral vectors125

Receptor-mediated endocytosis125

Indications for gastrointestinal gene therapy125

Gene therapy for inflammatory disorders of the bowel126

Gene transfer to the salivary glands127

Potential clinical applications of salivary gene therapy127



Gene therapy of hemophilia128

Hemophilia A129

Hemophilia B130

Concluding remarks about gene therapy of hemophilias130


Stem cell-based gene therapy and RNAi for sickle cell disease131

Gene therapy for ?-thalassemia132

Gene therapy of Fanconi's anemia133

Acquired hematopoietic disorders134

Chronic acquired anemias134



Concluding remarks about gene therapy of hemoglobinopathies136

Companies involved in gene thery of hematological disorders136

In utero gene therapy137

Fetal gene transfer techniques137

Animal models of fetal gene therapy138

Potential applications of fetal gene therapy138

Fetal gene therapy for cystic fibrosis138

Fetal intestinal gene therapy139

Hearing disorders139

Potential of gene therapy139

Vectors for gene therapy of hearing disorders140

Auditory hair cell replacement and hearing improvement by gene therapy140

Kidney diseases141

End-stage renal disease141

Methods of gene delivery to the kidney141

Gene transfer into kidney by adenoviral vectors142

Non-viral gene transfer to the kidneys142

Gene transfer into the glomerulus by HVJ-liposome142

Bone marrow stem cells for renal disease142

Mesangial cell therapy143

Liposome-mediated gene transfer into the tubules143

Gene transfer to tubules with cationic polymer polyethylenimine144

Gene therapy in animal experimental models of renal disease144

Genetic manipulations of the embryonic kidney144

Antisense intervention in glomerulonephritis145

Gene therapy for renal fibrosis145

Use of genetically engineered cells for uremia due to renal failure145

Concluding remarks146

Liver disorders146

Techniques of gene delivery to liver147

Direct injection of DNA into liver147

Local gene delivery by isolated organ perfusion147

Liposome-mediated direct gene transfer148

Retroviral vector for gene transfer to liver148

Adenoviral vectors for gene transfer to liver148

Receptor-mediated approach148

Cell therapy for liver disorders149

Transplantation of genetically modified hepatocytes149

Genetically modified hematopoietic stem cells149

Gene therapy by ex vivo transduced liver progenitor cells149

Gene therapy of genetic diseases affecting the liver150

Crigler-Najjar syndrome150

Hereditary tyrosinemia type I (HT1)150

Hereditary tyrosinemia type 3150

Gene therapy of acquired diseases affecting the liver151

Cirrhosis of liver151

Ophthalmic disorders151

Introduction to gene therapy of ophthalmic disorders151

Degenerative retinal disorders152

Age-related macular degeneration152

Inherited retinal degenerations154

Inherited disorders affecting vision155

Gene therapy for color blindness155

Leber congenital amaurosis155

Retinitis pigmentosa156

Stargardt disease157

Usher syndrome157

X-linked juvenile retinoschisis157

Proliferative retinopathies158

Methods of gene transfer to retinal cells158

DNA nanoparticles for nonviral gene transfer to the eye159

Prevention of complications associated with eye surgery159

Prevention of proliferative retinopathy by gene therapy159

DNA nanoparticles for gene therapy of retinal degenerative disorders160

Posterior capsule opacification after cataract surgery160

Autoimmune uveitis160

Retinal ischemic injury161

Corneal disorders161


Disorders of hearing162

Gene therapy for hearing loss162

Organ transplantation163


DNA vaccines for transplantation163

Gene therapy for prolonging allograft survival163

Gene therapy in lung transplantation164

Role of gene therapy in liver transplantation164

Gene therapy in kidney transplantation164

Veto cells and transplant tolerance165

Pulmonary disorders165

Techniques of gene delivery to the lungs166

Adenoviral vectors166

Non-viral vectors167

Aerosolization as an aid to gene transfer to lungs.167

Cystic fibrosis167

Genetics and clinical features167

Gene therapy for CF168

CFTR gene transfer in CF168

Concluding remarks about gene therapy of CF169

Miscellaneous pulmonary disorders170

Gene therapy for pulmonary arterial hypertension170

Gene therapy for bleomycin-induced pulmonary fibrosis171

Pulmonary complications of a1-antitrypsin deficiency171

Gene therapy for asthma172

Gene therapy for adult respiratory distress syndrome173

Gene therapy for lung injury173

Gene therapy for bronchopulmonary dysplasia173

Concluding remarks about gene therapy of lungs174

Companies involved in pulmonary gene therapy174

Skin and soft tissue disorders175

Gene transfer to the skin175

Electroporation for transdermal delivery of plasmid DNA175

Electroporation for transdermal delivery of DNA vaccines175

Liposomes for transdermal gene delivery176

Ultrasound and topical gene therapy176

Gene therapy in skin disorders176

Gene therapy of hair loss176

Gene therapy for xeroderma pigmentosa177

Gene therapy for lamellar ichthyosis177

Gene therapy for epidermolysis bullosa177

Gene transfer techniques for wound healing178

Urogenital disorders179

Gene therapy for urinary tract dysfunction179

Gene therapy for erectile dysfunction179

NOS gene transfer for erectile dysfunction179

Clinical trial of hMaxi-K Gene transfer in erectile dysfunction179

Gene therapy for erectile dysfunction due to nerve injury180

Concluding remarks on gene therapy for erectile dysfunction180

Veterinary gene therapy180

Gene therapy for mucopolysaccharidosis VII in dogs181

Gene therapy to increase disease resistance181

Gene therapy for infections181

Gene therapy for chronic anemia182

Gene therapy for endocrine disorders182

Gene therapy for arthritis182

Cancer gene therapy183

Brain tumors in cats and dogs183

Breast cancer in dogs184

Canine hemangiosarcoma184

Canine melanoma184

Canine soft tissue sarcoma185

Melanoma in horses185

4.Gene Therapy of Genetic Disorders187


Primary immunodeficiency disorders188

Severe combined immune deficiency189

Chronic granulomatous disease191

Wiskott-Aldrich syndrome191

Purine nucleoside phosphorylase deficiency192

Major histocompatibility class II deficiency192

Future prospects of gene therapy of inherited immunodeficiencies193

Metabolic disorders193


Canavan disease194

Lesch-Nyhan syndrome195

Ornithine transcarbamylase deficiency195



Tetrahydrobiopterin deficiency197

Lysosomal storage disorders.197

Batten disease198

Fabry's disease199

Farber's disease199

Gaucher disease199

Animals models of Gaucher's disease200

Gene therapy of Gaucher's disease200

Hunter syndrome201

Combination of cell and gene therapy for Krabbe's disease201

Metachromatic leukodystrophy202

Mucopolysaccharidosis type 1 (Hurler syndrome)202

Niemann-Pick type A disease203

Pompe disease203

Sanfilippo A syndrome204

Sly syndrome204

Tay-Sachs disease204

Future prospects of gene therapy of lysosomal storage disorders205

Trinucleotide repeat disorders205

Muscular dystrophies205

Duchenne muscular dystrophy (DMD)205

Animal models for gene therapy of DMD206

Antisense approach to DMD206

Liposome-mediated gene transfer207

Myoblast-based gene transfer in DMD207

Plasmid-mediated gene therapy208

Post-transcriptional modulation of gene expression in DMD208

Repair of dystrophin gene208

Routes of administration of gene therapy in DMD209

Types of dystrophin constructs209

Viral vectors for DMD210

Conclusions and future prospects of gene therapy of DMD211

Limb-girdle muscular dystrophy212

Myotonic dystrophy212

Spinal muscular atrophy212

Antisense gene therapy of SMA213

Hereditary neuropathies213

Charcot-Marie-Tooth disease213

Hereditary axonal neuropathies of the peripheral nerves214

Gene therapy of mitochondrial disorders214

Companies involved in gene therapy of genetic disorders215

5.Gene Therapy of Cancer217

Strategies for cancer gene therapy217

Direct gene delivery to the tumor218

Injection into tumor218

Direct injection of adenoviral vectors218

Direct injection of a plasmid DNA-liposome complex219

A polymer approach to local gene therapy for cancer219

Electroporation for cancer gene therapy219

Control of gene expression in tumor by local heat220

Radiation-guided gene therapy of cancer220

Nanoparticles to facilitate combination of hyperthermia and gene therapy221

Cell-based cancer gene therapy221

Adoptive cell therapy221

Cytokine gene therapy222

Genetic modification of human hematopoietic stem cells225

Immunogene therapy225

Cancer vaccines226

Genetically modified cancer cell vaccines226

GVAX cancer vaccines226

Genetically modified dendritic cells227

Nucleic acid-based cancer vaccines227

DNA cancer vaccines228

RNA vaccines228

Viral vector-based cancer vaccines228

Intradermal delivery of cancer vaccines by Ad vectors229

Future prospects of cancer vaccines229

Companies involved in nucleic acid-based cancer vaccines229

Monoclonal antibody gene transfer for cancer230

Transfer and expression of intracellular adhesion-1 molecules231

Other gene-based techniques of immunotherapy of cancer231

Fas (Apo-1)231


Major Histocompatibility Complex (MHC) Class I232

IGF (Insulin-Like Growth Factor)232

Inhibition of immunosuppressive function in cancer232

Delivery of toxic genes to tumor cells for eradication233

Gene-directed enzyme prodrug therapy233

Combination of gene therapy with radiotherapy233

Correction of genetic defects in cancer cells234

Targeted gene therapy for cancer234

Bacteria as novel anticancer gene vectors234

Cancer-specific gene expression235

Cancer-specific transcription235

Delivery of retroviral particles hitchhiking on T cells236

Electrogene and electrochemotherapy236

Epidermal growth factor-mediated DNA delivery236

Gene-based targeted drug delivery to tumors236

Gene expression in hypoxic tumor cells237

Genetically modified T cells for targeting tumors237

Genetically engineered stem cells for targeting tumors238

Hematopoietic stem cells for targeted cancer gene therapy239

Immunolipoplex for delivery of p53 gene239

Nanomagnets for targeted cell-based cancer gene therapy240

Nanoparticles for targeted site-specific delivery of anticancer genes240

Targeted cancer therapy using a dendrimer-based synthetic vector240

Tumor-targeted gene therapy by receptor-mediated endocytosis241

Virus-mediated oncolysis241

Targeted cancer treatments based on oncolytic viruses241

Oncolytic HSV242

Oncolytic adenoviruses242

Oncolytic vesicular stomatitis virus243

Oncolytic paramyxovirus244

Oncolytic vaccinia virus244

Cancer terminator virus244

Cytokine-induced killer cells for delivery of an oncolytic virus245

Monitoring of viral-mediated oncolysis by PET245

Oncolytic gene therapy245

Companies developing oncolytic viruses246

Apoptotic approach to improve cancer gene therapy246

Tumor suppressor gene therapy247

P53 gene therapy247

BRIT1 gene therapy247

Nitric oxide-based cancer gene therapy248

Nitric oxide synthase II DNA injection248

Gene therapy for radiosensitization of cancer248

Gene therapy of cancer of selected organs248

Gene therapy for bladder cancer248

Gene therapy for glioblastoma multiforme.249

Targeted adenoviral vectors250

Genetically engineered MSCs for gene delivery to intracranial gliomas251

Targeting normal brain cells with an AAV vector encoding interferon-?251

Viral oncolysis of brain tumors251

Autophagy induced by conditionally replicating adenoviruses252

Oncolytic virus targeted to brain tumor stem cells252

Antiangiogenic gene therapy253

Baculovirus vector for diphtheria toxin gene therapy253

Intravenous gene delivery with nanoparticles into brain tumors254

Gene therapy targeting hepatocyte growth factor254

RNAi gene therapy of brain cancer254

Ligand-directed delivery of dsRNA molecules targeted to EGFR255

Gene therapy for breast cancer255

Gene vaccine for breast cancer255

Recombinant adenoviral ErbB-2/neu vaccine256

Gene Therapy for ovarian cancer256

Gene therapy for malignant melanoma257

Gene therapy of lung cancer259

Intravenous nanoparticle formulation for delivery of FUS1 gene259

Aerosol gene delivery for lung cancer260

Gene therapy for cancer of prostate260

Experimental studies260

Nanoparticle-based gene therapy for prostate cancer260

Tumor suppressor gene therapy in prostate cancer261

Vaccines for prostate cancer261

Clinical trials261

Gene therapy of head and neck cancer262

Adenoviral vector based P53 gene therapy262

Gene therapy of pancreatic cancer262

Rexin-G? for targeted gene delivery in cancer263

Targeted Expression of BikDD gene263

Concluding remarks on gene therapy of pancreatic cancer263

Cancer gene therapy companies264

6.Gene Therapy of Neurological Disorders267


Gene transfer techniques for the nervous system268

Methods of gene transfer to the nervous system268

Ideal vector for gene therapy of neurological disorders268

Promoters of gene transfer268

Lentivirus-mediated gene transfer to the CNS269

AAV vector mediated gene therapy for neurogenetic disorders269

Gene transfer to the CNS using recombinant SV40-derived vectors270

Routes of delivery of genes to the CNS270

Direct injection into CNS270

Introduction of the genes into cerebral circulation271

Introduction of genes into cerebrospinal fluid271

Intravenous administration of vectors271

Delivery of gene therapy to the peripheral nervous system272

Cell-mediated gene therapy of neurological disorders272

Neuronal cells272

Neural stem cells and progenitor cells272


Cerebral endothelial cells273

Implantation of genetically modified encapsulated cells into the brain273

Gene therapy of neurodegenerative disorders273

Gene therapy for Parkinson disease273


Techniques of gene therapy for PD275

Delivery of neurotrophic factors by gene therapy278

Delivery of parkin gene279

Introduction of functional genes into the brain of patients with PD279

Nanoparticle-based gene therapy for PD279

Mitochondrial gene therapy for PD279

RNAi approach to PD280

Prospects of gene therapy for PD280

Companies developing gene therapy for PD281

Gene therapy for Alzheimer disease282


NGF gene therapy for AD282

Neprilysin gene therapy283

Targeting plasminogen activator inhibitor type-1 gene284

Gene vaccination284

Combination of gene therapy with other treatments for AD284

Gene therapy of Huntington disease284

Encapsulated genetically engineered cellular implants284

Viral vector mediated administration of neurotrophic factors285

RNAi gene therapy285

Gene therapy of amyotrophic lateral sclerosis285


Technique of gene therapy of ALS285

Gene therapy of cerebrovascular diseases287

Preclinical research in gene therapy for cerebrovascular disease287

Animal models of stroke relevant to gene therapy287

Transgenic mice as models for stroke287

Animal models for gene therapy of arteriovenous malformations288

Gene transfer to cerebral blood vessels288

Gene therapy for vasospasm following subarachnoid hemorrhage289

NOS gene therapy for cerebral vasospasm290

Gene therapy for stroke290

Gene therapy for stroke using neurotrophic factors291

Gene therapy of strokes with a genetic component292

Gene therapy for intracranial aneurysms292

Concluding remarks about gene therapy for stroke292

Gene therapy of injuries to the nervous system293

Traumatic brain injury293

Spinal cord injury293

Gene therapy of epilepsy294

Gene therapy for control of seizures294

Gene therapy for neuroprotection in epilepsy295

Gene therapy for genetic forms of epilepsy296

Gene therapy for multiple sclerosis296

Gene therapy for relief of pain296

Rationale of gene therapy for pain296

Vectors for gene therapy of pain297

Methods of gene delivery for pain297

Endogenous analgesic production for cranial neuralgias298

Gene delivery by intrathecal route298

Gene transfer for delivery of analgesics to the spinal nerve roots299

Gene therapy of peripheral neuropathic pain300

Gene transfer by injections into the brain substance300

Targets for gene therapy of pain300

Zinc finger DNA-binding protein therapeutic for chronic pain300

Gene therapy for producing enkephalin to block pain signals301

Targeting nuclear factor-?B301

Gene therapy targeted to neuroimmune component of chronic pain301

Potential applications of gene therapy for management of pain302

Concluding remarks on gene therapy for pain302

Gene therapy for psychiatric disorders303

Gene therapy for depression303

Gene therapy for enhancing cognition after stress304

Companies involved in gene therapy of neurological disorders304

7.Gene Therapy of Cardiovascular Disorders305


Techniques of gene transfer to the cardiovascular system305

Direct plasmid injection into the myocardium306

Catheter-based systems for vector delivery306

Ultrasound microbubbles for cardiovascular gene delivery307

Vectors for cardiovascular gene therapy307

Adenoviral vectors for cardiovascular diseases307

Plasmid DNA-based delivery in cardiovascular disorders307

Intravenous rAAV vectors for targeted delivery to the heart308

Hypoxia-regulated gene therapy for myocardial ischemia308

Angiogenesis and gene therapy of ischemic disorders308

Therapeutic angiogenesis vs vascular growth factor therapy309

Gene painting for delivery of targeted gene therapy to the heart309

Gene delivery to vascular endothelium310

Targeted plasmid DNA delivery to the cardiovascular system with nanoparticles310

Vascular stents for gene delivery310

Gene therapy for genetic cardiovascular disorders311

Genetic disorders predisposing to atherosclerosis311

Familial hypercholesterolemia (FH)311

Apolipoprotein E (apoE) deficiency313


Genetic factors for myocardial infarction314

Acquired cardiovascular diseases314

Coronary artery disease with angina pectoris314


Ischemic heart disease with myocardial infarction315

Myocardial repair with IGF-1 therapy316

Metalloproteinase-2 inhibitor gene therapy316

Congestive heart failure317

Rationale of gene therapy in CHF317

-ARKct gene therapy317

Intracoronary adenovirus-mediated gene therapy for CHF318

AAV-mediated gene transfer for CHF318

AngioCell gene therapy for CHF318

nNOS gene transfer in CHF319


Cardiac conduction disturbances319

Gene transfer approaches for biological pacemakers319

Genetically engineered biological pacemakers320

Gene therapy and heart transplantation321

Peripheral arterial disease321

Incidence and clinical features321

Current management322

Gene therapy for peripheral arterial disease322

Angiogenesis by gene therapy322

HIF-1? gene therapy for peripheral arterial disease322

HGF gene therapy for peripheral arterial disease323

Ischemic neuropathy secondary to peripheral arterial disease323

Prevention of restenosis after angioplasty323

Antisense approaches324

Gene therapy to prevent restenosis after angioplasty324

Techniques of gene therapy for restenosis325

NOS gene therapy for restenosis326

hTIMP-1 gene therapy to prevent intimal hyperplasia327

Maintaining vascular patency after surgery327

Companies involved in gene therapy of cardiovascular diseases327

Future prospects of gene therapy of cardiovascular disorders328

8.Gene therapy of viral infections331


Acquired Immunodeficiency Syndrome (AIDS)331

Current management of AIDS331

Gene therapy strategies in HIV/AIDS332

HIV/AIDS vaccines332

Insertion of protective genes into target cells.333

Cell/gene therapies for HIV/AIDS334

Transplantation of genetically modified T-cells334

Transplantation of genetically modified hematopoietic cells334

Anti-HIV ribozyme delivered in hematopoietic progenitor cells335

Inhibition of HIV-1 replication by lentiviral vectors335


Intracellular immunization336

Engineered cellular proteins such as soluble CD4s336

Intracellular antibodies336

Anti-rev single chain antibody fragment336

Use of genes to chemosensitize HIV-1 infected cells337

Autocrine interferon (INF)-? production by somatic cell gene therapy337

Antisense approaches to AIDS337

RNA decoys337

Antisense oligodeoxynucleotides337

RNA decoys338


RNAi applications in HIV/AIDS339

siRNA-directed inhibition of HIV-1 infection339

Role of the nef gene during HIV-1 infection and RNAi339

Bispecific siRNA constructs340

Targeting CXCR4 with siRNAs340

Targeting CCR5 with siRNAs340

Companies involved in developing gene therapy for HIV/AIDS341

Conclusions regarding gene therapy of HIV/AIDS342

Genetic vaccines for other viral infections342

Cytomegalic virus infections342

Viral hepatitis343

Vaccine for hepatitis B virus343

Vaccine for hepatitis C virus344

Vaccine for herpes simplex virus344

DNA vaccine against rabies344

DNA vaccine for Ebola345

Vaccines for avian influenza345

Future prospects of DNA vaccines for avian influenza346

Human trial of a DNA vaccine for avian influenza347

Companies developing genetic vaccines for infections other than AIDS347

9.Research, Development and Future of Gene Therapy349

Basic research in gene therapy349

R & D in gene therapy349

Animal models of human diseases for gene therapy research350

Lentiviral transgenesis350

Financing research and development350

Role of the NIH in gene therapy research350

National Gene Vector Laboratories350

Financing by the industry351

Clinical trials in gene therapy351

Clinical trials worldwide351

Clinical trials in cancer gene therapy352

Clinical trials in cardiovascular gene therapy352

Clinical trials in inherited monogenic diseases352

Clinical trials for other indications352

Clinical trials in the US353

Vectors used in gene therapy clinical trials353

Future prospects for the gene therapy354

How to improve gene therapy355

Promising areas of application of gene therapy355

Neurological disorders356

Gene therapy of cardiovascular disorders356

Cancer gene therapy357

Personalized gene therapy358

10.Regulatory, Safety and Ethical Issues of Gene Therapy359

Regulation of gene therapy in the United States359

US Federal guidelines for research involving recombinant DNA molecules359

Regulation of gene therapy in US359

Office of Biotechnology Activities359

Implantation of genetically manipulated cells360

Clinical trials in gene therapy360

Cell and gene therapy INDs placed on hold by the FDA360

Regulation of gene therapy in Germany361

Preclinical research361

Clinical Trials361

Marketing authorization362

Regulation of gene therapy in the United Kingdom362

Regulation of gene therapy in France363

Regulation of gene therapy in the Netherlands363

Regulation of gene therapy in Australia364

Regulation of gene therapy in Japan365

Regulation of gene therapy in China365

Safety issues of gene transfer365

Adverse effects of retroviral vectors365

Insertional mutagenesis365

Adverse effects of HSV vectors366

Neurotoxicity of HSV vectors366

Hepatotoxicity of HSV-tk/ganciclovir approach366

Adverse effects of adenoviral vectors366

Inflammatory effects of adenoviruses in lungs367

Inflammatory e

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Nicolas Bombourg



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