TABLE OF CONTENTS
Chapter 1: Executive Summary
- Adverse Events in Gene Therapy
- The Case of Jesse at University of Pennsylvania
- x-Severe Combined Immunode.ciency Syndrome (x-SCID)
- Approval of First Gene Therapy Product: Gendicine
- Adenovirus Takes p53 Gene into Malignant Cells
- Additional Good News
- Latest Development
- Managing Regulatory Issues in Europe
- Why Gene Therapy has not been Successful
- Recent Developments in Gene Therapy Research
Chapter 2: Market Prospects for Gene Therapy
Chapter 3: Introduction and Background to Gene Therapy
- The Science Behind Gene Therapy
- Delivery Mechanism in Gene Therapy
- In Vivo
- Ex Vivo
- Viral Delivery Systems
- Adenoviruses
- Adeno-Associated Viruses
- Herpes Simplex Virus
- Lentivirus Vectors
- HIV-Based Vectors
- Lentiviral Packaging Systems
- Lentiviral Vectors for Hematopoietic Stem Cells
- Diseases Treated by Viral Methods of DNA-transfer
- Gene Therapy for Cystic Fibrosis
- Liver-Directed Gene Therapy
- Therapy Against Retinitis Pigmentosa
- Non-Viral Methods of DNA Transfer
- Promoter Control for Gene Therapy
Chapter 4: Gene Therapy for Human Diseases
- Gene Therapy for Cancer
- Immuno-potentiation
- Oncogene Inactivation
- Tumor Suppressor Gene
- Molecular Chemotherapy
- Drug Resistance
- Gene Therapy of Cystic Fibrosis
- Wild-type Dominant Allele F of the CFTR Gene
- Gene Therapy for Transplantation
- Gene Therapy for HIV
- The Challenges in HIV
- Gene Therapy for HIV: Work in Progress
- University of Pennsylvania Study
- Clinical Trial Status of Gene Therapy
Chapter 5: Expert Opinions
- Alain P. Rolland, Pharm D., Ph.D., Vical Incorporated
- Dr. Brian Salmons, Ph.D., Science and Technology
- Dr. Jan J. Boesen, Ph.D.,Amsterdam Molecular Therapeutics BV
- Douglas Jolly Ph.D., Advantagene Inc.
- Dr. Nay Wei Soong, Ph.D., Maxygen Inc.
- Xiaobin Lu, Ph.D., VIRxSYS Corporation
- Ruxandra Draghia, MD, Ph.D., Advisys, Inc.
Chapter 6: Company Pro.les
- Advantagene
- AdViSYS Inc.
- Aldevron Inc.
- AMDL Inc.
- Amsterdam Molecular Therapeutics BV
- Angiogene Inc,
- Ark Therapeutics plc
- Athersys Inc
- Austrianova
- BioMedica Inc.
- Cell Genesys, Inc.
- Ceregene Inc.
- Corautus Genetics
- Epeius Biotechnologies Inc.
- GenVec, Inc.
- Introgen Therapeutics
- Targeted Genetics Corporation
- Vical Incorporated
- VIRxSYS Corporation
Appendix 1: Companies Operating in the GT Space
Appendix 2: Gene Therapy Experts
Appendix 3: Patents in Gene Therapy
Appendix 4: Recent Headlines in Gene Therapy
- Genetically Engineered Mouthwash May Delay Oral Cancer
- Gene Therapy Appears Effective for Recurrent Head and Neck Cancer
- Correction of Rapid Growth of Cancer Cells: One Approach
- Involves The Use of "Antisense Oligonucleotides"
- Bubble Boy Saved By Gene Therapy
Appendix 5: Literature and Additional Reading
TABLE OF EXHIBITS
- Exhibit 1.1 Principle of Gene Therapy
- Exhibit 1.2 Number of Publications in the Gene Therapy Area
- Exhibit 1.3 Worldwide Approval of Gene Therapy Clinical Trails
- Exhibit 1.4 Distribution of Gene Therapy Products in Different Stages of Development
- Exhibit 1.5 Publications Published in the Area of Gene Therapy and Cancer
- Exhibit 1.6 Publications Published in the Area of Gene Therapy and Atherosclerosis
- Exhibit 1.7 Publications Published in the Area of Gene Therapy and Duchenne Muscular Dystrophy
- Exhibit 1.8 Publications Published in the Area of Gene Therapy and Parksinsons Disease
- Exhibit 1.9 Publications Published in the Area of Gene Therapy and Cystic Fibrosis
- Exhibit 2.1 Emerging Indications Being Treated Using Gene Therapy Technology
- Exhibit 2.2 Gene Therapy Market Forecast
- Exhibit 2.3 Gene Therapy Clinical Trials by Continent (a) and by Indication (b)
- Exhibit 2.4 Gene Therapy Products Entering Phase III Clinical Trials
- Exhibit 3.1 Human Diseases Treated With Gene Therapy Strategies
- Exhibit 3.2 Gene Therapy Types
- Exhibit 3.3 Two Major Approaches for Gene Therapy
- Exhibit 3.4 Leading Methods of Gene Transfer in Gene Therapy
- Exhibit 3.5 Surrogate Cell Types Proposed as Delivery Vehicles
- Exhibit 3.6 Utilizing Stem Cells for Human Gene Therapy
- Exhibit 3.7 Gene Therapy: Segmentation of Vectors Utilized
- Exhibit 3.8 Mechanism of Action of Lentivirus
- Exhibit 3.9 Schematic of the Lentivirus Proteins, Spatial Organization and the Genome
- Exhibit 4.1 Human Gene Therapy Clinical Trials.
- Exhibit 4.2 Human Gene Therapy Trials, Distribution by Countries
- Exhibit 4.3 Status of Gene Therapy Clinical Trials Approved Worldwide 1989 -2005
- Exhibit 4.4 Utilization of Gene Therapy Strategies in the Treatment of Human Disease
- Exhibit 4.5 Distribution of Gene Therapy Products in Different Stages of Development
- Exhibit 5.1 Vical Inc. Plasmid-Based Gene Products-Company-Sponsored Clinical Trials Clinical and Research Development Pipeline of Advantagene
- Clinical Product Pipeline of Ark Therapeutics
- Oxford BioMedicas Research and Clinical Pipeline
- Vicals Product Development Status
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