Chapter 1: Executive Summary
Adverse Events in Gene Therapy
The Case of Jesse at University of Pennsylvania
x-Severe Combined Immunodeficiency 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 Profiles
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 1
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
