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Gene Therapy: Current Status and Prospects

Published by: Drug and Market Development Publishing

Published: Jun. 1, 2005 - 198 Pages


Table of Contents




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

Abstract

This Report provides a detailed breakdown of the applications and markets for gene therapy as well as an analysis of future growth trends. The Report details the latest developments of pre-clinical and clinical status in gene therapy areas. Delivery mechanisms in gene therapy such as viral and non-viral delivery systems are described in detail. The FDA regulation of gene therapy is overviewed as well. The Report contains company profiles as well as expert opinions from those active in this area.

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