Translating Gene Therapy to the Clinic - 1st Edition - ISBN: 9780128005637, 9780128005644

Translating Gene Therapy to the Clinic

1st Edition

Techniques and Approaches

Editors: Jeffrey Laurence Michael Franklin
eBook ISBN: 9780128005644
Hardcover ISBN: 9780128005637
Imprint: Academic Press
Published Date: 17th November 2014
Page Count: 346
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Description

Translating Gene Therapy to the Clinic, edited by Dr. Jeffrey Laurence and Michael Franklin, follows the recent, much-lauded special issue of Translational Research in emphasizing clinical milestones and critical barriers to further progress in the clinic. This comprehensive text provides a background for understanding the techniques involved in human gene therapy trials, and expands upon the disease-specific situations in which these new approaches currently have the greatest therapeutic application or potential, and those areas most in need of future research. It emphasizes methods, tools, and experimental approaches used by leaders in the field of translational gene therapy. The book promotes cross-disciplinary communication between the sub-specialties of medicine, and remains unified in theme.

Key Features

  • Presents impactful and widely supported research across the spectrum of science, method, implementation and clinical application
  • Offers disease-based coverage from expert clinician-scientists, covering everything from arthritis to congestive heart failure, as it details specific progress and barriers for current translational use
  • Provides key background information from immune response through genome engineering and gene transfer, relevant information for practicing clinicians contemplating enrolling patients in gene therapy trials

Readership

Translational researchers in both academia and industry as well as clinicians, some grad students and regulators and educators globally

Table of Contents

  • Preface
  • About the Editors
  • Chapter 1. Translating Genome Engineering to Survival
    • 1.1. Origins
    • 1.2. Synchronicity of Discoveries
    • 1.3. Gene Addition
    • 1.4. From Gene Addition to Gene Editing
    • 1.5. Therapy for Genetic Disorders
    • 1.6. Roadmap to the Future
    • Conflict of Interest
  • Chapter 2. Pluripotent Stem Cells and Gene Therapy
    • 2.1. Genetic Approaches to Pluripotency
    • 2.2. Transcription Factors Important for Reprogramming To Pluripotency
    • 2.3. Methods for Genetic Reprogramming
    • 2.4. Clinical Translation of IPSCS
    • 2.5. Conclusion
  • Chapter 3. Genome Engineering for Therapeutic Applications
    • 3.1. Introduction
    • 3.2. Customizable DNA-Targeting Proteins
    • 3.3. Genome Editing with Engineered Nucleases
    • 3.4. Synthetic Transcription Factors for Therapeutic Applications
    • 3.5. Conclusion
    • Glossary
  • Chapter 4. Immune System Obstacles to In vivo Gene Transfer with Adeno-Associated Virus Vectors
    • 4.1. Introduction
    • 4.2. AAV Vectors
    • 4.3. Innate Immunity in AAV Gene Transfer
    • 4.4. T-Cell Responses to Vectors
    • 4.5. Humoral Immunity
    • 4.6. Conclusions
    • Glossary
  • Chapter 5. Risks of Insertional Mutagenesis by DNA Transposons in Cancer Gene Therapy
    • 5.1. Insertional Mutagenesis—the Downside of Gene Therapy?
    • 5.2. Sleeping Beauty Transposon/Transposase System Adapted for Gene Therapy
    • 5.3. Plasticity of Genomes and Gene Expression in Humans
    • 5.4. Transposon-Mediated Gene Therapy in the Clinic
    • 5.5. Conclusions
  • Chapter 6. Arthritis Gene Therapy: A Brief History and Perspective
    • 6.1. Introduction
    • 6.2. Conception and Strategies
    • 6.3. Technology Development
    • 6.4. Unresolved Issues
    • 6.5. Clinical Trials
    • 6.6. Veterinary Applications
    • 6.7. Other Applications of Intra-Articular Gene Therapy
    • 6.8. Commercialization
    • 6.9. Perspectives
  • Chapter 7. Type 1 Diabetes Mellitus: Immune Modulation as a Prerequisite for Successful Gene Therapy Strategies
    • 7.1. Introduction
    • 7.2. Effective Immune Therapy/Modulation: A Prerequisite for Successful Gene Therapy of Type 1 Diabetes
    • 7.3. Targeted Islet Antigen Recognition and Antigen-Based Therapies
    • 7.4. Broad Immunosuppressive Therapies
    • 7.5. Immunotherapies that Target Events in T Cell Response
    • 7.6. Prospects for Immunotherapy in Protecting Neo-Beta Cells
    • 7.7. Conclusion
  • Chapter 8. Gene Therapy for Diabetes
    • 8.1. Introduction
    • 8.2. Generation of β Cells from Pancreatic Mature Non-β Cells
    • 8.3. Generation of β Cells from Tissue Progenitor Cells
    • 8.4. Generation of β Cells from Stem Cells
    • 8.5. Generation of New β Cells by Inducing Their Replication
    • 8.6. Closing Remarks
  • Chapter 9. Gene Therapy for Neurological Diseases
    • 9.1. Introduction
    • 9.2. Viral Vectors for Neurological Diseases
    • 9.3. Gene Therapy for Chronic Pain
    • 9.4. Gene Therapy for Epilepsy
    • 9.5. Parkinson’s Disease
    • 9.6. Conclusions
  • Chapter 10. Genetic and Cell-Mediated Therapies for Duchenne Muscular Dystrophy
    • 10.1. Introduction
    • 10.2. Gene Replacement Therapies
    • 10.3. Strategies Aimed at Correcting the Defective Dystrophin Gene
    • 10.4. Cell-Based Therapies for DMD
    • 10.5. Alternative Strategies to Restoration of Dystrophin Expression into Muscle
    • 10.6. Conclusion
    • Glossary
  • Chapter 11. Gene Therapy for Retinal Disease
    • 11.1. Introduction to the Retina and Inherited Retinal Diseases
    • 11.2. Cell-Specific Targeting within the Retina
    • 11.3. Promoter Choice for Expression in Specific Retinal Cell Targets
    • 11.4. AAV Treatment of Autosomal Recessive Models of Retinal Disease
    • 11.5. AAV Treatment of Autosomal Dominant Models of Retinal Disease
    • 11.6. AAV Delivery of Large Genes to the Retina
    • 11.7. Neuroprotection of the Retina Using AAV
    • 11.8. Human AAV Clinical Trials for the Treatment of IRD
    • 11.9. Summary
  • Chapter 12. Gene Therapy for Hemoglobinopathies: Progress and Challenges
    • 12.1. Why Gene Therapy for Hemoglobinopathies?
    • 12.2. Challenges to Human gene Therapy for Hemoglobinopathies
    • 12.3. Preclinical Studies in Animal Models and Human Cells
    • 12.4. Targeted Reactivation of Fetal Hemoglobin
    • 12.5. Clinical Trials for the Hemoglobinopathies
    • 12.6. Genome Toxicity
    • 12.7. Phenotypic Variability and Gene Transfer in Patients Affected by Hemoglobinopathies
    • 12.8. Future Perspectives
    • 12.9. Conclusion
  • Chapter 13. Hemophilia Gene Therapy
    • 13.1. Introduction
    • 13.2. Hemophilia B Gene Transfer
    • 13.3. AAV and Hemophilia A
    • 13.4. rAAV Dose and the Immune Response
    • 13.5. AAV-Mediated Transfer Lasts a Long Time
    • 13.6. Summary
  • Chapter 14. Gene Transfer for Clinical Congestive Heart Failure
    • 14.1. Introduction
    • 14.2. General Considerations for Cardiac Gene Transfer
    • 14.3. Candidates for CHF Gene Transfer
    • 14.4. Vectors and Methods for Cardiac Gene Transfer
    • 14.5. Gene Transfer Clinical Trials for CHF
    • 14.6. Conclusion
    • Glossary
  • Chapter 15. Gene Therapy for the Prevention of Vein Graft Disease
    • 15.1. Introduction to Vein Graft Disease
    • 15.2. Pathophysiology of Vein Graft Disease
    • 15.3. Gene Delivery Strategies
    • 15.4. Animal Models of Vein Graft Disease
    • 15.5. Gene Targets and Preclinical Studies
    • 15.6. The PREVENT Trials
    • 15.7. Additional Considerations for Translation
    • 15.8. Conclusions
  • Chapter 16. Gene Therapy in Cystic Fibrosis
    • 16.1. A Brief History of Cystic Fibrosis Genetics
    • 16.2. CFTR Mutations
    • 16.3. CF Gene Therapy Challenges
    • 16.4. CF Gene Therapy in Clinical Trials
    • 16.5. Mutant Protein Repair
    • 16.6. Conclusion
    • Glossary
  • Chapter 17. Genetic Engineering of Oncolytic Viruses for Cancer Therapy
    • 17.1. Introduction
    • 17.2. Conditionally Replicating Adenoviruses (CRADs)
    • 17.3. Herpes Simplex Virus (HSV)
    • 17.4. Vaccinia Virus (VV)
    • 17.5. Reovirus Type 3 Dearing (RT3D or Reolysin®)
    • 17.6. Vaccine Strains of Measles Virus (vMV)
    • 17.7. Vesicular Stomatitis Virus (VSV)
    • 17.8. Challenges to Oncolytic Virotherapy
    • 17.9. Conclusions and Future Directions
  • Chapter 18. T Cell-Based Gene Therapy of Cancer
    • 18.1. Introduction: T Cell-Based Immunotherapy
    • 18.2. Ex vivo T Cell Expansion
    • 18.3. Modification Strategies for T Cell Redirection
    • 18.4. Approaches to Enhance T Cell Activity
    • 18.5. Mitigation of Adverse Events and Safety Considerations
    • 18.6. Translation of Engineered T Cell Therapy to the Clinic
    • 18.7. Conclusions and Future Directions
    • Disclosure of Potential Conflicts of Interest
    • Glossary
  • Chapter 19. Current Status of Gene Therapy for Brain Tumors
    • 19.1. Introduction
    • 19.2. Gene Delivery Vehicles for Brain Tumors
    • 19.3. Gene Therapy Strategies for Brain Tumors
    • 19.4. Status of Clinical Trials for GBM
    • 19.5. Current Challenges and Future Directions
  • Index

Details

No. of pages:
346
Language:
English
Copyright:
© Academic Press 2015
Published:
Imprint:
Academic Press
eBook ISBN:
9780128005644
Hardcover ISBN:
9780128005637

About the Editor

Jeffrey Laurence

Jeffrey Laurence

Dr Laurence is pursuing the pathophysiology of cardiovascular and skeletal abnormalities linked to HIV disease and its therapies at Weill Cornell. Dr Laurence is the editor-in-chief of Translational Medicine, which Elsevier co-publishes with the CSCTR.

Affiliations and Expertise

Weill Cornell Medical College, New York, NY, USA

Michael Franklin

Affiliations and Expertise

University of Minnesota, Hematology, Oncology, and Transplantation Department of Medicine, Minneapolis, MN, USA

Reviews

"This is a unique book, not only because of the information it provides, but also because of the way it presents it. The editors have done a wonderful job of recruiting an excellent group of contributors and designing a wonderful book. Score: 100 - 5 Stars" --Doody's

Ratings and Reviews