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Translating Gene Therapy to the Clinic
Techniques and Approaches
1st Edition - November 14, 2014
Editors: Jeffrey Laurence, Michael Franklin
Language: English
Hardback ISBN:9780128005637
9 7 8 - 0 - 1 2 - 8 0 0 5 6 3 - 7
eBook ISBN:9780128005644
9 7 8 - 0 - 1 2 - 8 0 0 5 6 4 - 4
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 emphasi…Read more
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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.
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
Translational researchers in both academia and industry as well as clinicians, some grad students and regulators and educators globally
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
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
No. of pages: 346
Language: English
Edition: 1
Published: November 14, 2014
Imprint: Academic Press
Hardback ISBN: 9780128005637
eBook ISBN: 9780128005644
JL
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
MF
Michael Franklin
Affiliations and expertise
University of Minnesota, Hematology, Oncology, and Transplantation Department of Medicine, Minneapolis, MN, USA
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