Translating Gene Therapy to the Clinic: Techniques and Approaches

by Jeffrey Laurence (Editor), Michael Franklin (Editor)

Overview

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

Product Details

ISBN-13:	9780128005644
Publisher:	Elsevier Science
Publication date:	11/14/2014
Sold by:	Barnes & Noble
Format:	eBook
Pages:	346
File size:	13 MB
Note:	This product may take a few minutes to download.

About the Author

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.

Table of Contents

1. Introduction
2. Translating genome engineering to better clinical outcomes
3. Arthritis Gene Therapy and Its Tortuous Path into the Clinic
4. Mechanistic Basis of Immunotherapies for Type 1 Diabetes Mellitus
5. Gene therapy for retinal disease
6. Gene Therapy in Cystic Fibrosis
7. Evaluating Risks of Insertional Mutagenesis by DNA Transposons in Gene Therapy
8. Pluripotent Stem Cells and Gene Therapy
9. Gene therapy for hemoglobinopathies: progress and challenge
10. Hemophilia Clinical Gene Therapy-brief review
11. Gene Transfer for Congestive Heart Failure
12. Gene therapy for the prevention of vein graft disease
13. Current status of gene therapy for brain tumors
14. Oncolytic virus therapy for cancer: The first wave of translational clinical trials
15. T-Cell Gene Therapy
16. Immune responses to gene therapy

What People are Saying About This

From the Publisher

Reviews the latest research in gene transfer, genome engineering and clinical trials, as gene-based therapeutic techniques move from the lab to the clinic

Editorial Reviews

Reviewer: Luis F Escobar, MD, MS (Peyton Manning Children's Hospital)
Description: Gene therapy is an active field of research, but in recent years it has also become daily clinical practice in multiple centers around the world. This masterfully edited book, part of the Advances in Translational Medicine series, collects the work of many distinguished contributors in the area of gene therapy.
Purpose: The goal is to present updated information on gene therapy and provide the link between the basic science in gene therapy and its applicability to clinical practice. The authors have accomplished the difficult task of translating the complexity of gene therapy science into a more clinical perspective.
Audience: The information this book provides is of importance not only to early students in basic genetic science, but also to medical genetics clinicians who may have to educate patients on the utility of gene therapy. This book may not be suitable for undergraduate students, but it is essential information for midlevel trainees in the clinical sciences and it should stimulate the minds of young scientists interested in the field of gene therapy.
Features: The book provides reviews on well selected areas. Of particular interest to me is the chapter on translating genome engineering to survival. The author discusses the basic concepts of gene therapy and the relevance to current clinical practice. The chapter on pluripotent stem cells is unique, as there are very few current reviews in this area. Each chapter addresses the applicability of gene therapy to a different disorder such as cystic fibrosis, hemoglobinopathies, and retinal disease. The format of the book is traditional, but the design and the magnificent illustrations make it extremely attractive. The straightforward writing style and clear language make the book easy to read. The list of abbreviations at the beginning of each chapter is extremely helpful for readers not familiar with gene therapy terminology
Assessment: 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.

Doody's Review Service

"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

From the Publisher