Overview
A comprehensive review of different vector platforms by international experts in the field
Highlights advances on gene transfer vectors, their disease applications, and their toxicity profiles
Provides detailed discussions of vector type, the biomedical knowledge of the disease targets, and the complexities and toxicology of gene delivery
Includes supplementary material: sn.pub/extras
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Table of contents (9 chapters)
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Front Matter
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Back Matter
Keywords
About this book
In this book, leading international experts analyze state-of-the-art advances in gene transfer vectors for applications in inherited disorders and also examine the toxicity profiles of these methods. The authors discuss the strengths and weaknesses of available vectors in the clinical setting, and specifically focus on the challenges and possible solutions that researchers are testing in order to improve the safety of gene therapy for genetic diseases. This comprehensive and authoritative overview of vector development is a necessary text for researchers, toxicologists, pharmacologists, molecular biologists, physicians, and students in these fields.
Editors and Affiliations
About the editor
Nicola Brunetti-Pierri graduated in Medicine from Federico II University of Naples, Italy. After his residency in Pediatrics at Federico II University of Naples, he moved to Baylor College of Medicine, Houston, USA for a post-doctoral research fellowship and clinical trainings in medical genetics and biochemical genetics. He is currently an Associate Professor of Pediatrics at Federico II University, Naples, Italy and an Associate Investigator at the Telethon Institute of Genetics and Medicine (TIGEM), Pozzuoli, Italy. As a pediatrician and geneticist, he studies genetic diseases and inborn errors of metabolism and his research focuses at developing novel therapies for these disorders.
Bibliographic Information
Book Title: Safety and Efficacy of Gene-Based Therapeutics for Inherited Disorders
Editors: Nicola Brunetti-Pierri
DOI: https://doi.org/10.1007/978-3-319-53457-2
Publisher: Springer Cham
eBook Packages: Biomedical and Life Sciences, Biomedical and Life Sciences (R0)
Copyright Information: Springer International Publishing AG 2017
Hardcover ISBN: 978-3-319-53455-8Published: 01 June 2017
Softcover ISBN: 978-3-319-85158-7Published: 08 May 2018
eBook ISBN: 978-3-319-53457-2Published: 22 May 2017
Edition Number: 1
Number of Pages: IX, 220
Number of Illustrations: 13 b/w illustrations, 19 illustrations in colour
Topics: Pharmacology/Toxicology, Gene Therapy, Molecular Medicine