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Safety and Efficacy of Gene-Based Therapeutics for Inherited Disorders

Editat de Nicola Brunetti-Pierri
en Limba Engleză Paperback – 7 mai 2018
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.
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Specificații

ISBN-13: 9783319851587
ISBN-10: 3319851586
Pagini: 220
Ilustrații: IX, 220 p. 32 illus., 19 illus. in color.
Dimensiuni: 155 x 235 mm
Greutate: 0.33 kg
Ediția:Softcover reprint of the original 1st ed. 2017
Editura: Springer International Publishing
Colecția Springer
Locul publicării:Cham, Switzerland

Cuprins

Overview: gene transfer strategies, principles, applications.- Manufacturing viral gene therapy vectors: general approaches and challenges.- Retrovirus- and lentivirus-based vectors.- Preclinical and clinical applications of retroviral vectors.- Preclinical and clinical applications of lentiviral vectors.- Retrovirus and lentivirus integration.- Adenovirus-based vectors for gene therapy.- Adenoviral vector-host interactions.- Helper-dependent adenoviral vectors.- Gene therapy for cancer treatment.- Oncolytic adenoviruses for cancer treatment.- Vaccination by gene transfer vectors.- AAV vectors: general features and applications.- Adaptive immune response to viral vector delivery.- Herpes viruses: general features and applications.- RNA interference-based strategy for treatment of human diseases.- Antisense oligonucleotide based therapeutics.- Gene editing strategies.- Nonviral vectors.

Notă biografică

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.

Textul de pe ultima copertă

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.

Caracteristici

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