Safety and Efficacy of Gene-Based Therapeutics for Inherited Disorders

• Corporate Author: SpringerLink (Online service)
• Other Authors: Brunetti-Pierri, Nicola (Editor)
• Format: Electronic eBook
• Language: English
• Published: Cham : Springer International Publishing : Imprint: Springer, 2017.
• Subjects: Medicine.; Gene therapy.; Molecular biology.; Pharmacology.; Biomedicine.; Pharmacology/Toxicology.; Gene Therapy.; Molecular Medicine.

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 s...

Table of Contents:

• 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.