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Safety and Efficacy of Gene-Based Therapeutics for Inherited Disorders
Title
Safety and Efficacy of Gene-Based Therapeutics for Inherited Disorders [electronic resource] / edited by Nicola Brunetti-Pierri.
ISBN
9783319534572
Publication
Cham : Springer International Publishing : Imprint: Springer, 2017.
Physical Description
IX, 220 p. 32 illus., 19 illus. in color : online resource.
Local Notes
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Access restricted by licensing agreement.
Summary
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.
Variant and related titles
Springer ebooks.
Other formats
Printed edition:
Format
Books / Online
Language
English
Added to Catalog
June 02, 2017
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.
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