Safety and Efficacy of Gene-Based Therapeutics for Inherited Disorders

Buch | Softcover

IX, 220 Seiten

2018 | 1. Softcover reprint of the original 1st ed. 2017
Springer International Publishing (Verlag)
978-3-319-85158-7 (ISBN)

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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.

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.

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.

Erscheinungsdatum	05.03.2022
Zusatzinfo	IX, 220 p. 32 illus., 19 illus. in color.
Verlagsort	Cham
Sprache	englisch
Maße	155 x 235 mm
Gewicht	361 g
Themenwelt	Medizin / Pharmazie ► Medizinische Fachgebiete ► Pharmakologie / Pharmakotherapie
	Medizin / Pharmazie ► Pharmazie
	Studium ► 2. Studienabschnitt (Klinik) ► Humangenetik
Schlagworte	Adenoviral vector • Antisense oligonucleotide • gene therapy • gene transfer • immune response • Lentiviral vectors • viral gene therapy vectors
ISBN-10	3-319-85158-6 / 3319851586
ISBN-13	978-3-319-85158-7 / 9783319851587
Zustand	Neuware