Safety and Efficacy of Gene-Based Therapeutics for Inherited Disorders

Häftad, Engelska, 2018

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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.

Produktinformation

Utgivningsdatum2018-05-08
Mått155 x 235 x 13 mm
Vikt359 g
FormatHäftad
SpråkEngelska
Antal sidor220
FörlagSpringer International Publishing AG
ISBN9783319851587

Tillhör följande kategorier

Medicinsk genetik inom Medicin
Medicinsk utrustning och medicinska tekniker inom Medicin
Farmakologi inom Medicin

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.