Safety and Efficacy of Gene-Based Therapeutics for Inherited Disorders - Brunetti-Pierri, Nicola; SpringerLink (Online service) (Cham : Springer International Publishing : Imprint: Springer, 2017)
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.
Keywords: Pharmacology; Gene therapy; Molecular biology; Pharmacology/Toxicology.; Gene Therapy; Molecular Medicine