Safety and Efficacy of Gene-Based Therapeutics for Inherited 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.

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• Terapia genética
• Pesquisa médica
• Farmacologia

Adenoviral vector;Antisense oligonucleotide;gene transfer;immune response;lentiviral vectors;viral gene therapy vectors;gene therapy