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Full Description
Pharmacology and Toxicology in Therapeutic Gene Transfer provides essential concepts and methodology to understand the pharmacology (active dose, biodistribution, potency, persistence) and toxicology (toxicity, dose finding, vector-host interactions, side effects, genotoxicity) of therapeutic gene transfer. The book focuses on the two most commonly used gene transfer vectors, AAV vectors for in vivo gene delivery and retroviral vectors for ex vivo gene transfer in stem cells. The different chapters introduce the basic biology of the interactions between the two vector system and the human host, and they describe how to design pre-clinical studies and how to monitor efficacy and toxicity in patients, including a description of the most important methods and assays. At present, there is no book or manual that cover these aspects in a comprehensive fashion, and that puts these relatively complex concepts in the context of human gene therapy trials.
Contents
Introduction.- 1. Toxicology and Biodistribution.- 2. Design of Standard Toxicology and Biodistribution Studies.- 3. Toxicology Methods.- 4. Emerging Issues in AAV-Mediated in Vivo Gene Therapy.- 5. Retroviral Vectors-Host Genome Interaction.- 6. Analyzing the Genotoxity of Retroviral Vectors.- 7. Genome Editing.
