Safety and Efficacy of Gene-Based Therapeutics for Inherited Disorders

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. Front Matter....Pages i-ix Gene Transfer Strategies and Applications in Genetic Diseases....Pages 1-7 Safety and Efficacy of Retroviral and Lentiviral Vectors for Gene Therapy....Pages 9-35 Adenovirus Vector Toxicity....Pages 37-60 Helper-Dependent Adenoviral Vectors for Gene Therapy of Inherited Diseases....Pages 61-75 AAV Vector-Based Gene Therapy, Progress and Current Challenges....Pages 77-112 Physical Methods of Gene Delivery....Pages 113-135 Designer Effectors for Editing and Regulating Complex Genomes....Pages 137-157 siRNA Therapeutics to Treat Liver Disorders....Pages 159-190 Oligonucleotide Therapy....Pages 191-217 Back Matter....Pages 219-220