Springer 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. From the Back Cover 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. About the Author 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.