The CliniBook - Clinical Gene Transfer: State of the Art

This book provides a 2012 reference for state-of-the-art gene transfer technology and the different aspects of its clinicat translation with a focus on European-based initiatives. As examples of successful outcomes, recent clinicat trials are presented together with Ethical, Safety and Regulatory issues, which are discussed. The broad range of various technologies is covered whether addressing direct in vivo gene transfer like with AAV, Adeno or non-viral vectors or ex-vivo genetically engineered cens inctuding induced pluripotent stem cens IiPSI with integrating vectors such as retrovirus, lentivirus or transposon-derived systems.
The criticat path to clinicat implementation is covered in the second part describing currently avaitable tools - such as molecutar imaging, ex-vivo organ cultures and high-throughput technologies used for evaluation of criteria towards a go-or-no go decision to move to the clinic; in addition, utmost salient biosafety and immunotoxicology aspects are discussed. This book is ideat for postgraduates, undergraduates, scientists, clinicians, regulators and patients' advocacy groups looking for state-of-the-art information as well as emerging prospects - inctuding gene targeting and homotogous recombination - in gene transfer intended for clinicat translation.