The Clinibook

This book provides a 2012 reference for state-of-the-art gene transfer
technology and the different aspects of its clinical translation with a focus
on European-based initiatives. As examples of successful outcomes, recent
clinical 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 cells including induced
pluripotent stem cells (iPS) with integrating vectors such as retrovirus,
lentivirus or transposon-derived systems. The critical path to clinical
implementation is covered in the second part describing currently available
tools - such as molecular 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 ideal for postgraduates, undergraduates,
scientists, clinicians, regulators and patients' advocacy groups looking for
states-of-the-art information as well as emerging prospects - including gene
targeting and homologous recombination - in gene transfer intended for
clinical translation. Dr Odile Cohen-Haguenauer is the Coordinator of
Clingene-NoE, the European Network for the Advancement of Clinical Gene
Transfer and Therapy.