LEADER 03313nam 2200637 a 450 001 9910792058803321 005 20200520144314.0 010 $a1-299-27682-2 010 $a2-84254-237-1 024 7 $a10.1051/978-2-84254-237-5 035 $a(CKB)2560000000099461 035 $a(EBL)3155343 035 $a(SSID)ssj0000906519 035 $a(PQKBManifestationID)11536131 035 $a(PQKBTitleCode)TC0000906519 035 $a(PQKBWorkID)10843950 035 $a(PQKB)10824750 035 $a(MiAaPQ)EBC3155343 035 $a(Au-PeEL)EBL3155343 035 $a(CaPaEBR)ebr10667713 035 $a(CaONFJC)MIL458932 035 $a(OCoLC)922991221 035 $a(DE-B1597)634993 035 $a(DE-B1597)9782842542375 035 $a(MiAaPQ)EBC7156049 035 $a(Au-PeEL)EBL7156049 035 $a(OCoLC)1355220062 035 $a(OCoLC)834607434 035 $a(PPN)176628622 035 $a(EXLCZ)992560000000099461 100 $a20130313d2012 uy 0 101 0 $aeng 135 $aur|n|---||||| 181 $ctxt 182 $cc 183 $acr 200 04$aThe clinibook$b[electronic resource] $eclinical gene transfer : state of the art /$fedited by Odile Cohen-Haguenauer 210 $aParis $cEDP Sciences$d2012 215 $a1 online resource (589 p.) 300 $aDescription based upon print version of record. 311 $a2-84254-171-5 320 $aIncludes bibliographical references and index. 327 $apt. 1. Technologies and pre-clinical studies -- pt. 2. Clinical trials and regulatory issues. 330 $aThis 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. 606 $aGenetic transformation 615 0$aGenetic transformation. 676 $a571.96 700 $aCohen-Haguenauer$b Odile, $4aut$4http://id.loc.gov/vocabulary/relators/aut$01087562 701 $aCohen-Haguenauer$b Odile$01087562 801 0$bMiAaPQ 801 1$bMiAaPQ 801 2$bMiAaPQ 906 $aBOOK 912 $a9910792058803321 996 $aThe clinibook$93823776 997 $aUNINA