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I |
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IX |
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XV |
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XVII |
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XXXIII |
INTRODUCTION
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Lucio Luzzatto and Inder M. Verma
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1 |
Odile Cohen-Haguenauer
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8 |
PART I: TECHNOLOGIES AND PRE-CLINICAL STUDIES
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TECHNOLOGIES - Highlights on AAV mediated gene transfer
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Eduard Ayuso and Fatima Bosch
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29 |
Alexander J. Smith, Anastasios Georgiadis and Robin R. Ali
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35 |
Gabriella Cotugno, Patrizia Annunziata, Mark Haskins and Alberto Auricchio
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41 |
Helen Foster, Taeyoung Koo, Alberto Malerba, Susan Jarmin, Takis Athanasopoulos, Keith Foster and George Dickson
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46 |
Serena Zacchigna and Mauro Giacca
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55 |
Eduard Ayuso, Veronica Jimenez, David Callejas, Christopher Mann and Fatima Bosch
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62 |
Otto-Wilhelm Merten and Philippe Moullier
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71 |
Eduard Ayuso, Véronique Blouin, Christophe Darmon, Fatima Bosch, Martin Lock, Richard O. Snyder and Philippe Moullier
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83 |
TECHNOLOGIES - Retrovirus mediated gene transfer state-of-the-art
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Pedro E. Cruz and Manuel J.T. Carrondo
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91 |
Melanie Galla, Tobias Maetzig, Julia D. Suerth, Ute Modlich, Axel Schambach and Christopher Baum
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100 |
Thomas Schaser, Lydia Dürner, Klaus Cichutek and Christian J. Buchholz
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112 |
Ana Sofia Coroadinha, Dagmar Wirth, Ana F. Rodrigues, Leonor Gama-Norton, Caroline Duros, Alexandre Artus, Odile Cohen-Haguenauer, Paula Marques Alves, Pedro E. Cruz, Manuel J.T. Carrondo and Hansjörg Hauser
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118 |
TECHNOLOGIES - Highlights on lentivirus mediated gene transfer
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Matthias Schweizer and Klaus Cichutek
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125 |
Angélique Colin, Mathilde Faideau, Noëlle Dufour, Gwennaelle Auregan, Raymonde Hassig, Carole Escartin, Philippe Hantraye, Gilles Bonvento and Nicole Déglon
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129 |
Matthias Schweizer and Klaus Cichutek
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134 |
Caroline Duros, Alexandre Artus and Odile Cohen-Haguenauer
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138 |
Vanessa Bandeira, Cristina Peixoto, Ana Sofia Coroadinha, Pedro E. Cruz, Manuel J.T. Carrondo, Otto-Wilhelm Merten and Paula Marques Alves
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150 |
Leonor Gama-Norton, Hansjörg Hauser and Dagmar Wirth
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156 |
Els Verhoeyen and François-Loïc Cosset
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160 |
TECHNOLOGIES - Highlights on gene-modified cell therapy
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Gösta Gahrton
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183 |
Pedro E. Cruz, Helder J.S Cruz, Rita N. Bárcia, Jorge M. Santos, Susanne Pohl, Mari Gilljam, Kurt E.J. Dittmar, Werner Lindenmaier and Evren Alici
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187 |
Werner Lindenmaier, Lars Macke, Wilhelm Meyring, Henk S.P. Garritsen, Kurt E.J. Dittmar, Kristina Lachmann and Michael Thomas
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194 |
Evren Alici and Gösta Gahrton
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201 |
Michelle Rosenzwajg, François Lemoine and David Klatzmann
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208 |
Émilie Bayart, Caroline Duros, Alexandre Artus, Stéphanie Lemaire and Odile Cohen-Haguenauer
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216 |
TECHNOLOGIES - Adenovirus mediated gene transfer: current developments
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Stefan Kochanek
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227 |
Tanja Lucas and Stefan Kochanek
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232 |
Martina Anton and Bernd Gänsbacher
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238 |
Seppo Ylä-Herttuala
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242 |
Ana Carina Silva, Daniel Simão, Marcos F.Q. Sousa, Cristina Peixoto, Pedro E. Cruz, Manuel J.T. Carrondo and Paula Marques Alves
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245 |
TECHNOLOGIES - Non-viral based gene transfer: a new era
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Pascal Bigey, Michel-Francis Bureau, Gonzalo Cordova, Virginie Escriou, Antoine Kichler, Nathalie Mignet and Daniel Scherman
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251 |
Corinne Marie and Daniel Scherman
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266 |
Niels Heinz, Sandra Broll, Martin Schleef, Christopher Baum and Juergen Bode
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271 |
Marco Schmeer and Martin Schleef
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277 |
Zsuzsanna Izsvák and Zoltán Ivics
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284 |
Marco Schmeer, Anja Rischmüller and Martin Schleef
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290 |
Linda Popplewell, Jagjeet Kang, Alberto Malerba, Keith Foster and George Dickson
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295 |
TECHNOLOGIES - Highlights on emerging technologies, iPS induction and genetic stability
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Émilie Bayart and Odile Cohen-Haguenauer
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305 |
Thierry VandenDriessche and Marinee K.L. Chuah
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335 |
Frédéric Pâques and Julianne Smith
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341 |
Christien Bednarski, Eva-Maria Händel and Toni Cathomen
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354 |
PRE-CLINICAL STUDIES, BIOSAFETY AND ANIMAL MODELS - Preclinical assessment tools
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Yannic Waerzeggers, Thomas Viel, Sonja Schäfers, Parisa Monfared, Alexandra Winkeler and Andreas H. Jacobs
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369 |
Thomas Maldiney, Daniel Scherman and Cyrille Richard
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386 |
Dror Kolodkin-Gal, Shay Tayeb, Abed Khalaileh, Gidi Zamir, Nikolai Kunicher and Amos Panet
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394 |
PRE-CLINICAL STUDIES, BIOSAFETY AND ANIMAL MODELS - General biosafety: immune responses, immunotoxicity and genotoxicity
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Federico Mingozzi, Anne Galy and David Klatzmann
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403 |
Bertrand Bellier, Adrien Six, Véronique Thomas-Vaslin and David Klatzmann
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420 |
Manfred Schmidt, Stephanie Laufs, Alessandro Aiuti, Patrick Aubourg, Christopher Baum, Luca Biasco, Nathalie Cartier, Hansjörg Hauser, Eugenio Montini, Philippe Moullier, Richard O. Snyder, Dagmar Wirth and Christof von Kalle
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432 |
PART II Clinical trials and regulatory issues
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CLINICAL TRIALS
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Alexander J Smith and Robin R. Ali
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443 |
Nathalie Cartier, Salima Hacein-Bey-Abina, Cynthia C. Bartholomae, Manfred Schmidt, Christof von Kalle, Pierre Bougnères, Alain Fischer, Marina Cavazzana-Calvo and Patrick Aubourg
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452 |
Immacolata Brigida and Alessandro Aiuti
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459 |
Maria Eriksdotter-Jönhagen, Bengt Linderoth, Per Almqvist, Göran Lind, Helga Eyjolfsdottir, Erik Sundström, Åke Seiger and Lars Wahlberg
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465 |
Seppo Ylä-Herttuala
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475 |
Deepak Raj, Edward G.D. Tuddenham, Arthur W. Nienhuis, Ulrike Reiss, Andrew M. Davidoff and Amit C. Nathwani
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479 |
Stéphane Palfi, R. Scott Ralph and Kyriacos Mitrophanous
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486 |
ETHICAL AND REGULATORY ISSUES
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Nancy M.P. King, Odile Cohen-Haguenauer and Alastair Kent
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493 |
Odile Cohen-Haguenauer
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504 |
Odile Cohen-Haguenauer
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517 |
INTEGRATION AND DISSEMINATION
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Ruxandra Draghia-Akli
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529 |
Bernd Gänsbacher
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533 |
Thierry VandenDriessche, Bernd Gänsbacher, George Dickson, David Klatzmann, Seppo Ylä-Herttuala, Luigi Naldini, Alastair Kent and Odile Cohen-Haguenauer
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536 |
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541 |