The CliniBook: clinical gene transfer: state-of-the art / ed. by O.Cohen-Haguenauer. - Paris: Editions EDK; Les Ulis: EDP Siences, 2012. - xxxviii, 545 p.: ill., tab. - Incl. bibl. ref. - Auth. ind.: p.543-545. - ISBN 978-2-8425-4171-2 Шифр: (И/Р25-C61) 02  Место хранения:   02 | Отделение ГПНТБ СО РАН | Новосибирск

Оглавление / Contents

Clinigene partners and boards .................................. IX Aknowledgments ................................................. XV List of authors .............................................. XVII Introduction In-1 Foreword Lucio Luzzatto, Inder M. Verma ........................... 3 In-2 Main achievements and prospects downstream of the CliniGene-NoE Odile Cohen-Haguenauer ................................... 8 Part I: Technologies and pre-clinical studies TECHNOLOGIES - Highlights on aav mediated gene transfer A1-1 Highlights on AAV mediated gene transfer: introduction Eduard Ayuso, Fatima Bosch ................................ 31 Al-2 Preclinical studies of AAV gene therapy for inherited retinal dystrophies Alexander J. Smith, Anastasios Georgiadis, Robin R. Ali ... 35 Al-3 AAV-mediated gene therapy for MPS VI Gabriella Cotugno, Patrizia Annunziata, Mark Haskins, Alberto Auricchio ......................................... 41 Al-4 Microdystrophin and myostatin gene therapy for Duchenne muscular dystrophy using adeno-associated virus vectors Helen Foster, Taeyoung Koo, Alberto Malerba, Susan Jarmin, Takis Athanasopoulos, Keith Foster, George Dickson ................................................... 46 Al-5 AAV gene therapy for cardiovascular disorders Serena Zacchigna, Mauro Giacca ............................ 55 Al-6 AAV gene therapy for diabetes mellitus Eduard Ayuso, Veronica Jimenez, David Callejas, Christopher Mann, Fatima Bosch ............................ 62 Al-7 Approaches to large scale production of AAV-vectors Otto-Wilhelm Merten, Philippe Moullier .................... 71 А1-8 Reference materials for the characterization of adeno- associated viral vectors Eduard Ayuso, Véronique Blouin, Christophe Darmon, Fatima Bosch, Martin Lock, Richard O. Snyder, Philippe Moullier .................................................. 83 TECHNOLOGIES - Retrovirus mediated gene transfer state-of- the-art A2-1 Highlights on retrovirus mediated gene transfer Pedro E. Cruz, Manuel I.T. Carrondo ...................... 93 A2-2 Retroviral vector development: reducing genotoxicity of integrated DNA and creating virus-like particles for transient cell modification Melanie Galla, Tobias Maetzig, Julia D. Suerth, Ute Modlich, Axel Schambach, Christopher Baum ............... 100 A2-3 Replication competent γ-retrovial vectors for tumor therapy Thomas Schaser, Lydia Dürner, Klaus Cichutek, Christian J. Buchholz ................................... 112 A2-4 Modular retroviral producer cell lines 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, Hansjörg Hauser .......... 118 TECHNOLOGIES - Highlights on lentivirus mediated gene transfer A3-1 Introduction Matthias Schweizer, Klaus Cichutek ...................... 127 A3-2 MicroRNAs detargeting technology in the context of CNS applications Angélique Colin, Mathilde Faideau, Noëlle Dufour, Gwennaelle Auregan, Raymonde Hassig, Carole Escartin, Philippe Hantraye, Gilles Bonvento, Nicole Déglon ....... 129 A3-3 Development of SIVsmmPBj vectors for gene transfer into myeloid cells Matthias Schweizer, Klaus Cichutek ...................... 134 A3-4 Insulated retrovirus vectors using novel synthetic genetic insulator elements to circumvent enhancer- mediated genotoxicity Caroline Duros, Alexandre Artus, Odile Cohen- Haguenauer .............................................. 138 A3-5 Facing the challenges of downstream processing of Ientiviral vectors Vanessa Bandeira, Cristina Peixoto, Ana Sofia Coroadinha, Pedro E. Cruz, Manuel I.T. Carrondo, Otto-Wilhelm Merten, Paula Marques Alves ................ 150 A3-6 Restrictions and requirements for stable Ientiviral vector production in HEK293 cells Leonor Gama-Norton, Hansjörg Hauser, Dagmar Wirth ....... 156 АЗ-7 Novel Ientiviral vector pseudotypes for stable gene transfer into resting hematopoietic cells Els Verhoeyen, François-LOïc Cosset ..................... 160 TECHNOLOGIES - Highlights on gene-modified cell therapy A4-1 Cell therapy: introduction Gösta Gahrton ........................................... 185 A4-2 Ex-vivo expansion of human mesenchymal stem cells Pedro E. Cruz, Helder J.S. Cruz, Rita N. Bárcia, Jorge M. Santos, Susanne Pohl, Mari Gilljam, Kurt E.J. Dittmar, Werner Lindenmaier, Evren Alici ................ 187 A4-3 Closed bag cultivation systems for the production of gene modified dendritic cells and MSC for clinical use Werner Lindenmaier, Lars Маске, Wilhelm Meyring, Henk S.P. Garritsen, Kurt E.J. Dittmar, Kristina Lachmann, Michael Thomas ................................ 194 A4-4 Genetically modified NK cells for cancer treatment: facts and visions Evren Alici, Gösta Gahrton .............................. 201 A4-5 Regulatory T lymphocyte depletion for cancer immunotherapies Michelle Rosenzwajg, François Lemoine, David Klatzmann .. 208 A4-6 Gene therapy of Fanconi's anaemia aplastic syndrome Émilie Bayart, Caroline Duros, Alexandre Artus, Stéphanie Lemaire, Odile Cohen-Haguenauer ............... 216 TECHNOLOGIES - Adenovirus mediated gene transfer: current developments A5-1 Overview on adenovirus vectors Stefan Kochanek ......................................... 229 A5-2 Tumour barriers influencing adenovirus vector delivery and therapeutic efficacy Tanja Lucas, Stefan Kochanek ............................ 232 A5-3 Tumor imaging with adenoviral vectors Martina Anton, Bernd Gänsbacher ......................... 238 A5-4 Treatment of brain tumors with adenoviruses: preclinical development Seppo Ylä-Herttuala ..................................... 242 A5-5 Production and purification of Ad vectors: current status and future needs for adenovirus vector production Ana Carina Silva, Daniel Simão, Marcos F.Q. Sousa, Cristina Peixoto, Pedro E. Cruz, Manuel I.T. Carrondo, Paula Marques Alves ..................................... 245 TECHNOLOGIES - Non-viral based gene transfer: a new era A6-1 Non viral plasmid delivery and imaging of transgene expression Pascal Bigey, Michel-Francis Bureau, Gonzalo Cordova, Virginie Escriou, Antoine Kichler, Nathalie Mignet, Daniel Scherman ......................................... 253 A6-2 Overview of novel plasmid vectors and preclinical applications Corinne Marie, Daniel Scherman .......................... 266 A6-3 Fining a gap: S/MAR-based replicating minicircles Niels Heinz, Sandra Broil, Martin Schleef, Christopher Baum, Juergen Bode ...................................... 271 A6-4 Manufacturing and QC of plasmid based vectors Marco Schmeer, Martin Schleef ........................... 277 A6-5 Sleeping Beauty transposon based gene therapy Zsuzsanna Izsvák, Zoltán Ivies .......................... 284 A6-6 Development of minicircle vectors Marco Schmeer, Anja Rischmüller, Martin Schleef ......... 290 A6-7 Exon skipping therapy for DMD using antisense oligomer technology Linda Popplewell, Jagjeet Kang, Alberto Malerba, Keith Foster, George Dickson .................................. 295 TECHNOLOGIES - Highlights on emerging technologies, iPS induction and genetic stability A7-1 Induction of pluripotency from adult somatic cells: a review Émilie Bayart, Odile Cohen-Haguenauer ................... 307 A7-2 Genetic modification of adult stem cells and induced pluripotent stem cells with emerging transposon technologies Thierry Vanden Driessche, Marinee K.L. Chuah ............ 335 A7-3 Targeted genome engineering approaches based on rare- cutting endonucleases: a tentative summary Frédéric Pâques, Julianne Smith ......................... 341 A7-4 Targeted genome modifications with designer nucleases Christien Bednarski, Eva-Maria Händel, Toni Cathomen .... 354 PRE-CLINICAL STUDIES, BIOSAFETY AND ANIMAL MODELS - Preclinical assessment tools Bl-1 Preclinical assessment tools: imaging gene transfer to the brain Yannic Waerzeggers, Thomas Viel, Sonja Schäfers, Parisa Monfared, Alexandra Winkeler, Andreas H. Jacobs .. 371 Bl-2 Persistent luminescence nanoparticles for in vivo imaging: characteristics and targeting Thomas Maldiney, Daniel Scherman, Cyrille Richard Bl-3 Ex-vivo evaluation of gene-transfer vectors: efficacy, tropism and safety Dror Kolodkin-Gal, Shay Tayeb, Abed Khalaileh, Gidi Zamir, Nikolai Kunicher, Amos Panet ..................... 394 PRE-CLINICAL STUDIES, BIOSAFETY AND ANIMAL MODELS - General biosafety: immune responses, immunotoxicity and genotoxicity B2-1 Assessing and taming unwanted immune responses induced by AAV gene transfer: current status, ongoing questions and future prospects Federico Mingozzi, Anne Galy, David Klatzmann ........... 405 B2-2 Predicting immune responses to viral vectors and transgenes in gene therapy and vaccination: the coming of systems biology Bertrand Bellier, Adrien Six, Véronique Thomas-Vaslin, David Klatzmann ......................................... 420 B2-3 Biosafety analysis in preclinical and clinical studies 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, Christof von Kalle ...................................... 432 Part II: Clinical trials and regulatory issues CLINICAL TRIALS C1-1 A clinical trial of AAV-mediated gene therapy for Leber congenital amaurosis 2 Alexander J. Smith, Robin R. Ali ........................ 447 C1-2 Gene therapy for X-linked adrenoleukodystrophy based on lentiviral correction of hematopoietic stem cells Nathalie Cartier, Salima Hacein-Bey-Abina, Cynthia C. Bartholomae, Manfred Schmidt, Christof von Kalle, Pierre Bougnères, Alain Fischer, Marina Cavazzana- Calvo, Patrick Aubourg .................................. 452 Cl-3 Immune reconstitution after gene therapy for adenosine deaminase severe combined immunodeficiency (ADA-SCID) Immacolata Brigida, Alessandro Aiuti .................... 459 С1-4 Gene therapy in Alzheimer disease patients Maria Eriksdotter-Jönhagen, Bengt Linderoth, Per Almqvist, Göran Lind, Helga Eyjolfsdottir, Erik Sundström, Ǻke Seiger, Lars Wahlberg .................... 465 Cl-5 Cardiovascular gene therapy trials Seppo Ylä-Herttuala ..................................... 475 Cl-6 AAV-mediated gene therapy for haemophilia В Deepak Raj, Edward G.D. Tuddenham, Arthur W. Nienhuis, Ulrike Reiss, Andrew M. Davidoff, Amit C. Nathwani ...... 479 Cl-7 ProSavin®: a Ientiviral vector approach for the treatment of Parkinson's disease Stéphane Раlfi, R. Scott Ralph, Kyriacos Mitrophanous ... 486 ETHICAL AND REGULATORY ISSUES C2-1 Ethics in translation from research to therapy Nancy M.P. King, Odile Cohen-Haguenauer, Alastair Kent .. 495 C2-2 Centralised regulation of gene therapy in Europe Odile Cohen-Haguenauer .................................. 504 C2-3 The necessity for data sharing towards advancement of clinical translation: building up sample IMPD and substantiating master files Odile Cohen-Haguenauer .................................. 517 INTEGRATION AND DISSEMINATION C3-1 European Union support to gene transfer and gene therapy Ruxandra Draghia-Akli ................................... 531 C3-2 Database of clinical trials Bernd Gänsbacher ........................................ 533 C3-3 CliniGene and ESGCT shared vision for gene therapy in Europe: past, present and future prospects Thierry Vanden Driessche, Bernd Gänsbacher, George Dickson, David Klatzmann, Seppo Ylä-Herttuala, Luigi Naldini, Alastair Kent, Odile Cohen-Haguenauer .......... 536 AUTHOR INDEX .................................................. 541