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ОбложкаThe 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

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