> Detailanzeige
Cohen-Haguenauer, Odile
[VerfasserIn]
;
Aiuti, Alessandro
[MitwirkendeR];
Ali, Robin R.
[MitwirkendeR];
Alici, Evren
[MitwirkendeR];
Almqvist, Per
[MitwirkendeR];
Annunziata, Patrizia
[MitwirkendeR];
Anton, Martina
[MitwirkendeR];
Artus, Alexandre
[MitwirkendeR];
Athanasopoulos, Takis
[MitwirkendeR];
Aubourg, Patrick
[MitwirkendeR];
Auregan, Gwennaelle
[MitwirkendeR];
Auricchio, Alberto
[MitwirkendeR];
Bandeira, Vanessa
[MitwirkendeR];
Bartholomae, Cynthia C.
[MitwirkendeR];
Baum, Christopher
[MitwirkendeR];
Bayart, Émilie
[MitwirkendeR];
Bednarski, Christien
[MitwirkendeR];
Bellier, Bertrand
[MitwirkendeR];
Biasco, Luca
[MitwirkendeR];
Bigey, Pascal
[MitwirkendeR];
Blouin, Véronique
[MitwirkendeR];
Bode, Juergen
[MitwirkendeR];
Bonvento, Gilles
[MitwirkendeR];
Bosch, Fatima
[MitwirkendeR];
Bougnères, Pierre
[MitwirkendeR];
[...]
The CliniBook
: Clinical gene transfer state of the art
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- Medientyp: E-Book
- Titel: The CliniBook : Clinical gene transfer state of the art
-
Enthält:
Frontmatter
CliniGene Partners and Boards
ACKNOWLEDGMENTS
List of authors
Contents
INTRODUCTION
In-1 Foreword
In-2 Main achievements and prospects downstream of the CliniGene-NoE
PART I: TECHNOLOGIES AND PRE-CLINICAL STUDIES
TECHNOLOGIES - Highlights on AAV mediated gene transfer
A1-1 Highlights on AAV mediated gene transfer: introduction
A1-2 Preclinical studies of AAV gene therapy for inherited retinal dystrophies
A1-3 AAV-mediated gene therapy for MPS VI
A1-4 Microdystrophin and myostatin gene therapy for Duchenne muscular dystrophy using adeno-associated virus vectors
A1-5 AAV gene therapy for cardiovascular disorders
A1-6 AAV gene therapy for diabetes mellitus
A1-7 Approaches to large scale production of AAV-vectors
A1-8 Reference materials for the characterization of adeno-associated viral vectors
TECHNOLOGIES - Retrovirus mediated gene transfer state-of-the-art
A2-1 Highlights on retrovirus mediated gene transfer
A2-2 Retroviral vector development: reducing genotoxicity of integrated DNA and creating virus-like particles for transient cell modification
A2-3 Replication-competent y-retroviral vectors for tumor therapy
A2-4 Modular retroviral producer cell lines
TECHNOLOGIES - Highlights on lentivirus mediated gene transfer
A3-1 Introduction
A3-2 MicroRNAs detargeting technology in the context of CNS applications
A3-3 Development of SIVsmmPBj vectors for gene transfer into myeloid cells
A3-4 Insulated retrovirus vectors using novel synthetic genetic insulator elements to circumvent enhancer-mediated genotoxicity
A3-5 Facing the challenges of downstream processing of lentiviral vectors
A3-6 Restrictions and requirements for stable lentiviral vector production in HEK293 cells
A3-7 Novel lentiviral vector pseudotypes for stable gene transfer into resting hematopoietic cells
TECHNOLOGIES - Highlights on gene-modified cell therapy
A4-1 Cell therapy Introduction
A4-2 Ex-vivo expansion of human mesenchymal stem cells
A4-3 Closed bag cultivation systems for the production of gene modified dendritic cells and MSC for clinical use
A4-4 Genetically modified NK cells for cancer treatment: facts and visions
A4-5 Regulatory T lymphocyte depletion for cancer immunotherapies
A4-6 Gene therapy of Fanconi’s anaemia aplastic syndrome
TECHNOLOGIES - Adenovirus mediated gene transfer: current developments
A5-1 Overview on adenovirus vectors
A5-2 Tumour barriers influencing adenovirus vector delivery and therapeutic efficacy
A5-3 Tumor imaging with adenoviral vectors
A5-4 Treatment of brain tumors with adenoviruses: preclinical development
A5-5 Production and purification of Ad vectors: current status and future needs for adenovirus vector production
TECHNOLOGIES - Non-viral based gene transfer: a new era
A6-1 Non viral plasmid delivery and imaging of transgene expression A6-1 Non viral plasmid delivery and imaging of transgene expression
A6-2 Overview of novel plasmid vectors and preclinical applications
A6-3 Filling a gap: S/MAR-based replicating minicircles
A6-4 Manufacturing and QC of plasmid based vectors
A6-5 Sleeping Beauty transposon based gene therapy
A6-6 Development of minicircle vectors
A6-7 Exon skipping therapy for DMD using antisense oligomer technology
TECHNOLOGIES - Highlights on emerging technologies, iPS induction and genetic stability
A7-1 Induction of pluripotency from adult somatic cells: a review
A7-2 Genetic modification of adult stem cells and induced pluripotent stem cells with emerging transposon technologies
A7-3 Targeted genome engineering approaches based on rare-cutting endonucleases: a tentative summary
A7-4 Targeted genome modifications with designer nucleases
B1-1 Preclinical assessment tools: imaging gene transfer to the brain
B1-2 Persistent luminescence nanoparticles for in vivo imaging: characteristics and targeting
B1-3 Ex-vivo evaluation of gene-transfer vectors: efficacy, tropism and safety
B2-1 Assessing and taming unwanted immune responses induced by AAV gene transfer: current status, ongoing questions and future prospects
B2-2 Predicting immune responses to viral vectors and transgenes in gene therapy and vaccination: the coming of systems biology
B2-3 Biosafety analysis in preclinical and clinical studies
PART II Clinical trials and regulatory issues
CLINICAL TRIALS
C1-1 A clinical trial of AAV-mediated gene therapy for Leber congenital amaurosis 2
C1-2 Gene therapy for X-linked adrenoleukodystrophy based on lentiviral correction of hematopoietic stem cells
C1-3 Immune reconstitution after gene therapy for adenosine deaminase severe combined immunodeficiency (ADA-SCID)
C1-4 Gene therapy in Alzheimer disease patients
C1-5 Cardiovascular gene therapy trials
C1-6 AAV-mediated gene therapy for haemophilia B
C1-7 ProSavin®: a lentiviral vector approach for the treatment of Parkinson’s disease
ETHICAL AND REGULATORY ISSUES
C2-1 Ethics in translation from research to therapy
C2-2 Centralised regulation of gene therapy in Europe
C2-3 The necessity for data sharing towards advancement of clinical translation Building up sample IMPD* and substantiating master files
INTEGRATION AND DISSEMINATION
C3-1 European Union support to gene transfer and gene therapy
C3-2 Database of clinical trials
C3-3 CliniGene and ESGCT shared vision for gene therapy in Europe: past, present and future prospects
AUTHOR INDEX
- Beteiligte: Cohen-Haguenauer, Odile [VerfasserIn]; Aiuti, Alessandro [MitwirkendeR]; Ali, Robin R. [MitwirkendeR]; Alici, Evren [MitwirkendeR]; Almqvist, Per [MitwirkendeR]; Annunziata, Patrizia [MitwirkendeR]; Anton, Martina [MitwirkendeR]; Artus, Alexandre [MitwirkendeR]; Athanasopoulos, Takis [MitwirkendeR]; Aubourg, Patrick [MitwirkendeR]; Auregan, Gwennaelle [MitwirkendeR]; Auricchio, Alberto [MitwirkendeR]; Bandeira, Vanessa [MitwirkendeR]; Bartholomae, Cynthia C. [MitwirkendeR]; Baum, Christopher [MitwirkendeR]; Bayart, Émilie [MitwirkendeR]; Bednarski, Christien [MitwirkendeR]; Bellier, Bertrand [MitwirkendeR]; Biasco, Luca [MitwirkendeR]; Bigey, Pascal [MitwirkendeR]; Blouin, Véronique [MitwirkendeR]; Bode, Juergen [MitwirkendeR]; Bonvento, Gilles [MitwirkendeR]; Bosch, Fatima [MitwirkendeR]; Bougnères, Pierre [MitwirkendeR]; Brigida, Immacolata [MitwirkendeR]; Broll, Sandra [MitwirkendeR]; Buchholz, Christian J. [MitwirkendeR]; Bureau, Michel-Francis [MitwirkendeR]; Bárcia, Rita N. [MitwirkendeR]; Callejas, David [MitwirkendeR]; Carina Silva, Ana [MitwirkendeR]; Carrondo, Manuel J.T. [MitwirkendeR]; Cartier, Nathalie [MitwirkendeR]; Cathomen, Toni [MitwirkendeR]; Cavazzana-Calvo, Marina [MitwirkendeR]; Chuah, Marinee K.L. [MitwirkendeR]; Cichutek, Klaus [MitwirkendeR]; Cohen-Haguenauer, Odile [MitwirkendeR]; Colin, Angélique [MitwirkendeR]; Cordova, Gonzalo [MitwirkendeR]; Coroadinha, Ana Sofia [MitwirkendeR]; Cosset, François-Loïc [MitwirkendeR]; Cotugno, Gabriella [MitwirkendeR]; Cruz, Helder J.S [MitwirkendeR]; Cruz, Pedro E. [MitwirkendeR]; Darmon, Christophe [MitwirkendeR]; Davidoff, Andrew M. [MitwirkendeR]; Dickson, George [MitwirkendeR]; Dittmar, Kurt E.J. [MitwirkendeR]; Draghia-Akli, Ruxandra [MitwirkendeR]; Dufour, Noëlle [MitwirkendeR]; Duros, Caroline [MitwirkendeR]; Déglon, Nicole [MitwirkendeR]; Dürner, Lydia [MitwirkendeR]; Eriksdotter-Jönhagen, Maria [MitwirkendeR]; Escartin, Carole [MitwirkendeR]; Escriou, Virginie [MitwirkendeR]; Eyjolfsdottir, Helga [MitwirkendeR]; Faideau, Mathilde [MitwirkendeR]; Fischer, Alain [MitwirkendeR]; Foster, Helen [MitwirkendeR]; Foster, Keith [MitwirkendeR]; Gahrton, Gösta [MitwirkendeR]; Galla, Melanie [MitwirkendeR]; Galy, Anne [MitwirkendeR]; Gama-Norton, Leonor [MitwirkendeR]; Garritsen, Henk S.P. [MitwirkendeR]; Georgiadis, Anastasios [MitwirkendeR]; Giacca, Mauro [MitwirkendeR]; Gilljam, Mari [MitwirkendeR]; Gänsbacher, Bernd [MitwirkendeR]; Hacein-Bey-Abina, Salima [MitwirkendeR]; Hantraye, Philippe [MitwirkendeR]; Haskins, Mark [MitwirkendeR]; Hassig, Raymonde [MitwirkendeR]; Hauser, Hansjörg [MitwirkendeR]; Heinz, Niels [MitwirkendeR]; Händel, Eva-Maria [MitwirkendeR]; Ivics, Zoltán [MitwirkendeR]; Izsvák, Zsuzsanna [MitwirkendeR]; Jacobs, Andreas H. [MitwirkendeR]; Jarmin, Susan [MitwirkendeR]; Jimenez, Veronica [MitwirkendeR]; Kalle, Christof von [MitwirkendeR]; Kang, Jagjeet [MitwirkendeR]; Kent, Alastair [MitwirkendeR]; Khalaileh, Abed [MitwirkendeR]; Kichler, Antoine [MitwirkendeR]; King, Nancy M.P. [MitwirkendeR]; Klatzmann, David [MitwirkendeR]; Kochanek, Stefan [MitwirkendeR]; Kolodkin-Gal, Dror [MitwirkendeR]; Koo, Taeyoung [MitwirkendeR]; Kunicher, Nikolai [MitwirkendeR]; Lachmann, Kristina [MitwirkendeR]; Laufs, Stephanie [MitwirkendeR]; Lemaire, Stéphanie [MitwirkendeR]; Lemoine, François [MitwirkendeR]; Lind, Göran [MitwirkendeR]; Lindenmaier, Werner [MitwirkendeR]; Linderoth, Bengt [MitwirkendeR]; Lock, Martin [MitwirkendeR]; Lucas, Tanja [MitwirkendeR]; Luzzatto, Lucio [MitwirkendeR]; Macke, Lars [MitwirkendeR]; Maetzig, Tobias [MitwirkendeR]; Maldiney, Thomas [MitwirkendeR]; Malerba, Alberto [MitwirkendeR]; Mann, Christopher [MitwirkendeR]; Marie, Corinne [MitwirkendeR]; Marques Alves, Paula [MitwirkendeR]; Merten, Otto-Wilhelm [MitwirkendeR]; Meyring, Wilhelm [MitwirkendeR]; Mignet, Nathalie [MitwirkendeR]; Mingozzi, Federico [MitwirkendeR]; Mitrophanous, Kyriacos [MitwirkendeR]; Modlich, Ute [MitwirkendeR]; Monfared, Parisa [MitwirkendeR]; Montini, Eugenio [MitwirkendeR]; Moullier, Philippe [MitwirkendeR]; Naldini, Luigi [MitwirkendeR]; Nathwani, Amit C. [MitwirkendeR]; Nienhuis, Arthur W. [MitwirkendeR]; Palfi, Stéphane [MitwirkendeR]; Panet, Amos [MitwirkendeR]; Peixoto, Cristina [MitwirkendeR]; Pohl, Susanne [MitwirkendeR]; Popplewell, Linda [MitwirkendeR]; Pâques, Frédéric [MitwirkendeR]; Raj, Deepak [MitwirkendeR]; Ralph, R. Scott [MitwirkendeR]; Reiss, Ulrike [MitwirkendeR]; Richard, Cyrille [MitwirkendeR]; Rischmüller, Anja [MitwirkendeR]; Rodrigues, Ana F. [MitwirkendeR]; Rosenzwajg, Michelle [MitwirkendeR]; Santos, Jorge M. [MitwirkendeR]; Schambach, Axel [MitwirkendeR]; Schaser, Thomas [MitwirkendeR]; Scherman, Daniel [MitwirkendeR]; Schleef, Martin [MitwirkendeR]; Schmeer, Marco [MitwirkendeR]; Schmidt, Manfred [MitwirkendeR]; Schweizer, Matthias [MitwirkendeR]; Schäfers, Sonja [MitwirkendeR]; Seiger, Åke [MitwirkendeR]; Simão, Daniel [MitwirkendeR]; Six, Adrien [MitwirkendeR]; Smith, Alexander J [MitwirkendeR]; Smith, Alexander J. [MitwirkendeR]; Smith, Julianne [MitwirkendeR]; Snyder, Richard O. [MitwirkendeR]; Sofia Coroadinha, Ana [MitwirkendeR]; Sousa, Marcos F.Q. [MitwirkendeR]; Suerth, Julia D. [MitwirkendeR]; Sundström, Erik [MitwirkendeR]; Tayeb, Shay [MitwirkendeR]; Thomas, Michael [MitwirkendeR]; Thomas-Vaslin, Véronique [MitwirkendeR]; Tuddenham, Edward G.D. [MitwirkendeR]; VandenDriessche, Thierry [MitwirkendeR]; Verhoeyen, Els [MitwirkendeR]; Verma, Inder M. [MitwirkendeR]; Viel, Thomas [MitwirkendeR]; Waerzeggers, Yannic [MitwirkendeR]; Wahlberg, Lars [MitwirkendeR]; Winkeler, Alexandra [MitwirkendeR]; Wirth, Dagmar [MitwirkendeR]; Ylä-Herttuala, Seppo [MitwirkendeR]; Zacchigna, Serena [MitwirkendeR]; Zamir, Gidi [MitwirkendeR]
- Erschienen: Les Ulis: EDP Sciences, [2012]
- Umfang: 1 Online-Ressource (589 p.)
- Sprache: Englisch
- DOI: 10.1051/978-2-84254-237-5
- ISBN: 9782842542375
- Identifikator:
- Schlagwörter: Genetic transformation ; MEDICAL / Laboratory Medicine
- Entstehung:
-
Anmerkungen:
In English
- Beschreibung: 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
- Zugangsstatus: Eingeschränkter Zugang