%0 Generic

%T The CliniBook Clinical gene transfer state of the art

%A Cohen-Haguenauer, Odile

%A Aiuti, Alessandro

%A Ali, Robin R.

%A Alici, Evren

%A Almqvist, Per

%A Annunziata, Patrizia

%A Anton, Martina

%A Artus, Alexandre

%A Athanasopoulos, Takis

%A Aubourg, Patrick

%A Auregan, Gwennaelle

%A Auricchio, Alberto

%A Bandeira, Vanessa

%A Bartholomae, Cynthia C.

%A Baum, Christopher

%A Bayart, Émilie

%A Bednarski, Christien

%A Bellier, Bertrand

%A Biasco, Luca

%A Bigey, Pascal

%A Blouin, Véronique

%A Bode, Juergen

%A Bonvento, Gilles

%A Bosch, Fatima

%A Bougnères, Pierre

%A Brigida, Immacolata

%A Broll, Sandra

%A Buchholz, Christian J.

%A Bureau, Michel-Francis

%A Bárcia, Rita N.

%A Callejas, David

%A Carina Silva, Ana

%A Carrondo, Manuel J.T

%A Cartier, Nathalie

%A Cathomen, Toni

%A Cavazzana-Calvo, Marina

%A Chuah, Marinee K.L

%A Cichutek, Klaus

%A Cohen-Haguenauer, Odile

%A Colin, Angélique

%A Cordova, Gonzalo

%A Coroadinha, Ana Sofia

%A Cosset, François-Loïc

%A Cotugno, Gabriella

%A Cruz, Helder J.S

%A Cruz, Pedro E.

%A Darmon, Christophe

%A Davidoff, Andrew M.

%A Dickson, George

%A Dittmar, Kurt E.J

%A Draghia-Akli, Ruxandra

%A Dufour, Noëlle

%A Duros, Caroline

%A Déglon, Nicole

%A Dürner, Lydia

%A Eriksdotter-Jönhagen, Maria

%A Escartin, Carole

%A Escriou, Virginie

%A Eyjolfsdottir, Helga

%A Faideau, Mathilde

%A Fischer, Alain

%A Foster, Helen

%A Foster, Keith

%A Gahrton, Gösta

%A Galla, Melanie

%A Galy, Anne

%A Gama-Norton, Leonor

%A Garritsen, Henk S.P

%A Georgiadis, Anastasios

%A Giacca, Mauro

%A Gilljam, Mari

%A Gänsbacher, Bernd

%A Hacein-Bey-Abina, Salima

%A Hantraye, Philippe

%A Haskins, Mark

%A Hassig, Raymonde

%A Hauser, Hansjörg

%A Heinz, Niels

%A Händel, Eva-Maria

%A Ivics, Zoltán

%A Izsvák, Zsuzsanna

%A Jacobs, Andreas H.

%A Jarmin, Susan

%A Jimenez, Veronica

%A Kalle, Christof von

%A Kang, Jagjeet

%A Kent, Alastair

%A Khalaileh, Abed

%A Kichler, Antoine

%A King, Nancy M.P

%A Klatzmann, David

%A Kochanek, Stefan

%A Kolodkin-Gal, Dror

%A Koo, Taeyoung

%A Kunicher, Nikolai

%A Lachmann, Kristina

%A Laufs, Stephanie

%A Lemaire, Stéphanie

%A Lemoine, François

%A Lind, Göran

%A Lindenmaier, Werner

%A Linderoth, Bengt

%A Lock, Martin

%A Lucas, Tanja

%A Luzzatto, Lucio

%A Macke, Lars

%A Maetzig, Tobias

%A Maldiney, Thomas

%A Malerba, Alberto

%A Mann, Christopher

%A Marie, Corinne

%A Marques Alves, Paula

%A Merten, Otto-Wilhelm

%A Meyring, Wilhelm

%A Mignet, Nathalie

%A Mingozzi, Federico

%A Mitrophanous, Kyriacos

%A Modlich, Ute

%A Monfared, Parisa

%A Montini, Eugenio

%A Moullier, Philippe

%A Naldini, Luigi

%A Nathwani, Amit C.

%A Nienhuis, Arthur W.

%A Palfi, Stéphane

%A Panet, Amos

%A Peixoto, Cristina

%A Pohl, Susanne

%A Popplewell, Linda

%A Pâques, Frédéric

%A Raj, Deepak

%A Ralph, R. Scott

%A Reiss, Ulrike

%A Richard, Cyrille

%A Rischmüller, Anja

%A Rodrigues, Ana F.

%A Rosenzwajg, Michelle

%A Santos, Jorge M.

%A Schambach, Axel

%A Schaser, Thomas

%A Scherman, Daniel

%A Schleef, Martin

%A Schmeer, Marco

%A Schmidt, Manfred

%A Schweizer, Matthias

%A Schäfers, Sonja

%A Seiger, Åke

%A Simão, Daniel

%A Six, Adrien

%A Smith, Alexander J

%A Smith, Alexander J.

%A Smith, Julianne

%A Snyder, Richard O.

%A Sofia Coroadinha, Ana

%A Sousa, Marcos F.Q

%A Suerth, Julia D.

%A Sundström, Erik

%A Tayeb, Shay

%A Thomas, Michael

%A Thomas-Vaslin, Véronique

%A Tuddenham, Edward G.D

%A VandenDriessche, Thierry

%A Verhoeyen, Els

%A Verma, Inder M.

%A Viel, Thomas

%A Waerzeggers, Yannic

%A Wahlberg, Lars

%A Winkeler, Alexandra

%A Wirth, Dagmar

%A Ylä-Herttuala, Seppo

%A Zacchigna, Serena

%A Zamir, Gidi

%I EDP Sciences

%@ 9782842542375

%K Genetic transformation

%K MEDICAL / Laboratory Medicine

%D [2012]

%X Frontmatter

%X CliniGene Partners and Boards

%X ACKNOWLEDGMENTS

%X List of authors

%X Contents

%X INTRODUCTION

%X In-1 Foreword

%X In-2 Main achievements and prospects downstream of the CliniGene-NoE

%X PART I: TECHNOLOGIES AND PRE-CLINICAL STUDIES

%X TECHNOLOGIES - Highlights on AAV mediated gene transfer

%X A1-1 Highlights on AAV mediated gene transfer: introduction

%X A1-2 Preclinical studies of AAV gene therapy for inherited retinal dystrophies

%X A1-3 AAV-mediated gene therapy for MPS VI

%X A1-4 Microdystrophin and myostatin gene therapy for Duchenne muscular dystrophy using adeno-associated virus vectors

%X A1-5 AAV gene therapy for cardiovascular disorders

%X A1-6 AAV gene therapy for diabetes mellitus

%X A1-7 Approaches to large scale production of AAV-vectors

%X A1-8 Reference materials for the characterization of adeno-associated viral vectors

%X TECHNOLOGIES - Retrovirus mediated gene transfer state-of-the-art

%X A2-1 Highlights on retrovirus mediated gene transfer

%X A2-2 Retroviral vector development: reducing genotoxicity of integrated DNA and creating virus-like particles for transient cell modification

%X A2-3 Replication-competent y-retroviral vectors for tumor therapy

%X A2-4 Modular retroviral producer cell lines

%X TECHNOLOGIES - Highlights on lentivirus mediated gene transfer

%X A3-1 Introduction

%X A3-2 MicroRNAs detargeting technology in the context of CNS applications

%X A3-3 Development of SIVsmmPBj vectors for gene transfer into myeloid cells

%X A3-4 Insulated retrovirus vectors using novel synthetic genetic insulator elements to circumvent enhancer-mediated genotoxicity

%X A3-5 Facing the challenges of downstream processing of lentiviral vectors

%X A3-6 Restrictions and requirements for stable lentiviral vector production in HEK293 cells

%X A3-7 Novel lentiviral vector pseudotypes for stable gene transfer into resting hematopoietic cells

%X TECHNOLOGIES - Highlights on gene-modified cell therapy

%X A4-1 Cell therapy Introduction

%X A4-2 Ex-vivo expansion of human mesenchymal stem cells

%X A4-3 Closed bag cultivation systems for the production of gene modified dendritic cells and MSC for clinical use

%X A4-4 Genetically modified NK cells for cancer treatment: facts and visions

%X A4-5 Regulatory T lymphocyte depletion for cancer immunotherapies

%X A4-6 Gene therapy of Fanconi’s anaemia aplastic syndrome

%X TECHNOLOGIES - Adenovirus mediated gene transfer: current developments

%X A5-1 Overview on adenovirus vectors

%X A5-2 Tumour barriers influencing adenovirus vector delivery and therapeutic efficacy

%X A5-3 Tumor imaging with adenoviral vectors

%X A5-4 Treatment of brain tumors with adenoviruses: preclinical development

%X A5-5 Production and purification of Ad vectors: current status and future needs for adenovirus vector production

%X TECHNOLOGIES - Non-viral based gene transfer: a new era

%X A6-1 Non viral plasmid delivery and imaging of transgene expression A6-1 Non viral plasmid delivery and imaging of transgene expression

%X A6-2 Overview of novel plasmid vectors and preclinical applications

%X A6-3 Filling a gap: S/MAR-based replicating minicircles

%X A6-4 Manufacturing and QC of plasmid based vectors

%X A6-5 Sleeping Beauty transposon based gene therapy

%X A6-6 Development of minicircle vectors

%X A6-7 Exon skipping therapy for DMD using antisense oligomer technology

%X TECHNOLOGIES - Highlights on emerging technologies, iPS induction and genetic stability

%X A7-1 Induction of pluripotency from adult somatic cells: a review

%X A7-2 Genetic modification of adult stem cells and induced pluripotent stem cells with emerging transposon technologies

%X A7-3 Targeted genome engineering approaches based on rare-cutting endonucleases: a tentative summary

%X A7-4 Targeted genome modifications with designer nucleases

%X B1-1 Preclinical assessment tools: imaging gene transfer to the brain

%X B1-2 Persistent luminescence nanoparticles for in vivo imaging: characteristics and targeting

%X B1-3 Ex-vivo evaluation of gene-transfer vectors: efficacy, tropism and safety

%X B2-1 Assessing and taming unwanted immune responses induced by AAV gene transfer: current status, ongoing questions and future prospects

%X B2-2 Predicting immune responses to viral vectors and transgenes in gene therapy and vaccination: the coming of systems biology

%X B2-3 Biosafety analysis in preclinical and clinical studies

%X PART II Clinical trials and regulatory issues

%X CLINICAL TRIALS

%X C1-1 A clinical trial of AAV-mediated gene therapy for Leber congenital amaurosis 2

%X C1-2 Gene therapy for X-linked adrenoleukodystrophy based on lentiviral correction of hematopoietic stem cells

%X C1-3 Immune reconstitution after gene therapy for adenosine deaminase severe combined immunodeficiency (ADA-SCID)

%X C1-4 Gene therapy in Alzheimer disease patients

%X C1-5 Cardiovascular gene therapy trials

%X C1-6 AAV-mediated gene therapy for haemophilia B

%X C1-7 ProSavin®: a lentiviral vector approach for the treatment of Parkinson’s disease

%X ETHICAL AND REGULATORY ISSUES

%X C2-1 Ethics in translation from research to therapy

%X C2-2 Centralised regulation of gene therapy in Europe

%X C2-3 The necessity for data sharing towards advancement of clinical translation Building up sample IMPD* and substantiating master files

%X INTEGRATION AND DISSEMINATION

%X C3-1 European Union support to gene transfer and gene therapy

%X C3-2 Database of clinical trials

%X C3-3 CliniGene and ESGCT shared vision for gene therapy in Europe: past, present and future prospects

%X AUTHOR INDEX

%C EDP Sciences

%C Les Ulis

%U http://slubdd.de/katalog?TN_libero_mab2