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Licensed Unlicensed Requires Authentication Published by EDP Sciences 2012

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

From the book The CliniBook

  • Dror Kolodkin-Gal , Shay Tayeb , Abed Khalaileh , Gidi Zamir , Nikolai Kunicher and Amos Panet
https://doi.org/10.1051/978-2-84254-237-5.c049
© 2012 EDP Sciences, Les Ulis
From the book
The CliniBook
The CliniBook

Chapters in this book (68)

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
PRE-CLINICAL STUDIES, BIOSAFETY AND ANIMAL MODELS - Preclinical assessment tools
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
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
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
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