The CliniBook

Clinical gene transfer state of the art

Odile Cohen-Haguenauer

• Publisher: EDP Sciences
• Year: 2012
• Edition: 1
• eBook ISBN:
  9782842542375
• Topic: Medicine
• Subtopic: Medical Technology & Supplies

Table of contents

• CliniGene Partners and Boards
• ACKNOWLEDGMENTS
• List of authors
• Contents
• INTRODUCTION
• In-1 Foreword
• In-2 Main achievements and prospects downstream of the CliniGene-NoE
• 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