The CliniBook : : Clinical gene transfer state of the art / / Odile Cohen-Haguenauer.
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 is...
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Table of Contents:
- 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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