UID:
edocfu_9960821183902883
Format:
1 online resource (589 p.)
ISBN:
9782842542375
Content:
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.
Note:
Frontmatter --
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CliniGene Partners and Boards --
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ACKNOWLEDGMENTS --
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List of authors --
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Contents --
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INTRODUCTION --
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In-1 Foreword --
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In-2 Main achievements and prospects downstream of the CliniGene-NoE --
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PART I: TECHNOLOGIES AND PRE-CLINICAL STUDIES --
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TECHNOLOGIES - Highlights on AAV mediated gene transfer --
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A1-1 Highlights on AAV mediated gene transfer: introduction --
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A1-2 Preclinical studies of AAV gene therapy for inherited retinal dystrophies --
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A1-3 AAV-mediated gene therapy for MPS VI --
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A1-4 Microdystrophin and myostatin gene therapy for Duchenne muscular dystrophy using adeno-associated virus vectors --
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A1-5 AAV gene therapy for cardiovascular disorders --
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A1-6 AAV gene therapy for diabetes mellitus --
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A1-7 Approaches to large scale production of AAV-vectors --
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A1-8 Reference materials for the characterization of adeno-associated viral vectors --
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TECHNOLOGIES - Retrovirus mediated gene transfer state-of-the-art --
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A2-1 Highlights on retrovirus mediated gene transfer --
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A2-2 Retroviral vector development: reducing genotoxicity of integrated DNA and creating virus-like particles for transient cell modification --
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A2-3 Replication-competent y-retroviral vectors for tumor therapy --
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A2-4 Modular retroviral producer cell lines --
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TECHNOLOGIES - Highlights on lentivirus mediated gene transfer --
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A3-1 Introduction --
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A3-2 MicroRNAs detargeting technology in the context of CNS applications --
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A3-3 Development of SIVsmmPBj vectors for gene transfer into myeloid cells --
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A3-4 Insulated retrovirus vectors using novel synthetic genetic insulator elements to circumvent enhancer-mediated genotoxicity --
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A3-5 Facing the challenges of downstream processing of lentiviral vectors --
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A3-6 Restrictions and requirements for stable lentiviral vector production in HEK293 cells --
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A3-7 Novel lentiviral vector pseudotypes for stable gene transfer into resting hematopoietic cells --
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TECHNOLOGIES - Highlights on gene-modified cell therapy --
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A4-1 Cell therapy Introduction --
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A4-2 Ex-vivo expansion of human mesenchymal stem cells --
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A4-3 Closed bag cultivation systems for the production of gene modified dendritic cells and MSC for clinical use --
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A4-4 Genetically modified NK cells for cancer treatment: facts and visions --
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A4-5 Regulatory T lymphocyte depletion for cancer immunotherapies --
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A4-6 Gene therapy of Fanconi’s anaemia aplastic syndrome --
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TECHNOLOGIES - Adenovirus mediated gene transfer: current developments --
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A5-1 Overview on adenovirus vectors --
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A5-2 Tumour barriers influencing adenovirus vector delivery and therapeutic efficacy --
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A5-3 Tumor imaging with adenoviral vectors --
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A5-4 Treatment of brain tumors with adenoviruses: preclinical development --
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A5-5 Production and purification of Ad vectors: current status and future needs for adenovirus vector production --
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TECHNOLOGIES - Non-viral based gene transfer: a new era --
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A6-1 Non viral plasmid delivery and imaging of transgene expression A6-1 Non viral plasmid delivery and imaging of transgene expression --
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A6-2 Overview of novel plasmid vectors and preclinical applications --
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A6-3 Filling a gap: S/MAR-based replicating minicircles --
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A6-4 Manufacturing and QC of plasmid based vectors --
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A6-5 Sleeping Beauty transposon based gene therapy --
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A6-6 Development of minicircle vectors --
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A6-7 Exon skipping therapy for DMD using antisense oligomer technology --
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TECHNOLOGIES - Highlights on emerging technologies, iPS induction and genetic stability --
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A7-1 Induction of pluripotency from adult somatic cells: a review --
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A7-2 Genetic modification of adult stem cells and induced pluripotent stem cells with emerging transposon technologies --
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A7-3 Targeted genome engineering approaches based on rare-cutting endonucleases: a tentative summary --
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A7-4 Targeted genome modifications with designer nucleases --
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PRE-CLINICAL STUDIES, BIOSAFETY AND ANIMAL MODELS - Preclinical assessment tools --
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B1-1 Preclinical assessment tools: imaging gene transfer to the brain --
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B1-2 Persistent luminescence nanoparticles for in vivo imaging: characteristics and targeting --
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B1-3 Ex-vivo evaluation of gene-transfer vectors: efficacy, tropism and safety --
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PRE-CLINICAL STUDIES, BIOSAFETY AND ANIMAL MODELS - General biosafety: immune responses, immunotoxicity and genotoxicity --
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B2-1 Assessing and taming unwanted immune responses induced by AAV gene transfer: current status, ongoing questions and future prospects --
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B2-2 Predicting immune responses to viral vectors and transgenes in gene therapy and vaccination: the coming of systems biology --
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B2-3 Biosafety analysis in preclinical and clinical studies --
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PART II Clinical trials and regulatory issues --
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CLINICAL TRIALS --
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C1-1 A clinical trial of AAV-mediated gene therapy for Leber congenital amaurosis 2 --
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C1-2 Gene therapy for X-linked adrenoleukodystrophy based on lentiviral correction of hematopoietic stem cells --
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C1-3 Immune reconstitution after gene therapy for adenosine deaminase severe combined immunodeficiency (ADA-SCID) --
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C1-4 Gene therapy in Alzheimer disease patients --
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C1-5 Cardiovascular gene therapy trials --
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C1-6 AAV-mediated gene therapy for haemophilia B --
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C1-7 ProSavin®: a lentiviral vector approach for the treatment of Parkinson’s disease --
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ETHICAL AND REGULATORY ISSUES --
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C2-1 Ethics in translation from research to therapy --
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C2-2 Centralised regulation of gene therapy in Europe --
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C2-3 The necessity for data sharing towards advancement of clinical translation Building up sample IMPD* and substantiating master files --
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INTEGRATION AND DISSEMINATION --
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C3-1 European Union support to gene transfer and gene therapy --
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C3-2 Database of clinical trials --
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C3-3 CliniGene and ESGCT shared vision for gene therapy in Europe: past, present and future prospects --
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AUTHOR INDEX
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In English.
Language:
English
DOI:
10.1051/978-2-84254-237-5
URL:
https://doi.org/10.1051/978-2-84254-237-5
URL:
https://www.degruyter.com/isbn/9782842542375
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