Kooperativer Bibliotheksverbund

UID:

Format: XXI, 303 S. : , Ill., graph. Darst.

ISBN: 978-88-470-1642-2

Language: English

Subjects: Biology

URL: http://deposit.d-nb.de/cgi-bin/dokserv?id=3426813&prov=M&dok_var=1&dok_ext=htm

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Format: XXI, 238 Seiten , Ill., graph. Darst.

ISBN: 9788847019881

Note: Text ital.

Additional Edition: Erscheint auch als Druck-Ausgabe Mauro Giacca Gene therapy

Language: Italian

Keywords: Gentherapie

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Format: XXI, 303 p. , online resource.

Edition: 1st ed. 2010.

ISBN: 9788847016439

Content: This book presents to the reader a comprehensive and detailed, yet easily accessible, description of the whole field of gene therapy. It covers topics ranging from vector development to the results of the most recent clinical trials. It includes descriptions of lentiviral and AAV vector development, of therapeutic gene selection (including siRNAs, shRNAs, and microRNAs), and of the most recent clinical applications of gene therapy for diseases of the eye, the cardiovascular system, and the central nervous system. Of note, the book only considers conditions for which transfer of genetic material has already progressed to clinical applications or is close to doing so. The language is plain and, whenever possible, non-technical. Since the book is intended to be a textbook in the field of gene therapy in both the basic science and clinical fields, whenever technical descriptions are required these are provided. Readers will include graduate students in the life sciences area in their final years of study, PhD students, and medical doctors specializing in different fields of internal and specialist medicine, in addition to research scientists working in various areas of current biomedical research.

Note: to Gene Therapy -- Therapeutic Nucleic Acids -- Methods for Gene Delivery -- Clinical Applications of Gene Therapy -- Ethical and Social Problems of Gene Therapy.

In: Springer Nature eBook

Additional Edition: Printed edition: ISBN 9788847055551

Additional Edition: Printed edition: ISBN 9788847016422

Additional Edition: Printed edition: ISBN 9788847016446

Language: English

DOI: 10.1007/978-88-470-1643-9

URL: https://doi.org/10.1007/978-88-470-1643-9

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Format: XXI, 240 pagg. , online resource.

Edition: 1st ed. 2011.

ISBN: 9788847019898

Content: Questo volume presenta, in maniera comprensiva e dettagliata e allo stesso tempo accessibile, l'intero settore della terapia genica, partendo dalle proprietà dei diversi geni terapeutici per finire con i risultati delle sperimentazioni cliniche più avanzate,e includendo la descrizione delle principali tecniche per il trasferimento genico in vivo ed ex vivo. Il testo comprende argomenti relativi allo sviluppo dei vettori lentivirali e AAV, alle proprietà dei siRNA e dei microRNA, insieme alla descrizione dei risultati delle più recenti sperimentazioni nel campo delle cellule staminali ematopoietiche e delle malattie della retina, del cuore e del sistema nervoso centrale. Fornendo sia informazioni cliniche sia dettagli molecolari relativi alle malattie finora affrontate dalla terapia genica, il volume sarà un prezioso strumento di studio e approfondimento non solo per il ricercatore dell'area biomedica e lo studente di medicina negli ultimi anni di studio, ma anche per il clinico di medicina interna e delle discipline specialistiche.   Mauro Giacca è Direttore della Componente Italiana dell'International Centre for Genetic Engineering and Biotechnology (ICGEB), un'organizzazione internazionale di ricerca con sede a Trieste, e Professore Ordinario di biologia molecolare presso la Facoltà di Medicina e Chirurgia dell'Università di Trieste. Negli ultimi 20 anni,ha partecipato in prima linea allo sviluppo della terapia genica, pubblicando più di 200 articoli sulle riviste scientifiche internazionali. È considerato uno dei massimi esperti nello sviluppo dei vettori AAV e nella loro applicazione per la terapia genica delle malattie cardiovascolari.

In: Springer Nature eBook

Additional Edition: Printed edition: ISBN 9788847019881

Additional Edition: Printed edition: ISBN 9788847019904

Language: Italian

DOI: 10.1007/978-88-470-1989-8

URL: https://doi.org/10.1007/978-88-470-1989-8

UID:

Format: Online-Ressource (250pagg. 80 figg. a colori, digital)

ISBN: 9788847019898

Series Statement: SpringerLink

Content: Mauro Giacca

Content: Questo volume presenta, in maniera comprensiva e dettagliata e allo stesso tempo accessibile, lintero settore della terapia genica, partendo dalle proprietà dei diversi geni terapeutici per finire con i risultati delle sperimentazioni cliniche più avanzate,e includendo la descrizione delle principali tecniche per il trasferimento genico in vivo ed ex vivo. Il testo comprende argomenti relativi allo sviluppo dei vettori lentivirali e AAV, alle proprietà dei siRNA e dei microRNA, insieme alla descrizione dei risultati delle più recenti sperimentazioni nel campo delle cellule staminali ematopoietiche e delle malattie della retina, del cuore e del sistema nervoso centrale. Fornendo sia informazioni cliniche sia dettagli molecolari relativi alle malattie finora affrontate dalla terapia genica, il volume sarà un prezioso strumento di studio e approfondimento non solo per il ricercatore dellarea biomedica e lo studente di medicina negli ultimi anni di studio, ma anche per il clinico di medicina interna e delle discipline specialistiche. Mauro Giacca è Direttore della Componente Italiana dellInternational Centre for Genetic Engineering and Biotechnology (ICGEB), unorganizzazione internazionale di ricerca con sede a Trieste, e Professore Ordinario di biologia molecolare presso la Facoltà di Medicina e Chirurgia dellUniversità di Trieste. Negli ultimi 20 anni,ha partecipato in prima linea allo sviluppo della terapia genica, pubblicando più di 200 articoli sulle riviste scientifiche internazionali. È considerato uno dei massimi esperti nello sviluppo dei vettori AAV e nella loro applicazione per la terapia genica dellemalattie cardiovascolari.

Note: Includes bibliographical references and index , Title Page; Copyright Page; Prefazione; Ringraziamenti; Table of Contents; Abbreviazioni; CAPITOLO 1 Introduzione alla terapia genica; Geni come farmaci; Terapia genica: una visione d'insieme; Scelta del gene terapeutico; Modalità di somministrazione dei geni terapeutici; Sistemi di trasferimento genico; Targeting cellulare; Persistenza del gene terapeutico; Espressione del gene terapeutico; Risposta immunitaria alla terapia genica; Letture consigliate; CAPITOLO 2 Acidi nucleici con funzione terapeutica; Geni codificanti proteine; Proteine sostitutive di proteine cellulari assenti o mutate , Proteine che modulano funzioni cellulariFattori di crescita secreti e citochine; Proteine che regolano la sopravvivenza cellulare e l'apoptosi; Antigeni per la vaccinazione; Anticorpi e anticorpi intracellulari; Subunità del T-cell receptor (TCR); Controllo dell'espressione dei geni terapeutici; Acidi nucleici non codificanti; Oligonucleotidi; Oligonucleotidi modificati; Applicazioni cliniche degli oligonucleotidi; Acidi nucleici catalitici; Piccoli RNA con funzione regolatoria (siRNA, microRNA); MicroRNA (miRNA); Utilizzo terapeutico dell'RNAi; Decoy; Aptameri , Modalità di somministrazione o sintesi intracellulare dei piccoli RNA regolatoriSomministrazione esogena; Sintesi intracellulare di RNA regolatori; Letture consigliate; CAPITOLO 3 Metodologie per il trasferimento genico; Barriere cellulari al trasferimento genico; Endocitosi; Fuoriuscita dal compartimento vescicolare; Indirizzamento al nucleo; Metodologie per il trasferimento genico per la terapia genica: una visione d'insieme; Inoculazione diretta di DNA ed RNA; Metodi fisici; Elettroporazione; Iniezione idrodinamica intravascolare; Sonoporazione , Bombardamento con microparticelle ricoperte di DNA (gene gun)Iniezione di DNA con getti ad alta pressione (jet injection); Metodi chimici; Liposomi e lipidi cationici; Polimeri cationici; Proteine; Problematiche legate all'utilizzo dei metodi chimici per il trasferimento genico; Vettori virali; Vettori basati sui gammaretrovirus; Biologia molecolare e ciclo replicativo dei retrovirus; Struttura dei vettori basati sui gammaretrovirus; Produzione dei vettori gammaretrovirali; Varianti nella costruzione dei genomi dei vettori gammaretrovirali; Proprietà dei vettori gammaretrovirali , Vettori basati sui lentivirusStruttura e produzione dei vettori lentivirali; Proprietà dei vettori lentivirali; Vettori basati sugli adenovirus; Biologia molecolare e ciclo replicativo degli adenovirus; Struttura dei vettori adenovirali; Produzione dei vettori adenovirali; Proprietà dei vettori adenovirali; Vettori basati sul virus adeno-associato (AAV); Biologia molecolare e ciclo replicativo di AAV; Struttura e produzione dei vettori AAV; Proprietà dei vettori AAV; Vettori basati sul virus dell'herpes simplex (HSV); Biologia molecolare e ciclo replicativo degli herpesvirus , Struttura e produzione dei vettori derivati da HSV-1

Additional Edition: ISBN 9788847019881

Additional Edition: Buchausg. u.d.T. ISBN 978-88-470-1988-1

Language: Italian

DOI: 10.1007/978-88-470-1989-8

URL: Volltext

URL: Volltext  (lizenzpflichtig)

URL: Inhaltsverzeichnis

URL: Cover

UID:

Format: Online-Ressource , v.: digital

Edition: Online-Ausg. Springer eBook Collection. Biomedical and Life Sciences Electronic reproduction; Available via World Wide Web

ISBN: 9788847016439

Content: The overall purpose of this book is to expose the reader/student to a comprehensive and detailed, yet still easily accessible, overview of the whole field of gene therapy, ranging from vector development and therapeutic gene selection to the results of the most recent clinical trials. The language is plain and, whenever possible, non-technical. Since the book is intended to be a textbook in the field of gene therapy in both the basic science and clinical areas, when technical descriptions are required, they are provided either in the main text or in add-on explanatory boxes. For example, clinical readers might find it difficult to understand the principles of viral vector design without knowing some molecular details of viral genome organization and virus life cycle, and basic scientists might be unaware of the clinical and therapeutic advancements for the various disorders treated by the book. In these cases, basic explanations are provided. In this respect, it is worth pointing out that the book is truly a gene 'therapy' book; it only considers conditions and experimental approaches based on gene transfer that have already proceeded to the clinic or are close to doing so. Basic research on gene transfer or nucleic acid modification is only presented if it is when close to clinical application. Following the same principle, only diseases for which clinical trials have already been performed or are planned are considered, or those for which solid and encouraging pre-clinical, animal experimentation results are available.

Note: Includes bibliographical references and index , Electronic reproduction; Available via World Wide Web

Additional Edition: ISBN 9788847016422

Language: English

DOI: 10.1007/978-88-470-1643-9

URL: Volltext  (lizenzpflichtig)

UID:

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 -- , 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 , 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