Lentiviral Vectors and Gene Therapy

• Κύριοι συγγραφείς: Escors, David (Συγγραφέας), Breckpot, Karine (Συγγραφέας), Arce, Frederick (Συγγραφέας), Kochan, Grazyna (Συγγραφέας), Stephenson, Holly (Συγγραφέας)
• Συγγραφή απο Οργανισμό/Αρχή: SpringerLink (Online service)
• Μορφή: Ηλεκτρονική πηγή Ηλ. βιβλίο
• Γλώσσα: English
• Έκδοση: Basel : Springer Basel, 2012.
• Σειρά: SpringerBriefs in Biochemistry and Molecular Biology,
• Θέματα: Medicine.; Gene therapy.; Immunology.; Genetic engineering.; Oncology.; Cell biology.; Biomedicine.; Gene Therapy.; Genetic Engineering.; Cell Biology.

Gene therapy was conceived during the early and mid part of the 20th century. At first, it was considered a revolutionary biomedical procedure, which could potentially cure any disease for which the molecular bases were understood. Since then, gene therapy has gone through many stages and has evolve...

Πίνακας περιεχομένων:

• 1 Introduction to gene therapy.- 1.1 The concept of gene therapy
• 1.2 Origins of gene therapy
• 1.3 Gene therapy in the 1970s
• 1.4 Gene therapy in the 1980s
• 1.5 The breakthroughs in gene therapy from the 1990s and 2000s
• 1.6 Current human gene therapy and lentiviral vectors
• 1.7. Concluding remarks.- 2 Development of retroviral and lentiviral vectors.- 2.1 Retrovirus biology
• 2.2 Vectors based on γ -retroviruses
• 2.3 Vectors based on lentiviruses
• 2.4 Summary and conclusions.- 3 Cell and tissue gene targeting with lentiviral vectors
• 3.1 Introduction
• 3.2 Modification of lentivector tropism by pseudotyping (surface targeting)
• 3.3 Transcriptional targeting
• 3.4 Post-transcriptional targeting
• 3.5 Conclusions.-  4 Immunomodulation by genetic modification using lentiviral vectors
• 4.1 Introduction to genetic immunotherapy
• 4.2 Lentivector gene therapy for immunization
• 4.3 Lentivector gene therapy for the treatment of autoimmune disease
• 4.4 Conclusions
• 5 Clinical grade lentiviral vectors.- 5.1 Introduction
• 5.2 Good manufacturing practise guidelines and clinical grade vector preparations
• 5.3 Scaling-up lentivector production for clinical application
• 5.4 Purity of clinical grade lentivectors
• 5.5 Biosafety
• 5.6 Final considerations and conclusions.- 6 Human gene therapy with retrovirus and lentivirus vectors
• 6.1 Introduction
• 6.2 Correction of Severe Combined Immunodeficiency-X1
• 6.3 Correction of X-linked chronic granulomatous disease
• 6.4 Correction of X-linked adrenoleukodystrophy
• 6.5 Correction of b-thalassaemia
• 6.6 Correction of Wiscott-Aldrich syndrome
• 6.7 Conclusions and final considerations.