Lentiviral Vectors and Gene Therapy

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...

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Detalles Bibliográficos
Clasificación:Libro Electrónico
Autores principales: Escors, David (Autor), Breckpot, Karine (Autor), Arce, Frederick (Autor), Kochan, Grazyna (Autor), Stephenson, Holly (Autor)
Autor Corporativo: SpringerLink (Online service)
Formato: Electrónico eBook
Idioma:Inglés
Publicado: Basel : Springer Basel : Imprint: Springer, 2012.
Edición:1st ed. 2012.
Colección:SpringerBriefs in Biochemistry and Molecular Biology,
Temas:
Medical genetics.
Immunology.
Biotechnology.
Cytology.
Oncology.
Clinical Genetics.
Chemical Bioengineering.
Cell Biology.
Acceso en línea:Texto Completo
Tabla de Contenidos:
  • 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.

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