Lentiviral Vectors and Gene Therapy

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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Bibliographic Details
Main Authors: Escors, David (Author), Breckpot, Karine (Author), Arce, Frederick (Author), Kochan, Grazyna (Author), Stephenson, Holly, 1942- (Author)
Format: Electronic eBook
Language:English
Published: Basel : Springer Basel, 2012.
Series:SpringerBriefs in biochemistry and molecular biology.
Subjects:
Medicine.
Gene therapy.
Immunology.
Genetic engineering.
Oncology.
Cytology.
Biomedicine.
Gene Therapy.
Genetic Engineering.
Cell Biology.
Online Access:Connect to this title online
Table of Contents:
  • 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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