Concise overview of the development of lentivectors and gene therapy, integrating historical facts, the development and experimental application of lentivectors

Ideal introduction and book of reference for researchers, academics and students interested in this field

Each chapter can be read independently

Includes the very latest human clinical trials

Thorough treatise on an emerging subject?

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 ¿ -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.