EAN: 9781493904334

SPRINGERBRIEFS IN BIOCHEMISTRY AND MOLECULAR BI...

This Brief describes the concept and realization of gene therapy for HIV from the unique
historic perspective and insight of two pioneers of the clinical applications of stem cell gene
therapy for HIV. Gerhard Bauer applied ribozyme-anti-HIV and other vectors to manufacture
clinical grade  HIV-resistant hematopoietic stem cells for the first patients that received
stem cell gene therapy for HIV  including the first child in the world and the first fully
marrow-ablated HIV infected patient. Joseph Anderson developed the most recent and most potent
combination anti-HIV lentiviral vectors and pluripotent stem cell applications for HIV gene
therapy and tested these in the appropriate in vitro and vivo models  paving the way for novel
HIV gene therapy approaches to possibly cure patients. In Gene Therapy for HIV  Bauer and
Anderson discuss the unique aspects of this therapy  including its limitations and proper
safety precautions and outline a path for a possible functional cure for HIV using stem cell
gene therapy based on a cure already achieved with a bone marrow stem cell transplantation
performed in Germany using donor stem cells with a naturally arising CCR5 mutation. In addition
the Brief provides a thorough and methodical explanation of the basics of gene therapy  gene
therapy vector development  in vitro and in vivo models for HIV gene therapy and clinical
applications of HIV gene therapy  including Good Manufacturing Practices.

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