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Gene Therapy for HIV - From Inception to a Possible Cure (Paperback, 2014 ed.)
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Gene Therapy for HIV - From Inception to a Possible Cure (Paperback, 2014 ed.)
Series: SpringerBriefs in Biochemistry and Molecular Biology
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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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