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Books > Medicine > Clinical & internal medicine > Haematology
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Blood Cell Biochemistry - Hematopoiesis and Gene Therapy (Paperback, Softcover reprint of the original 1st ed. 1999)
Loot Price: R4,288
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Blood Cell Biochemistry - Hematopoiesis and Gene Therapy (Paperback, Softcover reprint of the original 1st ed. 1999)
Series: Blood Cell Biochemistry, 8
Expected to ship within 10 - 15 working days
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Since the first concepts of gene therapy were formulated, the
hemopoietic system has been considered the most natural first
target tissue for genetic manipulation. The reasons for this
include the fact that a very large number of inherited disorders
(including some of the most common disorders, such as the
hemoglobinopathies) are disorders of the hemopoietic system, and
the large amount of experience in hematopoietic transplantation
biology. The consequence of this resulted in the first clinical
trial of gene therapy in 1989, where two children suffering from
severe combined immune deficiency (ADA-SCID) were transplanted with
T-cells express ing adenosine deaminase (the defective enzyme in
patients with this disorder). The partial success of this treatment
was perhaps responsible for undue optimism among those proposing
other gene therapy treatments within the hematopoietic system, and
it has since become clear that there are a number of technical and
biological difficulties to overcome before hematopoietic gene
therapy becomes a mainstream therapeutic strategy. The chapters in
this book evaluate the need for gene therapy in the hematopoietic
system, discuss how efficient gene transfer and expression can be
achieved in the target cells, highlight areas of difficulty to be
addressed, and examine a number of potential applications of the
gene therapy approach. The book begins with a chapter by Testa and
colleagues, discussing the various sources of hematopoietic cells
for both transplantation and gene therapy.
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