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Books > Medicine > Clinical & internal medicine > Diseases & disorders > Immunology
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The Skin and Gene Therapy (Paperback, Softcover reprint of the original 1st ed. 2001)
Loot Price: R1,416
Discovery Miles 14 160
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The Skin and Gene Therapy (Paperback, Softcover reprint of the original 1st ed. 2001)
Expected to ship within 10 - 15 working days
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Gene therapy is a novel concept of treating diseases at the molec-
ular level by rescuing genetic defects through the introduction of
corrective genes. The knowledge of genetic defects underlying var-
ious diseases is being rapidly accumulated, and new genotypic
findings are being reported with striking frequency. The elucida-
tion of the genetic background of many diseases, including hered-
itary blistering and hyperkeratotic skin disorders, has provided
the necessary knowledge to correct inborn genetic aberrations in
ani- mal models and patient-derived tissue. However,skin is not
only a target organ for gene therapy ofcutaneous disorders. Dueto
its bio- logical properties, easy accessibility, and convenient,
established culture conditions and grafting techniques, skin has
rapidly be- come an important target for correcting other diseases
as well. Both skin fibroblasts and epidermal keratinocytes have
been successfully transfected ex vivo and in vivo using viral and
nonvir- al gene transfer methods. Notably,in the first corrective
gene ther- apy approach in 1987, fibroblasts from
adenosine-deaminase (ADA)-deficienthumans with severe combined
immunodeficiency (SCID) syndrome were transduced using retroviral
vectors. More- over, skin cells are capable of synthesizing various
proteins that are therapeutic, either locally or systemically.
Human epidermal grafts expressing human growth hormone after
retroviral gene transfer and grafting onto athymic mice released
human growth hormone into the bloodstream of the transplanted mice,
for a lim- ited time.
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