| Applications
 Hemoglobinopathies
Coagulopathies
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The
earliest applications of gene therapy were based on the principle that
a disease is caused by a faulty gene (or combination of genes), and if
such genes can be replaced with ‘correct’ versions, the disease
might be controlled, prevented or cured. Gene therapy is being applied
to many different genetic diseases, both congenital (since birth) and
acquired. However, most diseases involve multiple genetic factors
(they are polygenic).
Originally known as ‘genetic
replacement therapy’ during the early 1980s, ‘gene therapy’ has
now outgrown its original definition and is applied to all manner of
protocols that involve an element of gene transfer, either in vivo or
ex vivo, and not necessarily a gene that is known to cause a disease.
In vivo gene transfer is the introduction of genes to cells at the
site they are found in the body, for example to skin cells on an arm,
or to lung epithelial cells following inhalation of the gene transfer
vector. Ex vivo gene transfer is the transfer of genes into viable
cells that have been temporarily removed from the patient and are then
returned following treatment (e.g. bone marrow cells).
(From Gene
therapy: potential applications in clinical transplantation,
Jeremy W. Fry and Kathryn J. Wood)
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