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The ABCs of Gene CloningHuman Gene Therapy

The ABCs of Gene Cloning: Human Gene Therapy [There are more than 4000 known inherited disorders. The majority of them have minimal effects, but a few causes physical and mental abnormalities that may be life threatening. Genetic diseases that are candidates for gene therapies include severe combined immunodeficiency, thalassaemia, and cystic fibrosis, etc. (Fig. 19.1). Since these genetic diseases are each caused by a single defective gene, one potential treatment is to introduce a normal functional copy of the gene into the cell tissue that is affected. In effect, the normal (therapeutic) gene augments the defective gene in the patient. Gene therapy is not restricted to only treating genetic disorders. The general technology of transferring genetic materials into a patient is also applied to diseases such as cancer, AIDS, and cardiovascular diseases. Many of the approved clinical trials on gene therapy are for the treatment of diseases other than genetic disorders (Table 19.1).] http://www.deepdyve.com/assets/images/DeepDyve-Logo-lg.png

The ABCs of Gene CloningHuman Gene Therapy

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Publisher
Springer International Publishing
Copyright
© Springer International Publishing AG, part of Springer Nature 2018
ISBN
978-3-319-77762-7
Pages
177 –186
DOI
10.1007/978-3-319-77982-9_19
Publisher site
See Chapter on Publisher Site

Abstract

[There are more than 4000 known inherited disorders. The majority of them have minimal effects, but a few causes physical and mental abnormalities that may be life threatening. Genetic diseases that are candidates for gene therapies include severe combined immunodeficiency, thalassaemia, and cystic fibrosis, etc. (Fig. 19.1). Since these genetic diseases are each caused by a single defective gene, one potential treatment is to introduce a normal functional copy of the gene into the cell tissue that is affected. In effect, the normal (therapeutic) gene augments the defective gene in the patient. Gene therapy is not restricted to only treating genetic disorders. The general technology of transferring genetic materials into a patient is also applied to diseases such as cancer, AIDS, and cardiovascular diseases. Many of the approved clinical trials on gene therapy are for the treatment of diseases other than genetic disorders (Table 19.1).]

Published: May 27, 2018

Keywords: Approved Clinical Trials; Severe Combined Immunodeficiency; Retroviral Core Proteins; Bovine Leukemia Virus; Adeno-associated Virus

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