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Chapter 40 : Bioethics Case Study: Gene Therapy

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Bioethics Case Study: Gene Therapy, Page 1 of 2

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Abstract:

Leroy Walters, of Georgetown University’s Kennedy Institute of Bioethics, divided gene therapy into four possible categories. (1) Somatic-cell gene therapy for the cure or prevention of disease. Example: insertion of a DNA sequence into a person’s cells to allow production of an enzyme like adenosine deaminase. (2) Germ line gene therapy for cure or prevention of disease. Example: insertion of an adenosine deaminase sequence into early embryo or reproductive cells, which would affect not only the individual but all of his or her offspring. (3) Somatic-cell enhancement. Example: insertion of a DNA sequence to improve memory, increase height, or increase intelligence, which would affect only that individual. (4) Germ line enhancement. Example: insertion of a DNA sequence for enhancement into a blastocyst, sperm, or egg, which would affect future generations. Germ line therapy (altering disease genes so that the individual not only will be healthy but will pass on the healthy genes to his or her offspring) is considered desirable by some, but the techniques used to alter animal embryos have far too high a failure rate to consider their application to humans at this time. In the future, however, physicians will be able to detect many more defective genes than those that cause illnesses that are incurable and untreatable. Some genetic defects that are life threatening will be controllable with therapeutics (e.g., hemophilia). Other genetic defects will simply indicate a propensity to develop a disease.

Citation: Kreuzer H, Massey A. 2008. Bioethics Case Study: Gene Therapy, p 585-595. In Molecular Biology and Biotechnology: A Guide for Teachers, Third Edition. ASM Press, Washington, DC. doi: 10.1128/9781555816100.ch40

Key Concept Ranking

Gene therapy
0.518396
Human Growth Hormone
0.4885723
Severe Combined Immunodeficiency
0.43575367
0.518396
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Figures

Image of Figure 40.1
Figure 40.1

Enzyme replacement therapy for Gaucher's disease. In 1991, Roscoe Brady of NIH isolated enough glucocerebrosidase from human placentas to give injections of the missing enzyme to 12 patients. Even though the injections had dramatic effects, isolating the enzyme from placental tissue was not cost-effective. Using recombinant DNA techniques, the gene encoding the enzyme was engineered into yeast cells, making glucocerebrosidase replacement therapy a viable option. (Photographs courtesy of Roscoe Brady, NIH, NINDS.)

Citation: Kreuzer H, Massey A. 2008. Bioethics Case Study: Gene Therapy, p 585-595. In Molecular Biology and Biotechnology: A Guide for Teachers, Third Edition. ASM Press, Washington, DC. doi: 10.1128/9781555816100.ch40
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Image of Figure 40.1
Figure 40.1

Gene replacement therapy. For genetic defects that affect cells derived from bone marrow stem cells, such as severe combined immunodeficiency disease, bone marrow is removed from the patient, and the stem cells are multiplied in cell culture. A correct copy of the gene is inserted into a viral vector, using recombinant DNA techniques. The virus is cultured with the bone marrow cells. It infects the cells and inserts the replacement gene into some of them. Radiation destroys the patient's defective bone marrow cells, and the physician injects cultured cells, now containing the correct gene, into empty bone marrow cavities. To date, only a few of the gene replacement trials have been therapeutic.

Citation: Kreuzer H, Massey A. 2008. Bioethics Case Study: Gene Therapy, p 585-595. In Molecular Biology and Biotechnology: A Guide for Teachers, Third Edition. ASM Press, Washington, DC. doi: 10.1128/9781555816100.ch40
Permissions and Reprints Request Permissions
Download as Powerpoint

References

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