Biotechnology An Industry Comes of Age (1986) / Chapter Skim
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Human Gene Therapy
Human Gene Therapy
Pages 43-53
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From page 43... ...
to recombinant DNA coding for the blood protein hemoglobin, hoping that the bone marrow would incorporate the new genes and alleviate the patients' disease. Neither of these first two attempts at human gene therapy had an effect on the patients involved.
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From page 44... ...
It is difficult to conceive of how genetic engineering could ever be used to affect these complex human traits. But that does not mean that the broad moral and social implications of human gene therapy should not be the subjects of continuing reflection.
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From page 45... ...
Second, the diseases will be treatable through the genetic manipulation of bone marrow cells, because techniques have been developed to remove these cells from the body, transform them with recombinant DNA, and reintroduce them into the body. Perhaps in the future it will be possible to genetically manipulate skin cells and even tissues and whole organs, but for now bone marrow cells are the only cells conducive to this kind of treatment.
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From page 46... ...
By infecting the defective bone marrow cells of such patients with genetically engineered viruses containing the gene that codes for ADA, researchers hope to cure the disease. year, making this the most common of the initial candidates for gene therapy.
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From page 47... ...
After the bone marrow cells have been transformed, they will be reimplanted into the patient. A limited number of studies have THE GENETIC ENGINEERING OF HUMAN CELLS Retrovirus Human Cell with Functional Gene » DNA Equivalent of Retroviral » RNA with Major Genes Deleted DNA -Functional Gene Recombinant Retrovirus Bone Marrow Cell with Defective Gene Bone Marrow Cell Containing Functional Gene Upcoming attempts at human gene therapy will use retroviruses, infectious agents that have the ability to insert a single strand of DNA into the DNA of cells they infect.
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From page 48... ...
Nevertheless, a thorough review process has been set up to monitor the initial attempts at human gene therapy. After review by local Institutional Review Boards and Institutional Biosafety Committees, the research protocols for human gene therapy will have to be approved by a working group of NIH's Recombinant DNA Advisory Committee (RAC)
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From page 49... ...
The protocol then has to be approved at three separate levels within the National Institutes of Health. Finally, one or more other groups or individuals may have to approve the protocol, including the secretary of Health and Human Services, the commissioner of the Food and Drug Administration, the interagency coordinating committee proposed by the Cabinet Council Working Group on Biotechnology (discussed in Chapter 6)
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From page 50... ...
For instance, it may someday be possible to conduct gene therapy on cells within the body as well as on cells withdrawn from the body. If genetically engineered retroviruses could be designed that home in on certain types of somatic cells when injected into the body, DNA could be delivered to specific body tissues.
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From page 51... ...
Some of the mice treated with growth hormone genes suffered from gigantism, so that parts of their bodies grew disproportionately large, and the metabolic imbalances caused by the inappropriate expression of growth hormone left nearly all the genetically engineered female mice sterile. Because of these and other technical difficulties, there are no plans now being made to attempt germline gene therapy in human beings, and the prospects for any such attempts in at least the near future look dim.
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From page 52... ...
"But it is something that might very well come up in later years." This ability to change specific physiological indexes should be sharply distinguished from the type of genetic engineering that has generated the most concern among the public, according to Anderson. This latter type of genetic engineering involves changing complex human traits -- like intelligence, character, and physical appearance -- that are shaped by a subtle interplay of many interacting genes and environmental influences.
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From page 53... ...
1984. Human Gene Therapy -- Background Paper.
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