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December 11, 1981, Vol 246, No. 23 >
ARTICLE | December 11, 1981

Gene Therapy FREE

W. French Anderson, MD
[+] Author Affiliations

Reprint requests to Laboratory of Molecular Hematology, Bldg 10, Room 7D-18, National Heart, Lung, and Blood Institute, Bethesda, MD 20205 (Dr Anderson).


JAMA. 1981;246(23):2737-2739. doi:10.1001/jama.1981.03320230059029
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Article
References

Can Gene Therapy Help Me?  This article is designed to help the practicing physician answer the patient with a genetic disease who asks, "Can gene therapy help me?" The answer depends on the patient's disease, but for some genetic conditions, gene therapy should be a beneficial therapeutic procedure in the coming years. This brief review examines the present state of the art, points out what still needs to be accomplished before gene therapy will be practical, and concludes by suggesting the potential and the limitations of this promising, but not yet available, technology.Progress in the area of "genetic engineering" has been spectular in recent years. Not only are bacteria being programmed to produce medically useful compounds (insulin, growth hormone, etc), but genes have been transferred into mammalian cells successfully. Individual cells in tissue culture have been cured of genetic defects by insertion of a normal gene.1-3 Purified genes

REFERENCES

1 +
Pellicer A, Robins D, Wold B, et al:  Altering genotype and phenotype by DNA-mediated gene transfer . Science 1980;;209:1414-1422.
2 +
Mulligan RC, Berg P:  Expression of a bacterial gene in mammalian cells . Science 1980;; 209:1422-1427.
3 +
Anderson WF, Killos L, Sanders-Haigh L, et al:  Replication and expression of thymidine kinase and human globin genes microinjected into mouse fibroblasts . Proc Natl Acad Sci USA 1980;;77:5399-5403.
4 +
Cline MJ, Stang H, Mercola K, et al:  Gene transfer in intact animals . Nature 1980;;284:422-425.
5 +
Mercola KE, Stang HD, Browne J, et al:  Insertion of a new gene of viral origin into bone marrow cells of mice . Science 1980;;208:1033-1035.
6 +
Gordon JW, Scangos GA, Plotkin DJ, et al:  Genetic transformation of mouse embryos by microinjection of purified DNA . Proc Natl Acad Sci USA 1980;;77:7380-7384.
7 +
Wagner TE, Hoppe PC, Jollick JD, et al:  Microinjection of a rabbit β-globin gene into zygotes and its subsequent expression in the adult mice and their offspring . Proc Natl Acad Sci USA , to be published.
8 +
Kolata GB, Wade N:  Human gene treatment stirs new debate . Science 1980;;210:407.
9 +
Mercola KE, Cline MJ:  The potentials of inserting new genetic information . N Engl JMed 1980;;303:1297-1300.
10 +
Durant JR:  Science, ethics and due process . Ann Intern Med 1981;;95:116-117.
11 +
Pellicer A, Wigler M, Axel R, et al:  The transfer and stable integration of the HSV thymidine kinase gene into mouse cells . Cell 1978;;14:133-141.
12 +
Mulligan RC, Howard BH, Berg P:  Synthesis of rabbit β-globin in cultured monkey kidney cells following infection with a SV40 β-globin recombinant genome . Nature 1979;;277:108-114.
13 +
Hamer DH, Smith KD, Boyer SH, et al:  SV40 recombinants carrying rabbit β-globin gene coding sequences Cell 1979;;17:725-735.
14 +
Anderson WF, Diacumakos EG:  Genetic engineering in mammalian cells . Sci Am 1981;; 245:106-121.
15 +
Kurtz DT:  Hormonal inducibility of rat α2u-globulin genes in transfected mouse cells . Nature 1981;;291:629-631.
16 +
Anderson WF, Fletcher JC:  Gene therapy in human beings: When is it ethical to begin? N Engl J Med 1980;;303:1293-1297.

Figures

Tables

Interactive Graphics

Video

Country-Specific Mortality and Growth Failure in Infancy and Yound Children and Association With Material Stature

Use interactive graphics and maps to view and sort country-specific infant and early dhildhood mortality and growth failure data and their association with maternal

1 +
Pellicer A, Robins D, Wold B, et al:  Altering genotype and phenotype by DNA-mediated gene transfer . Science 1980;;209:1414-1422.
2 +
Mulligan RC, Berg P:  Expression of a bacterial gene in mammalian cells . Science 1980;; 209:1422-1427.
3 +
Anderson WF, Killos L, Sanders-Haigh L, et al:  Replication and expression of thymidine kinase and human globin genes microinjected into mouse fibroblasts . Proc Natl Acad Sci USA 1980;;77:5399-5403.
4 +
Cline MJ, Stang H, Mercola K, et al:  Gene transfer in intact animals . Nature 1980;;284:422-425.
5 +
Mercola KE, Stang HD, Browne J, et al:  Insertion of a new gene of viral origin into bone marrow cells of mice . Science 1980;;208:1033-1035.
6 +
Gordon JW, Scangos GA, Plotkin DJ, et al:  Genetic transformation of mouse embryos by microinjection of purified DNA . Proc Natl Acad Sci USA 1980;;77:7380-7384.
7 +
Wagner TE, Hoppe PC, Jollick JD, et al:  Microinjection of a rabbit β-globin gene into zygotes and its subsequent expression in the adult mice and their offspring . Proc Natl Acad Sci USA , to be published.
8 +
Kolata GB, Wade N:  Human gene treatment stirs new debate . Science 1980;;210:407.
9 +
Mercola KE, Cline MJ:  The potentials of inserting new genetic information . N Engl JMed 1980;;303:1297-1300.
10 +
Durant JR:  Science, ethics and due process . Ann Intern Med 1981;;95:116-117.
11 +
Pellicer A, Wigler M, Axel R, et al:  The transfer and stable integration of the HSV thymidine kinase gene into mouse cells . Cell 1978;;14:133-141.
12 +
Mulligan RC, Howard BH, Berg P:  Synthesis of rabbit β-globin in cultured monkey kidney cells following infection with a SV40 β-globin recombinant genome . Nature 1979;;277:108-114.
13 +
Hamer DH, Smith KD, Boyer SH, et al:  SV40 recombinants carrying rabbit β-globin gene coding sequences Cell 1979;;17:725-735.
14 +
Anderson WF, Diacumakos EG:  Genetic engineering in mammalian cells . Sci Am 1981;; 245:106-121.
15 +
Kurtz DT:  Hormonal inducibility of rat α2u-globulin genes in transfected mouse cells . Nature 1981;;291:629-631.
16 +
Anderson WF, Fletcher JC:  Gene therapy in human beings: When is it ethical to begin? N Engl J Med 1980;;303:1293-1297.

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