Gene Therapy for Human Genetic Disease?

@article{Friedmann1972GeneTF,
  title={Gene Therapy for Human Genetic Disease?},
  author={Theodore Friedmann and Richard Roblin},
  journal={Science},
  year={1972},
  volume={175},
  pages={949 - 955}
}
In our view, gene therapy may ameliorate some human genetic diseases in the future. For this reason, we believe that research directed at the development of techniques for gene therapy should continue. For the foreseeable future, however, we oppose any further attempts at gene therapy in human patients because (i) our understanding of such basic processes as gene regulation and genetic recombination in human cells is inadequate; (ii) our understanding of the details of the relation between the… 

THE PROSPECTS FOR GENE THERAPY IN HUMANS

  • J. Morrow
  • Biology
    Annals of the New York Academy of Sciences
  • 1976
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  • P. Boudes
  • Medicine, Biology
    European journal of internal medicine
  • 2014

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The purpose of this review is to focus on merit and demerit of gene therapy and to provide information about its future prospective.

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References

SHOWING 1-10 OF 42 REFERENCES

The Use of DNA for Gene Therapy—the Need, Experimental Approach, and Implications

  • H. Aposhian
  • Biology, Chemistry
    Perspectives in biology and medicine
  • 1970
These basic scientific findings are the foundation for many other technical developments that are needed for the eventual treatment or cure of certain kinds of human diseases.

Dietary treatment of inborn errors of metabolism.

In this review, disorders which exemplify each of these modes of therapy are described in sufficient detail to permit an appreciation of two problems: management of the disease under discussion, and evaluation of therapy of inborn errors in general.

DNA‐mediated transformation of mammalian cells in culture. Increased transforming efficiency following sonication

A statistical analysis of 100 experiments demonstrated that the increase in the number of resistant cells after treatment with sonicated resistant DNA (R‐DNA) was statistically significant, and that the efficiency of transformation was greatly increased by sonication of the DNA.

Uptake of Protein by Mammalian Cells: An Underdeveloped Area

With labeled proteins used as models, it is found that the transport proceeds at very low rates, requires little energy, and is markedly enhanced by polybasic compounds, suggesting that macromolecules have only a limited chance to express their biological activity in target cells.

Studies of the mechanism of action of the Shope rabbit papilloma virus. I. Concerning the nature of the induction of arginase in the infected cells.

These studies make plain that the Shope papilloma virus induces the production of an arginase in rabbit squamous epithelium, and provide evidence that the information for the synthesis of the enzyme

Enzyme Replacement in Fabry's Disease, an Inborn Error of Metabolism

Two patients with Fabry's disease were infused with normal plasma to provide active enzyme (ceramide trihexosidase) for hydrolysis of the plasma substrate, galactosylgalacto-glucosylceramide, and the amount of accumulated substrate in the plasma of these recipients decreased about 50 percent on day 10 after infusion.

DNA and gene therapy: uncoating of polyoma pseudovirus in mouse embryo cells.

Pseudovirions and polyoma virions are produced after infection of primary mouse embryo cells with polyoma virus and the implications of these experiments to the eventual use of DNA for gene therapy are discussed.

Prenatal genetic diagnosis. I.