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Gene Therapy in Man and Mice: Adenosine Deaminase Deficiency, Ornithine Transcarbamylase Deficiency, and Duchenne Muscular Dystrophy

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Part of the Advances in Experimental Medicine and Biology book series (AEMB,volume 309B)

Abstract

Gene therapy is defined as the delivery and expression of a functional gene into somatic tissues of patients (or animals) endogenously deficient in this gene. Depending on the natural tissue pattern of expression and the sites most affected by disease processes, different anatomic sites may be the targets for gene transduction. To date, retrovirus-mediated gene transfer into hemopoietic stem cells is the most advanced of gene therapy systems and the most promising in terms of clinical applications in the near future. There are, however, additional efforts under way in our laboratory to develop efficient means of transducing genes into the liver, intestine, skeletal and cardiac muscles.

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  • DOI: 10.1007/978-1-4615-7703-4_12
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References

  • Jones, S.N., Grompe, M., Munir, M.I., Veres, G., Craigen, W.J., and Caskey, C.T., 1990, Ectopic correction of ornithine transcarbamylase deficiency in sparse fur mice. J. Biol. Chem 265: 14–684.

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  • Lee, C.C., Pearlman, J.A., Chamberlain, J.S., and Caskey, C.T., 1991, Expression of recombinant dystrophin and its localization to the cell membrane. Nature 349: 334.

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© 1991 Plenum Press, New York

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Grompe, M., Mitani, K., Lee, CC., Jones, S.N., Caskey, C.T. (1991). Gene Therapy in Man and Mice: Adenosine Deaminase Deficiency, Ornithine Transcarbamylase Deficiency, and Duchenne Muscular Dystrophy. In: Harkness, R.A., Elion, G.B., Zöllner, N. (eds) Purine and Pyrimidine Metabolism in Man VII. Advances in Experimental Medicine and Biology, vol 309B. Springer, New York, NY. https://doi.org/10.1007/978-1-4615-7703-4_12

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  • DOI: https://doi.org/10.1007/978-1-4615-7703-4_12

  • Publisher Name: Springer, New York, NY

  • Print ISBN: 978-1-4615-7705-8

  • Online ISBN: 978-1-4615-7703-4

  • eBook Packages: Springer Book Archive