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Prospects for Gene Insertion Therapy in Early Life

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Molecular Genetics in Medicine
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Abstract

The deliberate insertion of specific DNA sequences into a person’s genome to alter the way his body functions is the recombinant DNA technique that comes closest to the popular concept of genetic engineering. It is also the prospect that has engendered the most extreme reactions from some members of the public. The scientists and clinicans working on these projects tend to be either hailed as the new stars of medicine — the gene doctors — who will neatly cure genetic diseases by replacing harmful genes, or they are regarded as a monstrous threat to society. In this contribution I indicate why some of the wilder misconceptions may have arisen, as well as putting the future contribution of gene therapy into some general perspective with respect to all the clinical services being developed to help families afflicted with serious genetic disease.

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References

  • Anderson, F. W. (1984) ‘Prospects for human gene therapy’, Science, vol. 226, pp. 401–9.

    Article  CAS  PubMed  Google Scholar 

  • Berenson, R. J., R. G. Andrews, W. I. Bensinger, D. Kalamasz, G. Knitter, C. D. Buckner and I. D. Bernstein (1988) ‘Antigen CD34+ marrow cells engraft lethally irradiated baboons’, Journal of Clinical Investigation, vol. 81, pp. 951–5.

    Article  PubMed Central  CAS  PubMed  Google Scholar 

  • Capecchi, M. R. (1989) ‘Altering the genome by homologous recombination’, Science, vol. 244, pp. 1288–92.

    Article  CAS  PubMed  Google Scholar 

  • Dick, J. E., M. C. Magli, D. Huszar, R. A. Phillips and A. Bernstein (1985) ‘Introduction of a selectable gene into primitive stem cells capable of long-term reconstitution of the hemopoietic system of W/Wv mice’, Cell, vol. 42, pp. 71–9.

    Article  CAS  PubMed  Google Scholar 

  • Dzierzak, E. A., T. Papayannopoulou and R. C. Mulligan (1988) ‘Lineage-specific expression of a human β-globin gene in murine bone marrow transplant recipients reconstituted with retrovirus-transduced stem cells’, Nature, vol. 331, pp. 35–41.

    Article  CAS  PubMed  Google Scholar 

  • Friedmann, T. (1989) ‘Progress toward human gene therapy’, Science, vol. 244, pp. 1275–81.

    Article  CAS  PubMed  Google Scholar 

  • Greaves, D. R., F. D. Wilson, G. Lang and D. Kioussis (1989) ‘Human CD2 3’-flanking sequences confer high-level, T cell-specific, position-independent gene expression in transgenic mic’, Cell, vol. 56, pp. 979–86.

    Article  CAS  PubMed  Google Scholar 

  • Hershfield, M. S., R. H. Buckley, M. L. Greenberg, A. L. Melton, R. Schiff, C. Hatem, J. Kurtzberg, M. L. Markert, R. H. Kobayashi, A. L. Kobayashi and A. Abuchowski (1987) ‘Treatment of adenosine deaminase deficiency with polyethylene glycol-modified adenosine deaminase’, New England Journal of Medicine, vol. 316, pp. 589–95.

    Article  CAS  PubMed  Google Scholar 

  • Holding, C. and M. Monk (1989) ‘Diagnosis of β-thalassaemia by DNA amplification in single blastomeres from mouse preimplantation embryos’, The Lancet, no. ii, pp. 532–5.

    Article  CAS  Google Scholar 

  • Kantoff, P. W., S. M. Freeman and W. French Anderson (1988) ‘Prospects for gene therapy for immunodeficiency diseases’, Annual Reviews in Immunology, vol. 6, pp. 581–94.

    Article  CAS  Google Scholar 

  • Keller, G., C. Paige, E. Gilboa and E. R. Wagner (1985) ‘Expression of a foreign gene in myeloid and lymphoid cells derived from multipotent haematopoietic precursors’, Nature, vol. 318, pp. 149–54.

    Article  CAS  PubMed  Google Scholar 

  • Ledley, F. D. (1987) ‘Somatic gene therapy for human disease’, The Journal of Paediatrics, vol. 110, pp. 1–8, 167–74.

    Article  CAS  Google Scholar 

  • Levinsky, R. J. (1989) ‘Recent advances in bone marrow transplantation’, Clinical Immunology and Immunopathology, vol. 50, pp. 124–32.

    Article  Google Scholar 

  • Pembrey, M. E., W. G. Wood, D. J. Weatherall and R. P. Perrine (1978) ‘Fetal haemoglobin production and the sickle gene in the oases of Eastern Saudi Arabia’, British Journal of Haematology, vol. 40, pp. 415–29.

    Article  CAS  PubMed  Google Scholar 

  • Smithies, O., R. G. Gregg, S. S. Boggs, M. A. Koralewski and R. S. Kucherlapati (1985) ‘Insertion of DNA sequences into the human chromosomal β-globin locus by homologous. recombination’, Nature, vol. 317, pp. 230–4.

    Article  CAS  PubMed  Google Scholar 

  • Spangrude, G. J., S. Heimfeld and I. L. Weissman (1988) ‘Purification and characterisation of mouse hematopoietic stem cells’, Science, vol. 241, pp. 58–61.

    Article  CAS  PubMed  Google Scholar 

  • Talbot, D., P. Collis, M. Antoniou, M. Vidal, F. Grosveld and D. R. Greaves (1989) ‘A dominant control region from the human 13-globin locus conferring integration site-independent gene expression’, Nature, vol. 338, pp. 352–5.

    Article  CAS  PubMed  Google Scholar 

  • Watt, S. M., K. Karhi, K. Gatter, A. J. W. Furley, F. E. Katz, L. J. Healy, L. J. Atass, N. J. Bradley, D. R. Sutherland, R. J. Levinsky and M. F. Greaves (1987) ‘Distribution and epitope analysis of the cell membrane glycoprotein (HPCA-1) associated with human hemopoietic progenitor cells’, Leukemia, vol. 1, pp. 417–26.

    CAS  PubMed  Google Scholar 

  • Williams, D. A. (1988) ‘Gene transfer and the prospects for somatic gene therapy’, Hematology/oncology clinics of North America, vol. 2, pp. 277–87.

    CAS  PubMed  Google Scholar 

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© 1991 The Galton Institute

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Pembrey, M. (1991). Prospects for Gene Insertion Therapy in Early Life. In: Roberts, D.F., Chester, R. (eds) Molecular Genetics in Medicine. Palgrave Macmillan, London. https://doi.org/10.1007/978-1-349-10874-9_7

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