Three recent reports describe the first in vivo attempts at fetal gene therapy. The results underline the need for more intensive studies of the scientific and ethical implications of this new and perhaps more preventive approach to gene therapy.
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References
Pitt, B.R. et al. Retrovirus-mediated gene transfer in lungs of living fetal sheep. Gene Ther. 2, 344–350 (1995).
McCray, P.B. et al. Adenoviral mediated gene transfer to fetal pulmonary epithelia in vitro and in vivo. J. din. Invest. 95, 2620–2632 (1995).
Tsukamoto, M., Ochiya, T., Yoshida, S., Sugimura, T. & Terada, M. Gene transfer and expression in progeny after intravenous DNA injection into pregnant mice. Nature Genet. 9, 243–248 (1995).
Coutelle, C., Caplen, N., Hart, S., Middleton, P.G. & Williamson, R. Towards gene therapy for cystic fibrosis. in Cystic Fibrosis, Current Topics, Vol. 2 (eds Dodge, J.A., Brock, D.J.H., Widdicombe, J.H.) 33–54 (Wiley, Chichester, 1994).
Reece, E.A., Homko, C., Goldstein, I. & Wiznitzer, A. Towards fetal therapy using needle embryofetoscopy. Ultrasound Obstet. Gynaecol. 5, 281–285 (1995).
Touraine, J.-L. et al. In utero transplantation of stem cells in humans: Immunological aspects and clinical follow-up of patients. Bone Marrow Transplant. 9 (suppl. 1), 121–126 (1992).
Gillman, J. The development of the gonads in man, with a consideration of the role of fetal endocrines and histogenesis of ovarian tumours. Contrib. Embryol. 32, 81–92 (1948).
Tizzano, E.F., Chitayat, D. & Buchwald, M. Cell-specific localization of CFTR mRNA shows devel-opmentally regulated expression on human fetal tissues. Hum. molec. Genet. 2, 219–224 (1993).
Trezise, A.E.O., Chambers, J.A., Wardle, C.J., Gould, S. & Harris, A. Expression of the cystic fibrosis gene in human foetal tissues. Hum. molec. Genet. 2, 213–218 (1993).
Boué, A. et al. Prenatal diagnosis in 200 pregnancies with a l-in-4 risk of cystic fibrosis. Hum. Genet. 74, 288–297 (1986).
Zabner, J. et al. Adenovirus-mediated gene transfer transiently corrects the transport defect in nasal epithelia of patients with cystic fibrosis. Cell 75, 207–216 (1993).
Crystal, R.G. et al. Administration of an aden-ovirus containing the human CFTR cDNA to the respiratory tract of individuals with cystic fibrosis. Nature Genet. 8, 42–51 (1994).
Caplen, N.J. et al. Liposome-mediated CFTR gene transfer to the nasal epithelia of patients with cystic fibrosis. Nature Med. 1, 39–46 (1995).
Clothier, C.M. Report of the Committee on the Ethics of Gene Therapy (HMSO, 1992).
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Coutelle, C., Douar, AM., Colledge, W. et al. The challenge of fetal gene therapy. Nat Med 1, 864–866 (1995). https://doi.org/10.1038/nm0995-864
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DOI: https://doi.org/10.1038/nm0995-864
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