Gene therapy trials give mixed messages.
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References
Daly, T.M. et al. Prevention of systemic clinical disease in MPS VII mice following AAV-mediated neonatal gene transfer. Gene Therapy 8, 1291 - 1298 (2001).
Song, S. et al. Stable therapeutic serum levels of human alpha-1 antitrypsin (AAT) after portal vein injection of recombinant adeno-associated virus (rAAV) vectors. Gene Therapy 8, 1299 - 1306 (2001).
Donsante, A. et al. Observed incidence of tumourigenesis in long-term rodent studies of rAAV vectors. Gene Therapy 8, 1343 - 1346 (2001).
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Prevention of systemic clinical disease in MPS VII mice following AAV-mediated neonatal gene transfer. Nature (2001). https://doi.org/10.1038/news010906-13
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DOI: https://doi.org/10.1038/news010906-13