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Published online 5 September 2001 | Nature | doi:10.1038/news010906-13
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Prevention of systemic clinical disease in MPS VII mice following AAV-mediated neonatal gene transfer
Gene therapy trials give mixed messages.
From the News and Features Section of the journal Nature
A promising approach to gene therapy received an important fillip this week with the publication of two papers suggesting that it can effectively treat rare diseases in mice.
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