Abstract
Gene therapy is usually reserved for severe and medically refractory disorders because of the toxicity, potential long-term risks and invasiveness of most gene transfer protocols. Here we show that an orally administered adeno-associated viral vector leads to persistent expression of a ß-galactosidase transgene in both gut epithelial and lamina propria cells, and that this approach results in long-term phenotypic recovery in an animal model of lactose intolerance. A gene 'pill' associated with highly efficient and stable gene expression might be a practical and cost-effective strategy for even relatively mild disorders, such as lactase deficiency.
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Acknowledgements
This work was supported by grants from the Juvenile Diabetes Foundation International, the New Zealand Health Research Council, New Zealand Marsden Fund and the New Zealand Lotteries Board.
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During, M., Xu, R., Young, D. et al. Peroral gene therapy of lactose intolerance using an adeno-associated virus vector. Nat Med 4, 1131–1135 (1998). https://doi.org/10.1038/2625
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DOI: https://doi.org/10.1038/2625
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