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Nat Med. 1998 Oct;4(10):1131-5.

Peroral gene therapy of lactose intolerance using an adeno-associated virus vector.

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Department of Molecular Medicine, University of Auckland School of Medicine, New Zealand.


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 beta-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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