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pubmed-article:9771745pubmed:abstractTextGene 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.lld:pubmed
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pubmed-article:9771745pubmed:articleTitlePeroral gene therapy of lactose intolerance using an adeno-associated virus vector.lld:pubmed
pubmed-article:9771745pubmed:affiliationDepartment of Molecular Medicine, University of Auckland School of Medicine, New Zealand. Matthew.During@mail.tju.edulld:pubmed
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