Publication Type Academic Article
Authors During M, Xu R, Young D, Kaplitt M, Sherwin R, Leone P
Journal Nat Med
Volume 4
Issue 10
Pagination 1131-5
Date Published 10/01/1998
ISSN 1078-8956
Keywords Dependovirus, Genetic Therapy, Genetic Vectors, Lactose Intolerance, beta-Galactosidase
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 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.
DOI 10.1038/2625
PubMed ID 9771745
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