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

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