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 |