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 -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.
-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.
