| abstract |
The present invention provides a therapeutic product to a subject using a medicament-preferably replication-defective viral composition (s) engineered with a visceral safety mechanism that permanently or temporarily removes the therapeutic gene product in response to an oral formulation such as a pill. Related to gene therapy systems designed for the delivery of. The present invention is based, in part, on the development of volunteers of an integrated approach to eliminate transgenes or to negatively regulate transgene expression, referred to herein as "PITA" (pharmaceutically induced transgene ablation). In this approach, replication-defective viruses are used to deliver a transgene encoding a therapeutic product (RNA or protein) to be expressed in a subject but specific for, for example, this gene or its RNA transcripts by administration of a medicament. It can be released reversibly or irreversibly by administration of small molecules that cause expression of the ablation. |