GENE THERAPY OF THE NERVOUS SYSTEM

摘要

A method of treating an adverse condition of the nervous system in a chordate host (preferably a mammalian host) comprising administering to the cerebrospinal fluid of the host an expression vehicle capable of transducing a cell in order to express a therapeutic agent in the nervous system. The expression vehicle includes a nucleic acid sequence encoding a therapeutic agent for treating an adverse condition of the nervous system, and is administered in an amount effective in treating the adverse condition of the nervous system. The method of the present invention is particularly applicable to the treatment of meningeal carcinomatosis, wherein producer cells containing a retroviral vector including a gene encoding a negative selective marker (such as Herpes Simplex thymidine kinase) are administered to the cerebrospinal fluid of a patient. Such producer cells generate viral particles which transduce the meningeal carcinomatosis cells. The transduced meningeal carcinomatosis cells then are killed upon administration of an interaction agent (such as ganciclovir) which interacts with the negative selective marker.