Use of Defective Herpes-Derived Plasmid Vectors

Genetic intervention is becoming increasingly useful in elucidating the molecular basis of various biological processes, including those of the brain. Many genes have now been isolated which encode key regulatory molecules such as signal transduction components or transcription factors, but relatively little is known about their effects on neuronal physiology or higher order brain function. Gene transfer experiments offer the potential to study the effects of highly specific alterations in gene products in their normal neuronal environment. In addition, treatments of nervous disorders may also be devised using the same technology. Gene therapy protocols may be targeted towards specific diseases (e.g., the treatment of Parkinson’s disease by expression of tyrosine hydroxylase in the striatum), or directed towards the treatment of general neuronal damage which may occur in many diseased conditions (e.g., prevention of cell death by expression of neurotrophins or neurotrophin receptors). Further knowledge of the molecular mechanisms underlying neuronal physiology may lead to insight into the nature of the defects in diseased conditions. The ability to manipulate normal physiological pathways specifically may also result in new treatments. For example, the regulation of neurotransmission is altered in many disorders such as Alzheimer’s disease, Parkinson’s disease, and epilepsy, and may be responsive to genetic intervention such as expression of signal transduction components controlling long-term changes in neurotransmitter release.