Intravenous GDNF Gene Therapy of Experimental Parkinson's Disease

Parkinson's disease is caused by the loss of cells in the brain that produce a chemical called dopamine, which is also called a monoamine. The loss of these cells has been delayed in pre-clinical models by the administration of GDNF (glial derived neurotrophic factor), a specialized protein known as a trophic factor which stimulates the dopamine-producing cells. However, this protein does not naturally cross the blood-brain barrier (BBB), meaning it cannot be administered orally (in pill form).

The present work will use a new form of gene therapy, which does not require the use of viruses to deliver the gene, and which can be administered non-invasively by periodic intravenous administration. The new approach uses Trojan horse liposomes to target the GDNF gene across the BBB. A specific "gene switch" will be used to insure the GDNF gene is produced only in the monamine producing cells in brain.