Gene Therapy for Alzheimer's Disease

Alzheimer’s Disease is associated with the death of a particular type of neuron in a particular region of the brain. These neurons require a protein, nerve growth factor (NGF), for their survival. Therefore, it is possible that increasing the concentration of nerve growth factor in the appropriate part of the brain may be a treatment for Alzheimer’s Disease. Gene therapy, a new approach to treating disease, involves the introduction of a gene into cells to cure or prevent disease. Thus, introduction of the nerve growth factor gene into the appropriate part of the brain may be a powerful new treatment for Alzheimer’s Disease. We have developed a new gene transfer procedure, using an inactivated Herpes Simplex Virus, which allows for the first time the introduction of a gene into brain cells. Hypothesis Introduction of the nerve growth factor gene into the appropriate brain cells using our new gene transfer procedure may be an effective gene therapy treatment for Alzheimer’s Disease.

Specific Aims

First, demonstrate that we can deliver the NGF gene into cultured brain cells and that expression of NGF can support the growth of nerve cells that require NGF. Second, determine the best type of brain cell in which to express NGF, for example neuron versus glia. Third, develop a safer Herpes Virus which can be used in humans.

Long Term Goals

Test our gene therapy treatment for Alzheimer’s Disease in animal models of  Alzheimer’s Disease and then proceed to clinical trials.