iPS-Derived Microglia-Based Gene Therapy for Alzheimer's

Biju K Chandu, PhD The University of Texas Health Science Center at San Antonio


Senlin K Li, MD University of Texas Health Science Center
Robert A. K Clark, MD University of Texas Health Science Center


Extracting bone marrow cells requires surgery, which may be strenuous or impossible for older patients. To meet large-scale demand for bone marrow cells in clinical trials for the treatment of Alzheimer's disease, we will generate bone marrow cells from blood, which we in turn will genetically modify to secrete drugs once these cells migrate to the brain. This new approach is expected to contribute to the development of an important therapy for Alzheimer's.

Project Details

Therapies using your own (self‐derived) bone marrow cells have promise for treating many different diseases, including Alzheimer's disease. This type of self‐recognizing stem cell therapy has an increased chance of success, because one can avoid the pesky problem of the immune system labeling the cells as foreign invaders and killing them before they have a chance to work. Dr. Biju Chandu and collaborators will be testing a new bone marrow cell treatment for Alzheimer's disease. First, they will isolate white blood cells that roam freely in the easily-accessible blood vessels. These cells will in turn be genetically modified to become bone marrow cells and re-purposed to release anti-Alzheimer's disease drugs. The modified cells are injected back in the blood stream, where they migrate to the parts of the brain that require treatment. After this new treatment is tested in cell culture and animals in this project, the researchers may then decide to move this technique into human clinical trials.