Neural Stem Cell Repair

The brain is among the many organs in the human body that contain functional stem cells throughout life. The discovery of neural stem cells has led UCLA faculty to deepen their understanding of how the activity of these cells can be harnessed and to examine whether neural cells produced from hESC and iPSC may be used to replace or regenerate damaged or diseased neural tissue. Our active research agenda may one day lead to treatments for neurodegenerative and neuromuscular diseases as well as nerve related injuries and stroke.

Our faculty aim to understand the developmental mechanisms that build the neural circuits in the brain that enable cognitive functions such as learning and memory, as well as motor circuits in the brainstem and spinal cord that enable us to breathe, stand, move, and interact with the world. The relative inaccessibility of the human nervous system has long stood as a major roadblock towards studying the basis of many neurological diseases and in developing effective drugs to combat these disorders. Using human stem cells, we seek to overcome these barriers by creating novel cell culture-based models to explore disease mechanisms and serve as a platform for drug discovery.

UCLA faculty were the first to show that iPSC can be differentiated into electrically active motor neurons, a discovery that may aid in studying and treating neurological disorders, such as ALS (Lou Gehrig’s Disease), as well as spinal cord injuries through the use of a patient’s own cells. Our faculty are currently partnering with UCLA clinical departments in order to obtain skin cells from patients with various currently untreatable neurologic diseases including ALS, Rett Syndrome, Lesch-Nyhan Disease and Duchenne's Muscular Dystrophy in the hopes of using iPSC technology to produce a limitless source of human cells harboring disease-causing mutations that will advance our understanding of these disorders and potentially lead to the development of effective treatments.

Faculty are also focused on the process by which stem cells produce new neurons in the adult brain. Enhancing the generation of new neurons may aid in the treatment of Alzheimer’s or Parkinsons disease. Studies of this process may also lead to a better understanding of the origins of brain tumors, which may result from adult neural stem cells that have gone awry. Finally, faculty are conducting basic research and translational/preclinical studies that may enable the transplantation of stem cells into brain tissue damaged by stroke in order to regenerate healthy tissue and hopefully regain lost function.