THEME 1
A Stem Cell Approach to Dissect the Molecular Basis of Neurodegenerative Diseases
T13-605/18-W
Project Coordinator
Professor Nancy IP
The Hong Kong University of Science and Technology
Coordinating Institution
- The Hong Kong University of Science and Technology
Participating Institution(s)
- City University of Hong Kong
- The Chinese University of Hong Kong
- The University of Hong Kong
Abstract
This project represents our continuing effort to address the urgent need for new and innovative therapies that can treat age-related neurodegenerative disorders, such as the highly prevalent and devastating Alzheimer's disease (AD). We have established the capability and platforms for iPSC differentiation into specific brain cell types and brain organoids. The current project builds upon these findings and further explores the pathological mechanism of AD using innovative approaches that harness advanced stem cell and genome-editing technologies. With these technologies, we have succeeded in generating and utilizing patient-derived iPSCs that carry genetic risk variants for AD to conduct detailed investigations on the disease pathophysiology. The establishment of the iPSC-derived neuronal platform provides a useful tool to evaluate the efficacy and safety of drug candidates on human neurons. Furthermore, we have developed a novel strategy using brain-wide genome-editing technology that can reduce AD pathologies in genetically modified AD mouse models. This advanced technology has garnered significant attention and awards, including the 2021 "Major Breakthrough in Neuroscience" award from the Chinese Neuroscience Society and a Gold Medal at the 49th International Exhibition of Inventions Geneva, for its potential to be translated into a novel long-acting therapeutic treatment for AD patients. It is among the first selected for the “Research, Academic and Industry Sectors One-plus (RAISe+) Scheme” by the Innovation and Technology Commission of the HKSAR Government.
Research Impact
We have succeeded in developing advanced capabilities and generating valuable data towards establishing groundwork to develop novel therapies to treat incurable neurological diseases, such as AD, and hence addressing critical unmet medical needs and easing the associated financial burdens. In the long run, these newly established advanced capabilities and technologies will drive research excellence at local institutions, increase collaborations with mainland China and international institutes, provide training opportunities for young scientists, place Hong Kong on the map for advanced neural regenerative medicine and stem cell research, and will ultimately strengthen Hong Kong's position as an international innovation and technology centre.
The Hong Kong University of Science and Technology
Professor Nancy Ip received her PhD degree in Pharmacology from Harvard University, after which she held the position of Senior Staff Scientist at Regeneron Pharmaceuticals Inc. in New York. Professor Ip joined HKUST in 1993 and is currently The Morningside Professor of Life Science and Chair Professor in the Division of Life Science. She is internationally renowned for her significant contributions to the field of neuroscience, particularly in understanding the complex mechanisms that underlie proper brain functions and in drug discovery for neurodegenerative diseases. Her outstanding research has resulted in more than 330 scientific papers and over 70 patents. She was elected as Fellows of the Chinese Academy of Sciences, the US National Academy of Sciences, and the American Academy of Arts and Sciences, among other academies, and is a recipient of numerous awards and honors, including the National Natural Science Awards and the L’OREAL-UNESCO for Women in Science Award.