The project aims 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).  AD primarily affects the elderly, and is characterized by memory loss and impaired movement, reasoning and judgment.  The disease progressively worsens over time, resulting in massive disease burden to patients, their caregivers, and societies.  There are currently no effective treatments to reverse or halt the progression of AD, whereas disease prevalence is rapidly increasing due to aging populations worldwide.  Without effective clinical interventions, healthcare services around the world will eventually face dire consequences.

This project builds upon our previous TRS-funded research, which unraveled some of the complex mechanisms governing the regulatory pathways that underlie neurogenesis and differentiation of neural stem cells, and led to the discovery of a number of small molecules that stimulate endogenous neurogenesis.  Here, we aim to dissect the pathological mechanism of AD by leveraging our previous findings.  To identify disease pathways and novel therapeutic targets, we plan to draw on the power of human induced pluripotent stem cell (iPSC) and CRISPR-Cas9 genome-editing technologies. These state-of-the-art technologies will enable us to generate and utilize patient-derived iPSCs to conduct detailed investigations on the pathophysiology of AD.  In our foundational TRS, we identified several potential drug leads with memory enhancing activity. Hence, we will also undertake preclinical development of these drug leads.

Successful completion of the project will bring us one step closer to developing novel therapies that can halt or reverse the devastating effects of AD, thus improving millions of lives worldwide.  The project will also enhance Hong Kong’s growing reputation as a center for research excellence, while placing Hong Kong on the map for advanced neural regenerative medicine and stem cell research.