The research team at Hong Kong Center for Neurodegenerative Diseases (HKCeND) has developed a novel strategy using brain-wide genome-editing technology that can effectively eliminate genetic mutations of familial Alzheimer’s disease (AD) and ameliorate disease pathologies in AD mouse models. This advanced technology offers immense potential to be translated into a new long-acting therapeutic strategy for AD patients.
Familial Alzheimer’s disease (FAD) accounts for 3-5% of AD cases. Although it has a clear genetic cause, no effective disease-modifying treatments currently exist. Genome-editing technology has the potential as a therapeutic strategy for FAD, which can eliminate disease-causing genetic mutations before symptoms appear and has “once-and-for-all” long-acting treatment effects. However, several hurdles have prevented its clinical development and application - most notably the lack of an effective, efficient, and non-invasive means to deliver genome-editing agents into the brain. Furthermore, existing genome-editing technologies are unable to generate beneficial outcomes throughout the whole brain. This brain-wide genome-editing technology uses a newly engineered delivery vehicle for genome-editing and achieves efficient brain-wide genome editing through a single non-invasive intravenous administration, representing a promising approach for FAD treatment.
Hong Kong Center for Neurodegenerative Diseases (HKCeND) was established under the government-funded InnoHK initiative. HKCeND harnesses the power of science to make a translational impact in the development of innovative diagnostic tools and therapeutic strategies for neurodegenerative diseases.
Building on the pioneering work of HKUST’s State Key Laboratory of Molecular Neuroscience led by neuroscientist Prof. Nancy Ip, HKCeND has fostered cross-institutional R&D collaboration with University College London and Stanford University.
This interdisciplinary collaboration is bringing together prominent scientists in the areas of neuroscience, stem cell biology and artificial intelligence to advance cutting-edge research and deliver promising breakthroughs that improve and transform the live of people worldwide.