On 17 January 2025, Kam W. Leong, an academician of the National Academy of Engineering (NAE), a member of the National Academy of Medicine (NAM), and a professor of the Department of Biomedical Engineering at Columbia University, visited our institute and gave a lecture entitled 'Focused Ultrasound-Enhanced Gene Editing: A Potential Therapeutic Approach for Alzheimer’s Disease'. The lecture was held in Conference Room A105, Mingde Building, Ren'ai Road Campus, and was actively attended by faculty and students from the School of Biomedical Engineering, University of Science and Technology of China (USTC).

In this lecture, Professor Leong discussed the potential of gene editing as a novel therapeutic strategy for the treatment of neurodegenerative diseases, including the complex and under-researched condition of Alzheimer's disease (AD). Among the molecular factors associated with late-onset Alzheimer's disease, the APOE4 gene has been identified as a significant genetic risk factor in the pathogenesis of AD. Although CRISPR technology has shown great potential in targeted gene-editing therapies, its application in brain diseases is severely hampered by the blood-brain barrier (BBB), which also limits the effective delivery of gene-editing components. Professor Leung Kam Wing's team has developed a non-invasive, safe, and efficient method using focused ultrasound (FUS) technology to temporarily open the blood-brain barrier, thereby enhancing the delivery of therapeutic drugs to the brain. Professor Leung also shared the latest progress made by his team in achieving gene editing in the mouse brain through viral and non-viral vectors, and explored the potential applications and impacts of this technology in treating Alzheimer's disease and other neurodegenerative diseases.

At the end of the lecture, Professor Leung had an in-depth discussion with the attending teachers and students on the application of gene editing technology in the treatment of neurodegenerative diseases, the breakthrough of the blood-brain barrier and the key issues of focused ultrasound technology, which further deepened the understanding of the attending teachers and students on the potential application of gene editing technology in the treatment of Alzheimer's disease. The lecture was successfully concluded with warm applause.