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. 研究影響 我們擁有先進的研究能力並取得重要數據，為治理 AD 等無法 治癒的神經系統疾病提供了理論基礎，有助應對醫療需求缺口 及減輕相關的經濟重擔。長遠而言，這些先進的科研能力和技 術將有助推動本港卓越研究，加強與內地和國際機構的合作， 為年輕科學家提供培訓機會，讓香港在先進神經再生醫學和幹 細胞研究領域居前，鞏固香港作為國際創新科技中心的地位。 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 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. 項目簡介 我們持續努力探尋創新療法應對與老齡相關的神經退化性疾 病，例如嚴重威脅人們健康的高發病 AD，以滿足迫切的需要。 我們建立了平臺，能將 iPSC 分化成特定腦細胞類型和產生腦 類器官。本項目在此基礎上，利用先進的幹細胞和基因組編輯 技術進一步探索 AD 的病理機制。我們建立了源自攜帶 AD 遺 傳風險變異患者的 iPSC，藉此深入研究病理生理學。這些 iPSC 衍生的神經元，亦可用作評估候選藥物的功效和安全性。 此外，我們成功研發出一種新型全腦基因編輯技術，在小鼠模 型中證明可以改善 AD 的病理症狀，有潛力發展成爲 AD 的新 型長效治療手段。該技術獲得了中國神經科學學會 2021 年度 「神經科學重大進展」殊榮，在第四十九屆日內瓦國際發明展 上取得金獎，並成為首批獲創新科技署「產學研 1+ 計劃」 （RAISe+）資助的項目之一。 First Members’ Meeting held on 12 Dec 2018 首次會員大會於2018年12月12日召開 Fourth Annual Symposium held on 09 Jan 2024 第四屆年度研討會於2024年1月9日舉行 7
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