Short Biography of Project Coordinator 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. Project Summary Mission: Address the urgent need for new and innovative therapies that can treat age-related neurodegenerative disorders. Goal: Use human induced pluripotent stem cell (iPSC) and genome-editing technologies to investigate the pathological mechanism of Alzheimer's disease (AD). Objectives: • Establish a human iPSC core for in vitro modeling and functional studies. • Examine how genetic variants contribute to AD. • Perform drug screening and preclinical drug development using iPSC-derived platforms and established animal models. Deliverables: • Identify and elucidate the molecular pathways that contribute to AD pathogenesis. • Use genome-editing technologies to introduce or correct specific mutations in iPSCs. • Identify potential molecular targets for therapeutic intervention of AD. 項目概要 使命： 聚焦老齡相關的神經退行性疾病，探尋創新療法以應 對迫切需求。 目標： 利用人源誘導多能幹細胞（iPSC）和基因編輯技 術，研究阿茲海默症 (AD) 的病理機制。 目的： • 建立人源 iPSC 核心平臺，進行體外建模和功能研究。 • 研究遺傳變異如何導致 AD。 • 使用 iPSC 衍生平臺和動物模型進行藥物篩選和臨床 前藥物開發。 成果： • 辨識及闡明 AD 致病機制的分子途徑。 • 利用基因組編輯技術引入或修正 iPSC 中的特定突變。 • 辨識治療 AD 的潛在分子靶點。 項目統籌人簡介 葉玉如教授於美國哈佛大學獲藥理學博士學位，其後在紐約 Regeneron 製藥公司擔任高級科學家。她於 1993 年起受聘於 科大，現任晨興生命科學教授及生命科學部講座教授。葉教授 是國際知名學者，在解析大腦功能的複雜機制以及研發神經退 化性疾病新藥方面有重大貢獻，發表了超過 330 篇論文和綜 述，擁有 70 多項科技發明專利。葉教授還當選為多間學術機 構的院士，包括中國科學院、美國國家科學院、美國人文與 科學院，並獲頒多個國內外重要學術獎項，包括國家自然科學 獎、歐萊雅聯合國教科文組織「世界傑出女科學家成就獎」。 The level of amyloid plaque deposition (white shades in area encircled by green dotted lines), a pathological hallmark of AD, is high in the brain of AD mouse (left) and decreases after the application of the genome editing therapy throughout the brain (right). AD小鼠大腦（左圖）中有大量澱粉樣斑塊沉積，這是AD最主要的 病理性標誌物（綠色虛線區域中的白色陰影）。經過基因編輯治療 後，白色陰影明顯下降（右圖）。 6 A Stem Cell Approach to Dissect the Molecular Basis of Neurodegenerative Diseases 以幹細胞方法解析神經退行性疾病的分子基礎 T13-605/18-W Project Coordinator Professor Nancy IP The Hong Kong University of Science and Technology 項目統籌人 葉玉如教授 香港科技大學 THEME 主題 1 | Understanding Diseases and Disease Prevention 剖析疾病及疾病預防 Participating Institutions City University of Hong Kong The Chinese University of Hong Kong The University of Hong Kong 參與院校 香港城市大學 香港中文大學 香港大學
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