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治疗老年痴呆、逆转衰老:RNA疗法带来新希望 ?
Hu Xiu· 2025-09-22 03:16
本文来自微信公众号:返朴 (ID:fanpu2019),受访者:付向东,访谈人:叶水送,原文标题:《专访著名生物学家付向东:治疗老年痴呆、逆转衰 老,RNA疗法为人类带来新的希望》,题图来自:AI生成 RNA,作为细胞信号传导的重要使者,在许多疾病中扮演着重要的角色,无论是神经退行性疾病还是衰老,都有研究者在开发相应的RNA疗法。 西湖大学讲席教授付向东自1992年建实验室时起,就一直在做RNA方面的基础研究和转化应用。他于2010年入选美国科学促进会成员,2016年获Ray Wu Society终身成就奖,是国际知名的RNA研究专家。在近期我们对付向东教授的一次专访中,他表示:"RNA既是一个调节因子,也是一个药物靶标,同时 也可以自身作为药物。" 近年,付向东教授将工作重点转移至开发各种RNA疗法上,他认为对于复杂疾病的治疗近年取得的真正进展还是比较小,但随着RNA领域一些基础理论 取得突破,可以带来全新的希望。 问:首先请您介绍下什么是RNA?它跟DNA、蛋白质之间的关系是怎样的? 付向东:一提到这个话题,我们大概会想到中心法则:从DNA到RNA再到蛋白质,RNA发挥了一种承上启下的功能,只要有细胞都符 ...
一项突破性技术延长寿命20%,股价暴涨787%
Core Viewpoint - The article discusses the promising clinical data from Klotho Neurosciences (KLTO) regarding the secreted Klotho protein (s-KL), which has shown potential in extending lifespan and combating age-related diseases [2][10]. Group 1: Clinical Findings - KLTO announced that the delivery of s-KL via AAV9 vector effectively increased serum s-KL levels in mice, leading to a 20% increase in lifespan [2][7]. - The expression of the Klotho gene can reduce age-related degeneration across multiple organs, promoting healthy aging [2][10]. Group 2: Market Impact - Following the positive clinical data, KLTO's stock price surged by 787.8% in the U.S. market, with trading volume exceeding 1 billion shares [3]. - The company has established a strong intellectual property position, securing global exclusive licenses for s-KL from the University of Barcelona and ICREA, along with patents in the U.S., Europe, and China [10]. Group 3: Product Pipeline - KLTO's product pipeline includes KLTO-202 for ALS and MS (expected IND submission in Q4 2025), KLTO-101 for Alzheimer's, Parkinson's, and Huntington's diseases (preclinical), and KLTO-301 for atherosclerosis and kidney diseases (preclinical) [11]. - The focus on neurodegenerative diseases represents a significant market opportunity with unmet needs, and the approach targets the fundamental processes of aging rather than just symptoms [11].