撰文丨王聪 编辑丨王多鱼 排版丨水成文 用于监测和操控神经元活动的基因靶向方法在灵长类大脑研究中并未得到广泛应用，这主要是由于缺乏 细胞类型特异性的靶向方法。 | | | 2025 年 7 月 10 日，中国科学院脑智卓越创新中心/上海脑科学与类脑研究中心 | | 刘真 | | --- | --- | --- | --- | --- | | 研究组、脑智卓越中心 | | 孙怡迪 | 研究组、临港实验室 李昊 | 研究组 | | 合作 | （ | 刘真研究组博士 | 研究生 | 李灵 | | 、 | 陆登宇 | 、研究助理 | 傅吉强 、临港实验室/上海科技大学研究 | | | 生 | 黄奕铭 | 为该论文并列第一作者 | ） | 在国际顶尖学术 | | 期刊 | Cell | 发表了题为： | Identification and Application of Cell Type | | | Specific Enhancers for the Macaque Brain | | | 的研究论文。 | | 该研究率先建立了灵长类大脑神经细胞类型特异性标记、神经活性调控及观测的工具集，为深入理 解灵长类脑结构、 ...

Core Insights - REGENXBIO Inc. announced preclinical results showing that a microdystrophin gene therapy construct with the C-terminal (CT) domain provides improved functional benefits for patients with Duchenne Muscular Dystrophy compared to a construct without the CT domain [1][4][5] - RGX-202 is the only investigational microdystrophin gene therapy candidate that includes the CT domain, making it closest to naturally occurring dystrophin [2][8] Group 1: Research Findings - The preclinical study published in Molecular Therapy Methods and Clinical Development demonstrated that the microdystrophin with the CT domain was maintained at higher levels in transduced muscles and effectively recruited the dystrophin-associated protein complex to the muscle membrane [4][5] - The incorporation of the CT domain enhances the microdystrophin design, allowing for higher accumulation levels in muscle and potentially improving functional benefits [5][7] Group 2: Clinical Trial Insights - Interim results from the Phase I/II AFFINITY DUCHENNE trial indicated that RGX-202 showed consistent evidence of positively changing the disease trajectory in patients with Duchenne and had a favorable safety profile [5][6] - REGENXBIO is currently enrolling participants in the pivotal portion of the Phase I/II/III AFFINITY DUCHENNE trial and plans to submit a Biologics License Application (BLA) via the accelerated approval pathway in mid-2026 [6][9] Group 3: Company Overview - REGENXBIO is a biotechnology company focused on gene therapy, with a late-stage pipeline that includes RGX-202 for Duchenne, among other treatments for rare diseases [11] - The company has pioneered AAV gene therapy since its founding in 2009 and has treated thousands of patients with its AAV platform [11]

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PBFT02 Development and Preclinical Results - PBFT02 is an AAV gene therapy designed to deliver functional PGRN to the brain for the treatment of FTD-GRN [13] - In Grn-/- mice, AAV.hGRN vector ICV administration improved lysosomal function, reduced lipofuscin fluorescence in the thalamus, and reduced brain hexosaminidase activity [21, 23] - AAV1 was selected as the vector serotype due to superior hPGRN levels in CSF compared to AAV5 and AAVhu68 in NHPs [28, 29] - In Grn-/- mice, PBFT02 reduced lipofuscin deposition and neuroinflammation in the brain after intra-CSF delivery [34, 37] - ICM administration of PBFT02 enables PGRN delivery throughout the CNS [40] - In NHPs, PBFT02 dose-dependently increased PGRN in CSF up to day 14 [46, 48] - In NHPs, PBFT02 at Dose 1 resulted in approximately 10e4 GC/ug DNA throughout the brain [43] Clinical Trial (upliFT-D) and Safety - The upliFT-D trial is a global Phase 1/2 multi-center, open-label, dose-escalation study with PBFT02 [52, 55] - FTD-GRN Cohort 1 (n = 5) dosing is complete [56] - All four Cohort 1 participants who received a revised immunosuppression regimen had no SAEs or significant immune responses [57] - Cohort 1 interim data shows PBFT02 administration leads to robust and sustained increases in CSF PGRN [58]

在基因编辑研究领域， Cre-loxP 系统凭借其精准高效的特性，已成为众多实验室的"标配"。为帮助大家解 决Cre工具鼠应用中的实际问题，我们于数月前开设了两期 Cre系列课程 ，通过 系统讲解+在线答疑 的方 式，获得了众多科研工作者的积极反馈。 这段时间我们注意到，针对Cre-loxP结合AAV的应用问题持续受到大家关注： 为此，工具鼠系列课程将再次返场！ 6月12日（周四）晚7点 ，由 赛业生物高级产品经理刘凡瑞、赛业 生物AAV基因治疗项目经理熊泽浩 共同带来 「Cre-loxP系统结合AAV的应用」 主题课程。 本次课程围绕 粉丝朋友们提出的问题展开 ，深入探索Cre-loxP系统与AAV技术的 核心原理 ，解析技术 联用中的 关键优化策略 ，并通过典型应用案例揭示 高分文章背后的实验设计智慧 。扫码提交感兴趣的 问题，讲师将在直播中针对性解答，成功报名课程还可抽取 SKG颈部按摩仪（价值500元） 等好礼！ 以下文章来源于赛业生物订阅号 ，作者小赛 赛业生物订阅号 . 分享生命科学领域的前沿资讯、解读行业动态、讲解实用的学科知识、实验方法和技巧。 为什么有的文献选择通过AAV递送Cre酶，而非传 ...

在基因编辑研究领域， Cre-loxP 系统凭借其精准高效的特性，已成为众多实验室的"标配"。为帮助大家解 决Cre工具鼠应用中的实际问题，我们于数月前开设了两期 Cre系列课程 ，通过 系统讲解+在线答疑 的方 式，获得了众多科研工作者的积极反馈。 以下文章来源于赛业生物订阅号 ，作者小赛 赛业生物订阅号 . 分享生命科学领域的前沿资讯、解读行业动态、讲解实用的学科知识、实验方法和技巧。 这段时间我们注意到，针对Cre-loxP结合AAV的应用问题持续受到大家关注： 为此，工具鼠系列课程将再次返场！ 6月12日（周四）晚7点 ，由 赛业生物高级产品经理刘凡瑞、赛业 生物AAV基因治疗项目经理熊泽浩 共同带来 「Cre-loxP系统结合AAV的应用」 主题课程。 本次课程围绕 粉丝朋友们提出的问题展开 ，深入探索Cre-loxP系统与AAV技术的 核心原理 ，解析技术 联用中的 关键优化策略 ，并通过典型应用案例揭示 高分文章背后的实验设计智慧 。扫码提交感兴趣的 问题，讲师将在直播中针对性解答，成功报名课程还可抽取 SKG颈部按摩仪（价值500元） 等好礼！ 为什么有的文献选择通过AAV递送Cre酶，而非传 ...

撰文丨王聪 编辑丨王多鱼 排版丨水成文 阿尔茨海默病 （AD） 是一种破坏性的神经退行性疾病，其特征在于认知功能的逐渐衰退，包括学习、记忆和语言处理能力。随着全球老龄化的加剧 ，迫切需要 能够减缓或阻止阿尔茨海默病进展的疗法。 阿尔茨海默病 （AD） 是一种破坏性的神经退行性疾病，其特征为突触逐渐丧失和认知能力下降。增强内在神经保护通路的基因疗法，为减缓神经退行性病变和 防止认知能力进一步下降提供了一种很有前景的策略。 小窝蛋白-1 （ Caveolin-1， Cav-1） 是一种膜脂支架蛋白，它在质膜微结构域中调节多种促生长和促存活的信号通路。此前，该团队发现，将 AAV9- Synapsin 促进的 Cav-1 （ SynCav1 ） 递送至无症状期阿尔茨海默病小鼠模型体内，可保持其认知功能以及与海马长时程增强相关的神经营养信号传导。然 而，SynCav1 在阿尔茨海默病出现症状阶段的治疗潜力尚未得到测试。 因此，在这项新研究种，研究团队在两种具有淀粉样蛋白病理特征的阿尔茨海默病临床前模型 （PSAPP 小鼠和 APPKI 小鼠） 中，对症状出现时给予海马体 SynCav1 递送的效果进行了探究。 细胞外 ...

Regenxbio (RGNX) Q1 2025 Earnings Call May 12, 2025 04:30 PM ET Company Participants Patrick Christmas II - Chief Legal OfficerCurran Simpson - Board Member, President & CEOSteve Pakola - Chief Medical OfficerMitchell Chan - CFOMani Foroohar - Senior Managing DirectorGena Wang - MD - Biotech Equity ResearchTejas Wein - Associate Director - Biotechnology Equity ResearchAnnabel Samimy - Managing DirectorShelby Hill - Biotech Equity Research AssociateSean McCutcheon - Vice President - Biotechnology Equity Rese ...

DALLAS, May 08, 2025 (GLOBE NEWSWIRE) -- Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a clinical-stage biotechnology company focused on advancing adeno-associated virus (AAV)-based gene therapies for severe monogenic diseases of the central nervous system (CNS), today announced that it will report its financial results for the first quarter ended March 31, 2025, and host a corporate update conference call and webcast on Thursday, May 15, 2025, at 8:30 AM Eastern Time. Conference Call DetailsThursday, May 15, ...

Research highlights AAV manufacturing optimization via Lexeo’s Sf9-baculovirus processNEW YORK, May 01, 2025 (GLOBE NEWSWIRE) -- Lexeo Therapeutics, Inc. (Nasdaq: LXEO), a clinical stage genetic medicine company dedicated to pioneering novel treatments for cardiovascular diseases, announced today that new data supporting its AAV manufacturing approach will be presented at the 28th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) taking place May 13-17, 2025 in New Orleans, LA. “The data ...