基因编辑
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我国自主研发的“榫卯”基因编辑系统问世
Ke Ji Ri Bao· 2025-12-08 10:03
记者12月2日从北京大学现代农业研究院获悉,该院张华伟研究员团队和中国科学院遗传与发育生物学 研究所李家洋院士团队合作研发出"榫卯"基因编辑系统。相关研究日前发表于国际期刊《分子植物》。 在作物育种领域,实现精准、无瘢痕的DNA片段插入与替换,是突破品种改良瓶颈和保障国家粮食安 全的核心技术。该研究成功开发出一种名为"榫卯连接系统"(MT)的新型基因编辑系统,可实现靶向 DNA高效插入与替换,有望为大片段基因编辑开辟新方法。张华伟表示,该系统的核心设计灵感源于 中国古建筑传统木工中的"榫卯"结构,即通过构建相互匹配的"榫头"与"卯眼",实现DNA片段的精准整 合。 为验证该系统的编辑性能研究,研究团队在水稻多个基因位点开展实验。张华伟表示,该系统提供了一 种"结构适配、定向整合"的编辑新思路。最高59.47%的精准编辑效率和无瘢痕特性及稳定遗传表现,使 其在水稻功能基因研究和优良性状快速导入等领域具有广阔应用前景。 "该系统具有特异性强、功能全面、适应性广和发展潜力大的优势,为基因组精准编辑提供了崭新的有 力工具。"李家洋表示,未来他们将对该系统的供体递送效率和供体制备等技术进行探索和优化,旨在 实现基因组D ...
【科技日报】我国自主研发的“榫卯”基因编辑系统问世
Ke Ji Ri Bao· 2025-12-03 03:20
"该系统具有特异性强、功能全面、适应性广和发展潜力大的优势,为基因组精准编辑提供了崭新 的有力工具。"李家洋表示,未来他们将对该系统的供体递送效率和供体制备等技术进行探索和优化, 旨在实现基因组DNA大片段精准插入和编辑,拓展该系统更多应用场景。 中国科学院院士钱前表示,该系统有助于在育种实践中赋能主粮、经济作物的性状改良,加速高 产、抗逆、优质新品种培育,助力破解"抗逆必减产"等瓶颈。该系统的自主知识产权属性,将推动我国 精准编辑领域从"技术引进"向"自主创新"转型,增强在全球种业竞争中的话语权。 北京大学现代农业研究院张华伟研究员团队和中国科学院遗传与发育生物学研究所李家洋院士团队 合作研发出"榫卯"基因编辑系统。相关研究日前发表于国际期刊《分子植物》。 在作物育种领域,实现精准、无瘢痕的DNA片段插入与替换,是突破品种改良瓶颈和保障国家粮 食安全的核心技术。该研究成功开发出一种名为"榫卯连接系统"(MT)的新型基因编辑系统,可实现 靶向DNA高效插入与替换,有望为大片段基因编辑开辟新方法。张华伟表示,该系统的核心设计灵感 源于中国古建筑传统木工中的"榫卯"结构,即通过构建相互匹配的"榫头"与"卯眼",实 ...
我国自主研发“榫卯”基因编辑系统问世
Ke Ji Ri Bao· 2025-12-02 13:50
记者2日从北京大学现代农业研究院获悉,该院张华伟研究员团队和中国科学院遗传与发育生物学研究 所李家洋院士团队合作研发出"榫卯"基因编辑系统。相关研究日前发表于国际期刊《分子植物》。 在作物育种领域,实现精准、无瘢痕的DNA片段插入与替换,是突破品种改良瓶颈和保障国家粮食安 全的核心技术。该研究成功开发出一种名为"榫卯连接系统"(MT)的新型基因编辑系统,可实现靶向 DNA高效插入与替换,有望为大片段基因编辑开辟新方法。 张华伟表示,该系统的核心设计灵感源于中国古建筑传统木工中的"榫卯结构",即通过构建相互匹配 的"榫头"与"卯眼",实现DNA片段的精准整合。 "该系统具有特异性强、功能全面、适应性广和发展潜力大的优势,为基因组精准编辑提供了崭新的有 力工具。"李家洋认为,未来,将对该系统的供体递送效率和供体制备等技术进行探索和优化,旨在实 现基因组DNA大片段精准插入和编辑,拓展该系统在更多作物中的应用场景。 中国科学院院士钱前表示,该系统有助于在育种实践中,赋能主粮、经济作物的性状改良,加速高产、 抗逆、优质新品种培育,助力破解"抗逆必减产"等瓶颈。更重要的是,该系统的自主知识产权属性,将 推动我国精准编辑 ...
类器官研究揭示大脑天生预置“操作系统”
Huan Qiu Wang Zi Xun· 2025-12-01 06:23
了解类器官能够自发地产生活体大脑的基本神经结构,为更好地理解人类神经发育、神经系统疾病以及 环境毒素对大脑的影响开辟了多种可能性。这些模型具备捕捉复杂神经动力学的基础能力,而这些动力 学很可能与某些发病机制密切相关。未来,团队将在临床前层面探索开发新的化合物、药物疗法或基因 编辑工具。 【总编辑圈点】 种子还深埋于土壤中时,就具备破土而出的"潜力"。这不是种子后天"学会"的,而是生命进化写入基因 的底层程序。同理,人脑在发育之初,神经元就可以自发地发出复杂的电信号,与世界进行初步互动, 为后续处理更复杂的外部感官信号做准备,这也是人类基因的底层编码决定的。也就是说,新生婴儿的 大脑并非白纸一张,而是带着基因为人脑预设的"出厂设置"。这一发现,有助于科学家更好地理解人脑 发育机制,并有望为利用基因编辑等手段治疗先天性神经疾病提供启发。 在这项研究中,团队引导干细胞发育成脑组织,然后使用类似计算机芯片的专用微电极阵列来记录其电 活动。在观察脑组织从干细胞自我组装成能够处理感官信息,并最终产生语言和意识思维的复杂结构的 过程中发现,在发育的最初几个月内,远在人脑能够接收和处理视觉、听觉等复杂外部感官信息之前, 其内 ...
登上Cell子刊封面:复旦大学开发出最强Fanzor基因编辑系统,单个AAV递送,实现高效体内基因编辑
生物世界· 2025-11-30 08:00
Core Viewpoint - The article discusses the development and potential of the Fanzor system, a compact RNA-guided DNA nuclease, for precise genome editing in eukaryotic cells, highlighting its advantages over traditional Cas9 systems and its application in disease treatment [3][9]. Group 1: Fanzor System Overview - Fanzor is a newly discovered CRISPR-like system that allows for precise genome editing in eukaryotic organisms, with a compact size of 400-700 amino acids, making it suitable for delivery via a single AAV vector [3][9]. - The traditional AAV delivery system has a maximum packaging capacity of 4.7 kb, which is insufficient for larger Cas proteins like SpCas9 and AsCas12a, necessitating the use of dual AAV vectors [3]. Group 2: Research Developments - A study published by a team from Fudan University introduced an engineered hypercompact Fanzor-ωRNA system, which demonstrated enhanced genome editing activity, achieving efficiency improvements of 6-129 times compared to previous versions [4][11]. - The engineered SpuFz1 V4 system, derived from soil fungus, showed strong genome editing capabilities in human cells, marking it as the most active member of the Fanzor family [9][11]. Group 3: Applications and Potential - The compact structure of SpuFz1 V4 allows for effective in vivo delivery using a single AAV, enabling robust genome editing in animal models, such as efficient editing of the Nfkb1 gene in mouse retinas [10][11]. - The research indicates that the Fanzor platform can be developed into base editors, facilitating efficient single-base editing, which has significant implications for genetic research and therapeutic applications [5][9].
研发人员降超三成,“基因编辑第一股”今日申购
2 1 Shi Ji Jing Ji Bao Dao· 2025-11-29 11:49
Core Viewpoint - The company BaiO Saite (688796.SH) is set to launch an IPO on the Sci-Tech Innovation Board, focusing on gene editing and related services, following its previous listing on the Hong Kong Stock Exchange in 2022 as the "first gene editing stock" [3]. Group 1: Company Overview - BaiO Saite operates four technical platforms: gene-edited model animal preparation, innovative model animal breeding and supply, preclinical pharmacology and efficacy evaluation, and antibody drug discovery [3]. - The company has developed a stable and efficient gene editing platform, completing approximately 5,300 customized gene editing projects and creating over 4,300 gene-edited animal and cell line models by June 30, 2025 [7]. Group 2: Financial Information - The IPO price is set at 26.68 yuan per share, with an institutional offering price of 27.88 yuan, resulting in a market capitalization of 10.66 billion yuan [4]. - The company’s earnings per share (EPS) is projected at a price-to-earnings (P/E) ratio of 519.12, significantly higher than the industry average P/E of 39.31 [4]. Group 3: Fundraising and Investment Plans - The company plans to allocate raised funds as follows: 4.54 billion yuan (38.28%) for early drug development services, 3.16 billion yuan (26.70%) for antibody drug research and evaluation, 1.65 billion yuan (13.92%) for preclinical research, and 2.50 billion yuan (21.10%) for working capital [7]. Group 4: Business Performance - The company has shifted to a direct sales model, with the mode animal sales segment accounting for 44.2% of total revenue in the first half of the year, generating 274 million yuan [9]. - Gilead Sciences, Inc. is the largest client, contributing 88 million yuan, or 14.25% of total revenue in the same period [9]. Group 5: Workforce Changes - The number of R&D personnel decreased from 627 at the end of 2022 to 403 by mid-2025, a reduction of over 30%, attributed to the completion of a large-scale R&D phase [8].
研发人员降超三成 “基因编辑第一股”今日申购丨打新早知道
2 1 Shi Ji Jing Ji Bao Dao· 2025-11-28 01:08
Core Viewpoint - The company, BaiAo SaiTu, is launching an IPO on the Sci-Tech Innovation Board, focusing on gene editing and related services, despite a significant reduction in R&D personnel. Group 1: Company Overview - BaiAo SaiTu is recognized as the "first stock in gene editing" and was listed on the Hong Kong Stock Exchange in 2022 [2] - The company operates four technical platforms: gene-edited model animals, innovative model animal breeding and supply, preclinical pharmacology and efficacy evaluation, and antibody drug discovery [2] - As of June 30, 2025, BaiAo SaiTu has completed approximately 5,300 customized gene editing projects and developed over 4,300 gene-edited animal and cell line models [2] Group 2: R&D and Personnel Changes - The number of R&D personnel at BaiAo SaiTu decreased from 627 at the end of 2022 to 403 by mid-2025, a reduction of over 30% [3] - The company stated that the large-scale R&D phase concluded in Q3 2023, leading to a significant drop in internal R&D projects and subsequent personnel optimization [3] Group 3: Business Performance - BaiAo SaiTu's business segments include preclinical pharmacology and efficacy evaluation, model animal sales, antibody development, and gene editing services [3] - Model animal sales have become increasingly significant, generating revenue of 274 million yuan in the first half of the year, accounting for 44.2% of total revenue [3] - The largest client, Gilead Sciences, Inc., contributed 88 million yuan in revenue during the first half of the year, representing 14.25% of total revenue [3]
研发人员降超三成,“基因编辑第一股”今日申购丨打新早知道
2 1 Shi Ji Jing Ji Bao Dao· 2025-11-28 00:53
Core Viewpoint - The company, Bai'ao Saitu, is set to launch an IPO on the Sci-Tech Innovation Board, focusing on gene editing and related services, after previously being listed in Hong Kong as the "first gene editing stock" [1]. Group 1: Company Overview - Bai'ao Saitu operates four technical platforms: gene-edited model animal preparation, innovative model animal breeding and supply, preclinical pharmacology and efficacy evaluation, and antibody drug discovery [1]. - The company has completed approximately 5,300 customized gene editing projects and developed over 4,300 types of gene-edited animals and cell line models by June 30, 2025 [6]. Group 2: Financial Aspects - The company plans to raise funds for various projects, including 4.54 billion for early drug development services (38.28%), 3.16 billion for antibody drug research and evaluation (26.70%), 1.65 billion for preclinical research (13.92%), and 2.50 billion for working capital (21.10%) [5]. - In the first half of this year, the revenue from model animal sales reached 274 million, accounting for 44.2% of total revenue [7]. Group 3: Human Resources - The number of R&D personnel decreased from 627 at the end of 2022 to 403 by June 30, 2025, a reduction of over 30% due to the completion of a large-scale R&D phase [6]. Group 4: Client Base - The primary clients of Bai'ao Saitu include pharmaceutical R&D, biotechnology companies, and research institutions, with Gilead Sciences, Inc. being the largest client, contributing 88 million in revenue, which is 14.25% of total revenue for the period [7].
双鹭药业(002038) - 002038双鹭药业投资者关系管理信息20251127
2025-11-27 09:36
Group 1: Impact of National Drug Procurement - The company has been significantly affected by the 8th and 9th batches of national drug procurement, with major products entering procurement and experiencing price reductions of up to 70% [2][3] - Temozolomide capsules saw a price drop of 62% during the 4th batch procurement, yet sales revenue increased by 109% despite the price cut [5][6] - The average price of several biological products decreased by approximately 15% due to regional alliance procurement [3] Group 2: Business Model and Product Structure - The company operates as a biopharmaceutical enterprise, covering drug research, production, and marketing, with a focus on expanding into the health sector [4] - The product structure has shifted from a high reliance on major products to a more diversified portfolio, enhancing risk resistance [6][11] - Oral solid preparations now account for over 50% of the product mix, reflecting a significant change from previous years when injectables dominated [12] Group 3: New Product Launches and Profit Contribution - The contribution of new and upcoming products to profits depends on their procurement success and market expansion of non-procurement products [6] - The company has a rich pipeline of new products, indicating a potential shift towards a multi-product profit model [6] Group 4: Production Capacity and Operations - The company has eight major production bases, with some operating at full capacity and others reserved for future product launches [7] - Plans are in place to transfer some production to lower-cost locations to participate in larger-scale procurement [7] Group 5: Sales Performance and Key Products - The top ten products by sales revenue in the first three quarters of 2025 include: - Fuzhifu: 59.6 million CNY - Temozolomide capsules: 55.2 million CNY - Oseltamivir phosphate capsules: 52.7 million CNY [8] - Changes in invoicing models have affected reported sales revenue, complicating the assessment of actual sales performance [8] Group 6: International Investments and Collaborations - The company holds a 30% stake in Diapin Therapeutics, focusing on innovative drug development for diabetes and cardiovascular diseases [9] - ATGC Company is developing a unique therapeutic antibody platform using genetically modified rabbits, with significant progress reported [10] Group 7: Market Position and Stock Performance - The company has faced a prolonged adjustment period due to policy changes and competitive pressures, with market capitalization remaining below 10 billion CNY for years [13] - Management emphasizes the importance of developing competitive products to improve performance and shareholder returns [13] Group 8: Recent Investments - The company invested 10 million CNY in Changfeng Pharmaceutical, which is now publicly listed, holding a 3.68% stake valued at nearly 500 million CNY [15] - The investment in Xuan Bamboo Biotechnology also resulted in a public listing, with a 0.34% stake valued at approximately 206 million CNY [15]
基因编辑民猪培育成功
Ke Ji Ri Bao· 2025-11-26 00:33
Core Viewpoint - The research teams from Heilongjiang Academy of Agricultural Sciences and Sun Yat-sen University have successfully developed the world's first single-gene and multi-gene edited Min pigs, marking a significant advancement in genetic editing for livestock breeding [1] Group 1: Single-Gene Editing Achievements - The teams achieved single-gene editing of the IGF2 gene, resulting in Min pigs with improved meat quality [1] - They also edited the BMP15 gene, leading to Min pigs with enhanced reproductive capabilities [1] Group 2: Multi-Gene Editing Achievements - The teams combined the BMP15, MSTN, and CD163 genes to create three-gene edited Min pigs that exhibit increased production, high reproductive capacity, and disease resistance [1] - Another combination of the TYR, SGK3, and GHRHR genes resulted in three-gene edited Min pigs characterized by hairlessness, albinism, and dwarfism [1] Group 3: Broader Implications and Future Research - The research also included the Five-Finger Mountain pig, resulting in growth-related PROP1 gene edited pigs and high reproductive capacity FecB gene edited pigs [1] - The findings provide new pathways for high-quality Min pig production and open new avenues for the breeding of medical model pigs and pet pigs, indicating significant theoretical and practical implications [1] - The teams plan to enhance trait evaluation of gene-edited pigs and continue research on other gene-edited pigs, contributing to the protection of local pig resources and the development of the life and health industry in China [1]