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速递|Nature头条:贺建奎前妻创办新公司,公开表示“CRISPR婴儿”时代或已来临
GLP1减重宝典· 2025-11-18 05:33
Core Insights - The article discusses the controversial topic of "CRISPR babies" and highlights the emergence of Cathy Tie, a biotech entrepreneur, who advocates for the consideration of gene editing in human embryos to prevent genetic diseases [2][8]. Company and Industry Summary - Cathy Tie, a Canadian entrepreneur, has founded multiple biotech companies over the past 11 years, including Locke Bio in 2020, which focuses on telemedicine and medical data mining services [4]. - Tie's latest venture, Manhattan Genomics, co-founded with Eriona Hysolli, aims to explore human embryo gene editing to prevent hereditary diseases [7][8]. - On the same day, another company, Preventive, announced nearly $30 million in funding to explore human embryo gene editing, indicating a growing interest in this controversial field [8]. - The scientific community generally views the commercialization of embryo gene editing as premature, citing significant safety and ethical concerns compared to existing CRISPR therapies approved for treating specific genetic disorders [8][9]. - CRISPR technology, awarded the Nobel Prize in Chemistry in 2020, is recognized as a revolutionary biotechnological advancement, yet the push for embryo editing has faced global condemnation and regulatory restrictions [9]. - Recent advancements in non-reproductive cell gene editing have been significant, with the first CRISPR-Cas9 therapy approved for hereditary blood disorders in 2023, showcasing the potential of gene editing while emphasizing the ethical divide between editing somatic and germline cells [9].
Nature头条:贺建奎前妻成立公司,宣称是时候考虑“CRISPR baby”了
生物世界· 2025-11-04 00:00
Core Viewpoint - The article discusses the controversial emergence of companies focusing on gene editing technologies, particularly in relation to human embryos, highlighting the recent activities of Cathy Tie and her company Manhattan Genomics, as well as the ethical and safety concerns surrounding such advancements [2][10][11]. Company Overview - Cathy Tie, a Canadian entrepreneur, has founded multiple biotechnology companies over the past 11 years, including her latest venture, Manhattan Genomics, which aims to edit human embryo genes to prevent genetic diseases [4][8]. - Manhattan Genomics was co-founded by Cathy Tie and Eriona Hysolli, who previously worked at Colossal Biosciences, a company focused on resurrecting extinct species through gene editing [9][10]. Industry Developments - On October 30, 2025, Manhattan Genomics announced key employee hires, including a bioethicist and experts in non-human primate reproductive biology, as part of their preparation for potential CRISPR baby projects [10]. - Preventive, another company exploring gene editing in human embryos, recently secured nearly $30 million in funding, indicating a growing interest and investment in this controversial area [10]. Ethical and Safety Concerns - The scientific community largely agrees that commercializing gene editing in human embryos is premature, given the significant safety risks and ethical dilemmas compared to existing CRISPR therapies approved for conditions like sickle cell disease and β-thalassemia [11][12]. - Editing embryos poses unique challenges, as changes would affect nearly every cell in the body and could be passed on to future generations, raising unpredictable consequences [13]. Technological Context - CRISPR technology, recognized as a groundbreaking biotechnological advancement, received the Nobel Prize in Chemistry in 2020, yet its application in human embryos remains heavily restricted in many countries, including the U.S. [12]. - Recent advancements in gene editing for non-reproductive cells have progressed rapidly, with the first CRISPR-Cas9 therapy approved in 2023 for genetic blood disorders, showcasing the potential of gene editing outside of embryo manipulation [12].
陈天桥宣布10亿美元算力支持发现式智能
Feng Huang Wang· 2025-10-29 07:04
Core Insights - The first "AI-Driven Scientific Symposium" was held in San Francisco, featuring Nobel laureates and industry leaders discussing how AI can drive scientific discovery [1][2] - Chen Tianqiao announced a $1 billion investment in computational power to support global scientists in "discovery-driven intelligence" research [1] - The symposium highlighted the importance of AI's role in constructing verifiable world models and enhancing human capabilities rather than replacing them [1] Group 1: AI in Scientific Research - Chen Tianqiao emphasized the need for "discovery-driven intelligence" to possess five key capabilities: neural dynamic structure, long-term memory, causal reasoning mechanisms, world models, and metacognitive systems [1] - Omar Yaghi showcased AI's application in materials science, demonstrating a portable device that extracts water from the atmosphere in low humidity conditions using ChatGPT for molecular optimization [1][2] - David Baker presented the RFDiffusion3 model, which enables reverse design of proteins, providing new pathways for research on diseases like Alzheimer's [2] Group 2: AI and Genetic Research - Jennifer Doudna discussed the integration of AI with CRISPR technology, highlighting its potential to enhance understanding of unknown gene functions and advance personalized gene therapy [2] - The symposium concluded with the "AI-Driven Science Prize," recognizing young scientists for their cutting-edge research, indicating a shift towards AI-driven paradigms across multiple disciplines [3] Group 3: Societal Implications of AI - John Hennessy reflected on the rapid adoption of AI, stressing the need for humans to retain key decision-making authority and ensure transparency in AI-generated content [2] - He warned about the potential depletion of global data for AI training in the coming years, noting that improvements in computational energy efficiency have not kept pace with growth [2]
背靠背两篇Nature:利用CRISPR基因编辑,增强CAR-T的癌症治疗效果
生物世界· 2025-09-29 04:05
Core Insights - CAR-T cell therapy represents a significant breakthrough in cancer treatment, showing potential beyond blood cancers to solid tumors and autoimmune diseases [3][7] - Despite its promise, CAR-T cell therapy faces major challenges, including T cell adaptability, limited proliferation, and immune suppression in the tumor environment [3][8] - Recent studies published in Nature utilized CRISPR technology to enhance CAR-T cell efficacy by identifying and knocking out specific genes [3][10] Group 1: Research Findings - The research from the Austrian Academy of Sciences introduced the CELLFIE platform for CRISPR screening to enhance CAR-T cells across multiple clinical targets [8] - Key findings included the identification of the RHOG gene knockout as a powerful method to enhance CAR-T cell function, outperforming standard CAR-T cells in various models [9] - The study established a foundation for optimizing cell-based immunotherapies through the characterization of CRISPR-enhanced CAR-T cells [9] Group 2: Additional Research Insights - A separate study from Harvard Medical School focused on identifying modifiers of CAR-T cell function in multiple myeloma using in vivo CRISPR screens [10][11] - The research highlighted specific genes, such as RASA2 and SOCS1, that enhance T cell expansion, while CDKN1B was identified as a critical factor limiting CAR-T cell adaptability [11][12] - The findings emphasize the dynamic nature of gene disruption effects on CAR-T cells over time and in different environments, suggesting CDKN1B as a promising target for developing effective CAR-T therapies for multiple myeloma [12]
自带导航,主动敲门,CRISPR有了新型“快递车”
Ke Ji Ri Bao· 2025-09-23 08:52
Core Insights - CRISPR technology represents a revolutionary gene-editing method that offers unprecedented hope for treating genetic disorders, cancer, and rare diseases by precisely modifying disease-causing genes [1] - A significant breakthrough has been achieved by a team from Northwestern University, which has developed a new delivery system for CRISPR tools, enhancing efficiency and safety in gene therapy applications [1][3] Delivery Mechanisms - Current methods for delivering CRISPR into cells primarily rely on modified viruses and lipid nanoparticles (LNPs), each with distinct limitations [2] - Modified viruses are efficient at entering cells but pose safety risks due to immune responses and limited cargo capacity [2] - LNPs are safer but have low delivery efficiency, often getting trapped in cellular compartments, which hinders the effectiveness of gene tools [2] New Delivery System - The new system, termed "Lipid Nanoparticle Spherical Nucleic Acids" (LNP-SNA), features a special DNA shell that enhances visibility and acceptance by cells, significantly improving delivery efficiency [3] - This innovative delivery vehicle has been shown to enter cells over three times more efficiently than traditional lipid particles, with reduced toxicity and a threefold increase in successful gene editing probability [3] - The accuracy of gene repair has improved by over 60%, which is crucial for minimizing health risks associated with erroneous edits [3] Versatility and Future Applications - The LNP-SNA technology is modular, allowing for tailored delivery to specific cell types, such as liver, brain, or cancer cells, enhancing precision in treatment [4] - This new system has demonstrated excellent delivery results across various human cell types, including skin, immune, kidney, and bone marrow stem cells [4] - Seven drugs based on similar spherical nucleic acid technology are currently in human clinical trials, with some targeting cancer treatment [4] - The advancement in delivery mechanisms is critical for the future of gene editing therapies, potentially enabling the treatment of previously untreatable diseases [4]
晚报 | 8月18日主题前瞻
Xuan Gu Bao· 2025-08-17 15:13
Quantum Computing - Tsinghua and Peking University research teams have made significant progress in quantum computing architecture by implementing the AshN instruction set architecture, which supports direct programming of arbitrary two-qubit gates [1] - The quantum computing industry is transitioning from laboratory experiments to industrial applications, driven by technological breakthroughs, policy support, and market demand, with a projected market size of hundreds of billions by 2025-2030 [1] Refrigerants - The Ministry of Ecology and Environment has proposed a ban on the production of household refrigerators and freezers using hydrofluorocarbons (HFCs) as refrigerants starting January 1, 2026, marking a shift towards environmentally friendly refrigerants in China's home appliance industry [2] - The ban is expected to accelerate the transition to fourth-generation eco-friendly refrigerants, with natural refrigerants like R290 (propane) and R600a (isobutane) gaining traction, projected to reach 30% and 15% market share in commercial freezers by 2025, respectively [2] Gene Editing - A clinical trial has successfully demonstrated the use of gene editing technology to restore insulin production in a patient with type 1 diabetes, indicating a potential shift in diabetes management [3] - The rapid development of gene editing technologies, particularly CRISPR, is expanding the range of treatable conditions, with over 6,000 diseases now considered for gene therapy applications [3] Commercial Space - The commercial space industry in China is projected to grow from over 1 trillion yuan in 2020 to 2.3 trillion yuan by 2024, with a compound annual growth rate of approximately 22% [4] - Recent successful launches of low-orbit satellites indicate accelerated development in satellite internet infrastructure, with significant investment opportunities emerging in the commercial space sector [4] Artificial Intelligence in Biomanufacturing - The Ministry of Industry and Information Technology has released a notice to promote the integration of artificial intelligence in biomanufacturing, aiming to enhance the entire industry chain [5] Medical Advertising Regulation - New guidelines have been issued to clarify the definition of medical advertising and restrict its publication to licensed medical institutions, aiming to combat misleading advertisements [5] Virtual Power Plants - Guangzhou's plan for virtual power plants aims to achieve a capacity of 500,000 kilowatts by the end of 2027, with annual financial support of up to 10 million yuan for applications [6] Carbon Emission Financing - Guangdong has introduced a system to provide judicial support for carbon emission quota pledge financing, marking a first in the nation [7] Satellite Internet Development - The CCF and GuoShun Quantum have initiated a collaboration plan focusing on superconducting quantum computing, aiming to address key challenges in the field [7] Electronic Industry Trends - The global electronics market is expected to show divergence by 2025, with data center demand driving growth, while traditional consumer electronics face challenges due to inflation and lack of innovation [8]
基因编辑:改写生命密码的“神笔”(知与不知)
Core Viewpoint - Gene editing technology is revolutionizing the understanding of life by allowing precise modifications of genetic sequences, akin to using a "molecular scissors" to correct genetic errors [2][3][4] Group 1: Technology Development - The evolution of gene editing technology has progressed rapidly, particularly with the advent of CRISPR technology in 2012, which significantly lowered the technical barriers and costs associated with gene editing [4][5] - Newer techniques such as base editing and guided editing have emerged, providing more precise tools for genetic modifications, enhancing both basic scientific research and translational medicine [5][6] Group 2: Applications in Medicine - Gene editing technology offers innovative treatment methods for genetic diseases, such as using CRISPR to edit hematopoietic stem cells for conditions like thalassemia, leading to significant symptom relief in patients [6] - In cancer treatment, gene editing is utilized in CAR-T therapy, which modifies patients' immune cells to better target and combat cancer cells [6] Group 3: Applications in Agriculture and Bio-manufacturing - In agriculture, gene editing has been used to develop new rice varieties that are resistant to diseases and environmental stressors, contributing to global food security [6] - The technology also plays a crucial role in bio-manufacturing, enhancing the efficiency of biofuel production and reducing costs in the synthesis of scarce drugs [6] Group 4: Ethical Considerations - The advancement of gene editing technology raises significant ethical concerns, particularly regarding the editing of human germline cells, which could permanently alter the human gene pool and pose risks to future generations [7][8] - There is a need for strict ethical guidelines and international collaboration to ensure responsible use of gene editing technologies, prioritizing non-heritable somatic cell editing for therapeutic purposes [7][8] Group 5: Regulatory Framework - In July 2024, the Ministry of Science and Technology released ethical guidelines for human genome editing research, addressing the ethical challenges and promoting healthy development in this field [8]
基因编辑:改写生命密码的“神笔”(瞰前沿)
Ren Min Wang· 2025-07-04 21:56
Core Viewpoint - The recent successful application of personalized gene editing therapy on a 6-month-old infant marks a significant milestone in the treatment of genetic diseases, opening new avenues for patients lacking effective treatment options [1] Group 1: Gene Editing Technology Overview - Gene editing technology allows for precise deletion, insertion, or replacement of specific genes, akin to a "molecular scissors" that can correct and modify defective genes [2][4] - Unlike transgenic technology, which randomly integrates foreign genes into an organism's genome, gene editing modifies the organism's own genes without disrupting the overall structure [2][4] - The evolution of gene editing technology has progressed rapidly, particularly since the advent of CRISPR technology in 2012, which has simplified the process and significantly reduced costs [5][6] Group 2: Applications in Medicine - Gene editing technology is being applied in the treatment of genetic diseases, such as thalassemia, where CRISPR can edit a patient's hematopoietic stem cells to restore normal gene expression [7] - In cancer treatment, CAR-T therapy utilizes gene editing to enhance the immune cells' ability to combat cancer cells, demonstrating the technology's potential in oncology [7] - The technology also aids in modeling complex diseases in research, accelerating drug development by allowing scientists to observe disease progression in genetically edited organisms [7] Group 3: Applications in Agriculture and Bio-manufacturing - In agriculture, gene editing has led to the development of new rice varieties that are resistant to diseases and environmental stress, contributing to global food security [8] - In bio-manufacturing, gene editing enhances production efficiency and reduces costs, such as in the production of biofuels and scarce pharmaceuticals [8] Group 4: Ethical Considerations - The advancement of gene editing technology raises ethical concerns, particularly regarding the editing of human germline cells, which could permanently alter the human gene pool [10] - Ethical guidelines emphasize the importance of prioritizing non-heritable somatic cell editing for therapeutic purposes and prohibiting germline editing in clinical applications [10][11] - The establishment of strict technical boundaries and international regulatory frameworks is essential to prevent ethical violations and ensure that gene editing serves societal welfare [10][11]
【特稿】全球首例 定制基因编辑疗法治疗婴儿罕见病初见成效
Xin Hua She· 2025-05-16 06:49
Core Insights - A rare disease case involving a baby named KJ Malden has shown promising results from a custom gene editing therapy, marking a potential breakthrough for treating rare genetic disorders [1][2] - The therapy utilized CRISPR technology to correct a genetic mutation causing CPS1 deficiency, a condition with high mortality rates and typically requiring liver transplants for treatment [1][3] Group 1: Treatment Details - KJ Malden, diagnosed with CPS1 deficiency shortly after birth, received the world's first custom gene editing therapy starting in February [1][2] - The treatment involved three intravenous infusions over several months, where lipid nanoparticles delivered "molecular scissors" to correct the mutation in liver cells [2] - Following the treatment, Malden has shown significant improvement, being able to consume a protein-rich diet and recover quickly from minor illnesses [2] Group 2: Industry Context - Approximately 350 million people worldwide suffer from rare diseases, many of which are caused by genetic mutations [3] - The CRISPR gene editing technology, which emerged in 2012, has gained recognition for its precision and efficiency, receiving the Nobel Prize in Chemistry in 2020 [3] - This case represents a significant step in applying gene editing technology to treat various rare diseases, with ongoing observation required to assess long-term effects [2][3]