Genome editing

基因组编辑
  • 文章类型: Journal Article
    癌症的全球负担和常规疗法的局限性凸显了聚集的规则间隔短回文重复序列-CRISPR相关蛋白9(CRISPR-Cas9)在重塑癌症治疗范式中的潜力。在这次审查中,我们已经研究了CRISPR的机制,细菌中的适应性免疫系统,能够在分子水平上进行高度精确的基因编辑。这种多功能工具通过基因敲除证明了其在人类癌症治疗中的功效,代谢中断,基本编辑,筛选,和免疫疗法增强而不影响正常的身体领域。尽管它优于其他核酸酶,如锌指核酸酶和转录激活因子样效应核酸酶,障碍,如脱靶效应,系统向靶细胞的低效递送,逃亡者的出现,并讨论了围绕基因组编辑的伦理辩论。在这篇文章中,我们回顾了CRISPR-Cas9在癌症治疗中的有希望的方法,同时探索其潜在的机制,优势,和相关的挑战。
    The global burden of cancer and the limitations of conventional therapies highlight the potential of clustered regularly interspaced short palindromic repeats-CRISPR-associated protein 9 (CRISPR-Cas9) in reshaping cancer treatment paradigms. In this review, we have investigated the mechanism of CRISPR, an adaptive immune system in bacteria that enables highly precise gene editing at the molecular level. This versatile tool demonstrates its efficacy in human cancer therapy through gene knockout, metabolic disruption, base editing, screening, and immunotherapy enhancement without affecting normal bodily domains. Despite its superiority over other nucleases like zinc-finger nucleases and transcription activator-like effector nucleases, hurdles such as off-target effects, inefficient delivery of the system to target cells, the emergence of escapers, and the ethical debate surrounding genome editing are discussed. In this article, we have reviewed the promising approaches of CRISPR-Cas9 in cancer treatment while exploring the underlying mechanism, advantages, and associated challenges.
    导出

    更多引用

    收藏

    翻译标题摘要

    我要上传

       PDF(Pubmed)

  • 文章类型: Journal Article
    世界上相当一部分人口患有罕见疾病。聚集的定期间隔短回文重复(CRISPR)及其相关的Cas蛋白为治疗罕见疾病提供了一种现代形式的治愈性基因治疗。遗传性转甲状腺素蛋白淀粉样变性,遗传性血管性水肿,杜氏肌营养不良症和Rett综合征是此类罕见疾病的一些例子。例如,CRISPR/Cas9,已用于治疗β-地中海贫血和镰状细胞病(Frangoul等人。,2021年;Pavani等人。,2021)[1,2]。神经系统疾病如亨廷顿氏症也被集中在一些涉及CRISPR/Cas的研究中(Yangetal.,2017;Yan等人。,2023年)[3,4]。通过载体和非载体介导的方法递送这些生物制品取决于靶细胞的类型,表达的特点,表达式的持续时间,外来遗传物质的大小等。例如,逆转录病毒由于其整合到宿主基因组中的能力而在体细胞中离体递送的情况下发现其适用性。这些已成功用于涉及X-SCID患者的基因治疗,据报道,不适当激活的发生率。另一方面,β-地中海贫血的离体基因治疗涉及使用BB305慢病毒载体在HSC中高水平表达CRISPR生物学。这些生物制品的有效性和安全性将决定它们作为有效的基因组编辑工具的未来应用,因为它们将在人类临床试验的进一步阶段进行。这篇综述的重点是基于CRISPR/Cas的疗法,这些疗法处于治疗罕见疾病的临床试验的不同阶段,以及与之相关的限制和伦理问题。
    A considerable fraction of population in the world suffers from rare diseases. Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) and its related Cas proteins offer a modern form of curative gene therapy for treating the rare diseases. Hereditary transthyretin amyloidosis, hereditary angioedema, duchenne muscular dystrophy and Rett syndrome are a few examples of such rare diseases. CRISPR/Cas9, for example, has been used in the treatment of β-thalassemia and sickle cell disease (Frangoul et al., 2021; Pavani et al., 2021) [1,2]. Neurological diseases such as Huntington\'s have also been focused in some studies involving CRISPR/Cas (Yang et al., 2017; Yan et al., 2023) [3,4]. Delivery of these biologicals via vector and non vector mediated methods depends on the type of target cells, characteristics of expression, time duration of expression, size of foreign genetic material etc. For instance, retroviruses find their applicability in case of ex vivo delivery in somatic cells due to their ability to integrate in the host genome. These have been successfully used in gene therapy involving X-SCID patients although, incidence of inappropriate activation has been reported. On the other hand, ex vivo gene therapy for β-thalassemia involved use of BB305 lentiviral vector for high level expression of CRISPR biological in HSCs. The efficacy and safety of these biologicals will decide their future application as efficient genome editing tools as they go forward in further stages of human clinical trials. This review focuses on CRISPR/Cas based therapies which are at various stages of clinical trials for treatment of rare diseases and the constraints and ethical issues associated with them.
    导出

    更多引用

    收藏

    翻译标题摘要

    我要上传

    求助全文

  • 文章类型: Journal Article
    在植物衍生的次生代谢产物中,苄基异喹啉生物碱(BIA)在医学中起着至关重要的作用。罂粟中最常见的BIA是吗啡,可待因,蒂贝恩,罂粟碱,血根碱,和noscapine。BIA提供了丰富的临床上有用的药物,用于治疗各种疾病和疾病随着对这些草药的需求不断增加,罂粟植物的遗传改良似乎对于实现制药业的期望至关重要。随着成簇的规则间隔短回文重复(CRISPR)/CRISPR相关9(Cas9)的出现,代谢工程领域由于其吸引人的属性,其方法发生了范式转变,例如无转基因编辑能力,精度,选择性,鲁棒性,和多功能性。证明了CRISPR系统操纵罂粟代谢途径的潜力,但是,应进行有关在BIA途径工程中使用CRISPR的进一步研究,以将罂粟开发成工业规模合成BIA的生物反应器。在这方面,招募RNA指导的基因组编辑以敲除miRNA,负责花朵的基因,参与竞争途径的基因,和基础编辑进行了描述。迄今为止,这里提出的方法从未被建议或应用于罂粟。
    Among plant-derived secondary metabolites are benzylisoquinoline alkaloids (BIAs) that play a vital role in medicine. The most conspicuous BIAs frequently found in opium poppy are morphine, codeine, thebaine, papaverine, sanguinarine, and noscapine. BIAs have provided abundant clinically useful drugs used in the treatment of various diseases and ailments With an increasing demand for these herbal remedies, genetic improvement of poppy plants appears to be essential to live up to the expectations of the pharmaceutical industry. With the advent of clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated9 (Cas9), the field of metabolic engineering has undergone a paradigm shift in its approach due to its appealing attributes, such as the transgene-free editing capability, precision, selectivity, robustness, and versatility. The potentiality of the CRISPR system for manipulating metabolic pathways in opium poppy was demonstrated, but further investigations regarding the use of CRISPR in BIA pathway engineering should be undertaken to develop opium poppy into a bioreactor synthesizing BIAs at the industrial-scale levels. In this regard, the recruitment of RNA-guided genome editing for knocking out miRNAs, flower responsible genes, genes involved in competitive pathways, and base editing are described. The approaches presented here have never been suggested or applied in opium poppy so far.
    导出

    更多引用

    收藏

    翻译标题摘要

    我要上传

       PDF(Pubmed)

  • 文章类型: Journal Article
    蘑菇的遗传研究,在CRISPR-Cas9基因组编辑和RNA干扰等创新的推动下,改变我们对这些神秘真菌及其在农业中的多方面作用的理解,医学,和保护。这篇综合综述探讨了蘑菇遗传研究的基本原理和意义,深入研究道德,监管,和这个领域的生态维度。CRISPR-Cas9作为一种改变游戏规则的技术出现,实现精确的基因组编辑,靶向基因敲除,和路径操纵。RNA干扰通过下调基因以提高作物产量和增强病虫害抗性来补充这些努力。遗传研究还有助于保护稀有物种和开发更强大的蘑菇菌株,促进可持续种植实践。此外,它们释放了发现新的药用化合物的潜力,为制药和营养品提供新的视野。随着新兴技术和伦理考虑塑造了蘑菇研究的未来,这些研究有望彻底改变我们与这些真菌的关系,为更可持续和创新的世界铺平道路。
    Genetic studies in mushrooms, driven by innovations such as CRISPR-Cas9 genome editing and RNA interference, transform our understanding of these enigmatic fungi and their multifaceted roles in agriculture, medicine, and conservation. This comprehensive review explores the rationale and significance of genetic research in mushrooms, delving into the ethical, regulatory, and ecological dimensions of this field. CRISPR-Cas9 emerges as a game-changing technology, enabling precise genome editing, targeted gene knockouts, and pathway manipulation. RNA interference complements these efforts by downregulating genes for improved crop yield and enhanced pest and disease resistance. Genetic studies also contribute to the conservation of rare species and developing more robust mushroom strains, fostering sustainable cultivation practices. Moreover, they unlock the potential for discovering novel medicinal compounds, offering new horizons in pharmaceuticals and nutraceuticals. As emerging technologies and ethical considerations shape the future of mushroom research, these studies promise to revolutionize our relationship with these fungi, paving the way for a more sustainable and innovative world.
    导出

    更多引用

    收藏

    翻译标题摘要

    我要上传

    求助全文

  • 文章类型: Journal Article
    基因组编辑是一种在细胞或生物体的DNA中进行特定改变的技术。它极大地改变了生命科学的格局,促进建立极其定制的遗传修饰。在各种基因组编辑技术中,CRISPR/Cas9系统,特定的核酸内切酶诱导双链DNA断裂,并能够对基因组进行修饰,已经成为一种强大而适应性强的工具。它的重要性怎么强调都不为过,因为它不仅允许在模型生物中操纵基因组,而且在医学上具有革命性进步的巨大潜力,特别是在治疗遗传疾病方面。这篇综述论文探讨了CRISPR/Cas9的非凡旅程,它的自然功能,机制,以及对基因组编辑的变革性影响,最后是人工智能和其他智能制造工具的使用。引言提供了基因组编辑的背景,强调CRISPR/Cas9的出现和意义。随后的章节全面阐述了它的自然功能,疾病建模,农业,和生物技术,解决治疗应用,和正在进行的临床试验,同时还讨论前景和伦理影响。我们总结了主要发现,这表明CRISPR/Cas9增强了疾病特异性动物模型的创建能力。这为致病机制提供了宝贵的见解,并为药物发现开辟了新途径,重申CRISPR/Cas9对基因组编辑的变革性影响。最后,我们讨论了继续研究和合作的重要性,以综合利用这种分子精密工具在塑造即将到来的进步中的固有能力。
    Genome editing is a technology to make specific changes in the DNA of a cell or an organism. It has significantly altered the landscape of life sciences, facilitating the establishment of exceedingly customized genetic modifications. Among various genome editing technologies, the CRISPR/Cas9 system, a specific endonuclease induces a double stranded DNA break and enabling modifications to the genome, has surfaced as a formidable and adaptable instrument. Its significance cannot be overstated, as it not only allows for the manipulation of genomes in model organisms but also holds great potential for revolutionary advances in medicine, particularly in treating genetic diseases. This review paper explores the remarkable journey of CRISPR/Cas9, its natural function, mechanisms, and transformative impact on genome editing and finally the use of artificial intelligence and other intelligent manufacturing tools used. The introduction provides the background on genome editing, emphasizing the emergence and significance of CRISPR/Cas9. Subsequent sections comprehensively elucidate its natural function, disease modeling, agriculture, and biotechnology, address therapeutic applications, and ongoing clinical trials while also discussing prospects and ethical implications. We summarized the key findings, indicating that CRISPR/Cas9 has empowered the creation of disease-specific animal models. This provides invaluable insights into pathogenic mechanisms and opens new avenues for drug discovery, reaffirming the transformative impact of CRISPR/Cas9 on genome editing. Finally we discussed the importance of continued research and collaboration for comprehensive utilization of the inherent capabilities of this molecular precision tool in shaping forthcoming advancements.
    导出

    更多引用

    收藏

    翻译标题摘要

    我要上传

       PDF(Pubmed)

  • 文章类型: Journal Article
    结论:该综述在三个主题下讨论了小小米的生长和干旱响应机制:干旱逃逸,抗旱和抗旱。干旱是影响植物生长的最突出的非生物胁迫之一,性能,和生产力。在气候变化的背景下,在全球许多农业地区,干旱的患病率和严重性预计将增加。谷子(粗粮)是在世界各地的干旱和半干旱地区种植的一组小种子草,是人类和牲畜的重要食物和饲料来源。虽然是小小米,即,谷草,手指小米,proso小米,谷子,kodo小米和小小米通常比谷物和主要小米(高粱和小米)更坚硬,更耐旱,理解他们的反应,应对干旱的过程和策略更为有限。这里,我们在三个主题下回顾了小小米的抗旱策略:干旱逃逸(例如,作物周期短,短植物期,同化物的发育可塑性和再动员),避免干旱(例如,根系性状更好的吸水率和叶片性状控制水分流失),和耐旱性(例如,渗透调节,维持光合能力和抗氧化潜力)。总结了来自“组学”研究的数据,以概述在耐旱性中重要的分子机制。此外,最后一节强调了知识差距和改善小小米的挑战。鉴于与气候相关的干旱增加,本综述旨在增强主要谷物和小米本身。
    CONCLUSIONS: The review discusses growth and drought-response mechanisms in minor millets under three themes: drought escape, drought avoidance and drought tolerance. Drought is one of the most prominent abiotic stresses impacting plant growth, performance, and productivity. In the context of climate change, the prevalence and severity of drought is expected to increase in many agricultural regions worldwide. Millets (coarse grains) are a group of small-seeded grasses cultivated in arid and semi-arid regions throughout the world and are an important source of food and feed for humans and livestock. Although minor millets, i.e., foxtail millet, finger millet, proso millet, barnyard millet, kodo millet and little millet are generally hardier and more drought-resistant than cereals and major millets (sorghum and pearl millet), understanding their responses, processes and strategies in response to drought is more limited. Here, we review drought resistance strategies in minor millets under three themes: drought escape (e.g., short crop cycle, short vegetative period, developmental plasticity and remobilization of assimilates), drought avoidance (e.g., root traits for better water absorption and leaf traits to control water loss), and drought tolerance (e.g., osmotic adjustment, maintenance of photosynthetic ability and antioxidant potential). Data from \'omics\' studies are summarized to provide an overview of the molecular mechanisms important in drought tolerance. In addition, the final section highlights knowledge gaps and challenges to improving minor millets. This review is intended to enhance major cereals and millet per se in light of climate-related increases in aridity.
    导出

    更多引用

    收藏

    翻译标题摘要

    我要上传

    求助全文

  • 文章类型: Systematic Review
    新植物育种技术的进步已成为提高作物生产力的有希望的工具,质量,面对全球挑战的韧性,比如气候变化和粮食安全。然而,这些技术的成功实施还依赖于公众对这项创新的接受。了解是什么塑造了公众对新植物育种技术的看法和接受对于有效的科学交流至关重要,决策,以及这些创新的可持续采用。本系统综述的目的是综合现有的关于公众对新植物育种技术应用于粮食作物的看法的研究,并探索影响接受的社会心理决定因素。2015年至2023年之间发表的20篇论文包括各种新的植物育种技术及其受到公众的欢迎。影响从新植物育种技术获得的粮食作物接受的决定因素分为六个方面:社会人口统计学因素,感知的利益和风险,对科学的态度,沟通策略,个人价值观,和产品特性。
    Advancements in New Plant Breeding Techniques have emerged as promising tools for enhancing crop productivity, quality, and resilience in the face of global challenges, such as climate change and food security. However, the successful implementation of these techniques relies also on public acceptance of this innovation. Understanding what shapes public perception and acceptance of New Plant Breeding Techniques is crucial for effective science communication, policymaking, and the sustainable adoption of these innovations. The objective of this systematic review was to synthesize existing research on the public perception of New Plant Breeding Techniques applied to food crops and explore the psychosocial determinants that influence acceptance. Twenty papers published between 2015 and 2023 were included on various New Plant Breeding Techniques and their reception by the general public. Determinants affecting the acceptance of food crops derived from New Plant Breeding Techniques were categorized into six areas: sociodemographic factors, perceived benefits and risks, attitudes toward science, communication strategies, personal values, and product characteristics.
    导出

    更多引用

    收藏

    翻译标题摘要

    我要上传

       PDF(Pubmed)

  • 文章类型: Journal Article
    CRISPR/Cas9基因编辑系统是一种多功能的基因修饰技术,在植物功能基因研究和遗传育种中起着至关重要的作用,动物,真菌,和微生物。本文综述了该技术在食用菌基因研究和遗传育种中的应用。审查涵盖了各个方面,包括Cas9和sgRNA的传递和表达策略,遗传转化方法,突变筛选,以及DNA双链断裂后靶位点的修复策略。此外,综述了该技术在食用菌中应用的主要挑战和优化策略。最后,讨论了该技术在食用菌研究中的未来应用潜力,借鉴作者个人的研究背景。
    The CRISPR/Cas9 gene editing system is a versatile technology for modifying gene, playing a crucial role in the study of functional genes and genetic breeding of plants, animals, fungi, and microorganisms. This review provides a comprehensive analysis of the application of this technology in gene research and genetic breeding of edible fungi. The review covers various aspects, including the delivery and expression strategies of Cas9 and sgRNA, genetic transformation methods, mutant screening, and repair strategies for target sites following DNA double-strand breaks. Additionally, the review summarizes the main challenges and optimization strategies associated with the application of this technology in edible fungi. Lastly, the future application potential of this technology in edible fungi research is discussed, drawing from the authors\' personal research background.
    导出

    更多引用

    收藏

    翻译标题摘要

    我要上传

    求助全文

  • 文章类型: Journal Article
    癌症是导致死亡的主要原因之一。人类基因组随着时间的推移保持稳定。然而,人类活动和环境因素有能力影响某些类型的突变的流行。这涉及到外源性生物和工业发展的过度进步,这正在扩大癌症发展的领域。涉及针对癌症的免疫应答的机制被广泛研究。基因组编辑改变了人体内基于基因组的免疫治疗过程,开启了癌症治疗的新纪元。在这次审查中,最近癌症免疫疗法和基因组工程技术的使用主要集中在。
    Cancer is one of the foremost causes of mortality. The human genome remains stable over time. However, human activities and environmental factors have the power to influence the prevalence of certain types of mutations. This goes to the excessive progress of xenobiotics and industrial development that is expanding the territory for cancers to develop. The mechanisms involved in immune responses against cancer are widely studied. Genome editing has changed the genome-based immunotherapy process in the human body and has opened a new era for cancer treatment. In this review, recent cancer immunotherapies and the use of genome engineering technology are largely focused on.
    导出

    更多引用

    收藏

    翻译标题摘要

    我要上传

       PDF(Pubmed)

  • 文章类型: Journal Article
    CRISPR-Cas9已经成为一种革命性的工具,可以对遗传物质进行精确有效的修饰。本文综述了CRISPR-Cas9技术及其在基因组编辑中的应用。我们首先描述CRISPR-Cas9技术的基本原理,解释该系统如何利用单个指导RNA(sgRNA)将Cas9核酸酶引导到基因组中的特定DNA序列,导致有针对性的双链断裂。在这次审查中,我们对CRISPR-Cas9技术及其在农业中的应用进行了深入的探索,医学,环境科学,渔业,纳米技术,生物信息学,和生物技术。我们还强调了它的潜力,正在进行的研究,以及围绕其使用的道德考虑和争议。这篇综述可能有助于理解CRISPR-Cas9技术及其在各个领域的意义。为这种变革性技术的未来发展和负责任的应用铺平道路。
    CRISPR-Cas9 has emerged as a revolutionary tool that enables precise and efficient modifications of the genetic material. This review provides a comprehensive overview of CRISPR-Cas9 technology and its applications in genome editing. We begin by describing the fundamental principles of CRISPR-Cas9 technology, explaining how the system utilizes a single guide RNA (sgRNA) to direct the Cas9 nuclease to specific DNA sequences in the genome, resulting in targeted double-stranded breaks. In this review, we provide in-depth explorations of CRISPR-Cas9 technology and its applications in agriculture, medicine, environmental sciences, fisheries, nanotechnology, bioinformatics, and biotechnology. We also highlight its potential, ongoing research, and the ethical considerations and controversies surrounding its use. This review might contribute to the understanding of CRISPR-Cas9 technology and its implications in various fields, paving the way for future developments and responsible applications of this transformative technology.
    导出

    更多引用

    收藏

    翻译标题摘要

    我要上传

       PDF(Pubmed)

公众号