首页 > 最新文献

Biochemist最新文献

英文 中文
Meeting Report 会议报告
Q4 Biochemistry, Genetics and Molecular Biology Pub Date : 2023-09-07 DOI: 10.1042/bio_2023_138
Daniel R. Dries
{"title":"Meeting Report","authors":"Daniel R. Dries","doi":"10.1042/bio_2023_138","DOIUrl":"https://doi.org/10.1042/bio_2023_138","url":null,"abstract":"","PeriodicalId":35334,"journal":{"name":"Biochemist","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2023-09-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"135048295","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
From unique challenges to inspired solutions: how vector engineering makes in vivo gene therapy possible 从独特的挑战到灵感的解决方案:载体工程如何使体内基因治疗成为可能
Q4 Biochemistry, Genetics and Molecular Biology Pub Date : 2023-08-24 DOI: 10.1042/bio_2023_127
Rodanthi Lyraki
Recent advances in designing safe and efficient viral vectors have spearheaded unprecedented growth in clinical trials and regulatory approvals for experimental gene therapies. Still, important challenges remain for gene therapies that require systemic administration into the circulation, including the patient’s immune response and the need for precise activation in the target cell population while sparing other tissues. Extensive research in engineering the vector’s external surface and genome has led to clever solutions, allowing novel in vivo therapies for previously untreatable genetic diseases such as spinal muscular atrophy and Duchenne muscular dystrophy to reach the clinic. In the future, we expect novel strategies, such as different viral vectors or non-viral delivery vehicles, to overcome the shortcomings of viral therapy.
最近在设计安全有效的病毒载体方面取得的进展,使实验性基因疗法的临床试验和监管批准出现了前所未有的增长。尽管如此,需要全身给药进入血液循环的基因疗法仍然面临重大挑战,包括患者的免疫反应,以及在不影响其他组织的情况下精确激活靶细胞群的需求。对载体外表面和基因组工程的广泛研究已经带来了巧妙的解决方案,使以前无法治疗的遗传疾病(如脊髓性肌萎缩症和杜氏肌营养不良症)的新型体内疗法进入临床。在未来,我们期待新的策略,如不同的病毒载体或非病毒运载工具,以克服病毒治疗的缺点。
{"title":"From unique challenges to inspired solutions: how vector engineering makes in vivo gene therapy possible","authors":"Rodanthi Lyraki","doi":"10.1042/bio_2023_127","DOIUrl":"https://doi.org/10.1042/bio_2023_127","url":null,"abstract":"Recent advances in designing safe and efficient viral vectors have spearheaded unprecedented growth in clinical trials and regulatory approvals for experimental gene therapies. Still, important challenges remain for gene therapies that require systemic administration into the circulation, including the patient’s immune response and the need for precise activation in the target cell population while sparing other tissues. Extensive research in engineering the vector’s external surface and genome has led to clever solutions, allowing novel in vivo therapies for previously untreatable genetic diseases such as spinal muscular atrophy and Duchenne muscular dystrophy to reach the clinic. In the future, we expect novel strategies, such as different viral vectors or non-viral delivery vehicles, to overcome the shortcomings of viral therapy.","PeriodicalId":35334,"journal":{"name":"Biochemist","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2023-08-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"41939221","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Gene therapy: yesterday, today and tomorrow 基因治疗:昨天,今天和明天
Q4 Biochemistry, Genetics and Molecular Biology Pub Date : 2023-08-24 DOI: 10.1042/bio_2023_137
Heather Doran
This issue focuses on one of the most keenly anticipated and researched topics in medicine – gene therapy. With recent advancements in the field, the hope is that some of the most debilitating diseases, many that seriously affect children, can begin to be treated.It wouldn’t be right to cover this topic in The Biochemist without also acknowledging the ethical considerations of these treatments. First, there are decisions to be made on what conditions might be ‘fixed’ via these methods, if they need fixing at all and what is the outcome for those that undergo these treatments? Although it is heartening to see developments in the treatment of diseases that can have serious life-limiting effects, who gets to decide which ones are prioritized for research?The second factor, which is discussed in a number of articles in this issue, relates to the cost of gene therapy treatments. Research has taken many years and a single-point treatment needs to cover costs, but the extraordinary level of finance required for one of these treatments also limits who has access. How can we make sure all who need it can receive it? Currently, gene therapy treatments are the most expensive treatment method across all medicine, with a single treatment costing millions of dollars.Lastly, the question is what comes next now that this technology is in use? What limits could/should there be (or not be) on the use of gene therapy?Biology, medicine and research are never in isolation from society and this is one area that highlights how important it is to have engagement and involvement from patients and the public, along with law and experts in the social sciences in the discussions of the creation, limits and applications of these approaches.The possibilities of gene therapy took longer than expected to be realized. Now different delivery methods of viral and non-viral vectors have been created to make the process more efficient and safer. The articles in this issue are looking at in vivo gene therapies, the challenges for the future and the steps that have taken us to where we are today.
这期的重点是医学上最热切期待和研究的主题之一-基因治疗。随着该领域的最新进展,人们希望能够开始治疗一些最令人衰弱的疾病,其中许多严重影响儿童。如果不承认这些治疗的伦理考虑,在《生物化学家》中讨论这个话题是不对的。首先,需要决定哪些情况可以通过这些方法“修复”,如果它们需要修复,以及接受这些治疗的人的结果是什么?尽管在治疗可能严重限制生命的疾病方面取得进展令人振奋,但谁来决定哪些疾病应该优先进行研究呢?第二个因素与基因治疗的费用有关,这在本期的许多文章中都有讨论。研究已经进行了多年,单点治疗需要支付费用,但其中一种治疗所需的巨额资金也限制了谁能获得这种治疗。我们如何确保所有需要的人都能得到它?目前,基因疗法是所有药物中最昂贵的治疗方法,单次治疗就需要花费数百万美元。最后一个问题是,既然这项技术已经投入使用,接下来会发生什么?基因疗法的使用可能/应该(或不应该)有哪些限制?生物学、医学和研究从来都不是孤立于社会之外的,这一领域突出表明,让患者和公众以及法律和社会科学专家参与和参与讨论这些方法的创造、限制和应用是多么重要。基因治疗的可能性比预期的实现时间要长。现在,不同的病毒载体和非病毒载体的递送方法已经被创造出来,使这一过程更有效、更安全。本期的文章关注的是体内基因疗法,未来的挑战以及我们取得今天成就的步骤。
{"title":"Gene therapy: yesterday, today and tomorrow","authors":"Heather Doran","doi":"10.1042/bio_2023_137","DOIUrl":"https://doi.org/10.1042/bio_2023_137","url":null,"abstract":"This issue focuses on one of the most keenly anticipated and researched topics in medicine – gene therapy. With recent advancements in the field, the hope is that some of the most debilitating diseases, many that seriously affect children, can begin to be treated.It wouldn’t be right to cover this topic in The Biochemist without also acknowledging the ethical considerations of these treatments. First, there are decisions to be made on what conditions might be ‘fixed’ via these methods, if they need fixing at all and what is the outcome for those that undergo these treatments? Although it is heartening to see developments in the treatment of diseases that can have serious life-limiting effects, who gets to decide which ones are prioritized for research?The second factor, which is discussed in a number of articles in this issue, relates to the cost of gene therapy treatments. Research has taken many years and a single-point treatment needs to cover costs, but the extraordinary level of finance required for one of these treatments also limits who has access. How can we make sure all who need it can receive it? Currently, gene therapy treatments are the most expensive treatment method across all medicine, with a single treatment costing millions of dollars.Lastly, the question is what comes next now that this technology is in use? What limits could/should there be (or not be) on the use of gene therapy?Biology, medicine and research are never in isolation from society and this is one area that highlights how important it is to have engagement and involvement from patients and the public, along with law and experts in the social sciences in the discussions of the creation, limits and applications of these approaches.The possibilities of gene therapy took longer than expected to be realized. Now different delivery methods of viral and non-viral vectors have been created to make the process more efficient and safer. The articles in this issue are looking at in vivo gene therapies, the challenges for the future and the steps that have taken us to where we are today.","PeriodicalId":35334,"journal":{"name":"Biochemist","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2023-08-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"135464911","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Healing hearts: unlocking the potential of gene therapy in revolutionizing cardiovascular disease treatment 治愈心脏:释放基因疗法在心血管疾病治疗中的革命性潜力
Q4 Biochemistry, Genetics and Molecular Biology Pub Date : 2023-08-23 DOI: 10.1042/bio_2023_129
A. Beqqali, A. Baker
In medical science, few areas hold as much promise and excitement as gene therapy. It is a cutting-edge field that aims to harness the body’s own genetic machinery to combat diseases at their root cause. While gene therapy has shown potential across various diseases (notably in rare monogenetic disease areas), here we dive into the latest advancements specifically focused on the heart. With cardiovascular diseases remaining a leading cause of global morbidity and mortality, researchers are tirelessly exploring innovative ways to tackle rare and common heart-related ailments. In this article, we discuss the fascinating world of gene therapy, exploring recent breakthroughs as well as remaining challenges and their implications for cardiovascular health.
在医学科学中,很少有领域能像基因治疗那样充满希望和激动人心。这是一个前沿领域,旨在利用人体自身的基因机制,从根源上对抗疾病。虽然基因疗法已经在各种疾病(特别是罕见的单基因疾病领域)中显示出潜力,但在这里,我们将深入研究专门针对心脏的最新进展。由于心血管疾病仍然是全球发病率和死亡率的主要原因,研究人员正在不知疲倦地探索解决罕见和常见心脏相关疾病的创新方法。在这篇文章中,我们讨论了基因治疗的迷人世界,探索了最近的突破以及仍然存在的挑战及其对心血管健康的影响。
{"title":"Healing hearts: unlocking the potential of gene therapy in revolutionizing cardiovascular disease treatment","authors":"A. Beqqali, A. Baker","doi":"10.1042/bio_2023_129","DOIUrl":"https://doi.org/10.1042/bio_2023_129","url":null,"abstract":"In medical science, few areas hold as much promise and excitement as gene therapy. It is a cutting-edge field that aims to harness the body’s own genetic machinery to combat diseases at their root cause. While gene therapy has shown potential across various diseases (notably in rare monogenetic disease areas), here we dive into the latest advancements specifically focused on the heart. With cardiovascular diseases remaining a leading cause of global morbidity and mortality, researchers are tirelessly exploring innovative ways to tackle rare and common heart-related ailments. In this article, we discuss the fascinating world of gene therapy, exploring recent breakthroughs as well as remaining challenges and their implications for cardiovascular health.","PeriodicalId":35334,"journal":{"name":"Biochemist","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2023-08-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"45554140","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Foes to friends: do pathogens hold the key to gene therapy? 朋友们的烦恼:病原体是基因治疗的关键吗?
Q4 Biochemistry, Genetics and Molecular Biology Pub Date : 2023-08-21 DOI: 10.1042/bio_2023_131
M. Pearson, Rafael J. Yáñez-Muñoz
Imagine starting life with a disease where the chances of reaching a fifth birthday are unlikely, let alone making it to adulthood, and the only treatments available are palliative at best. This is the reality facing millions of individuals born with a rare genetic disease. Sometimes, due to a single DNA base change – out of a possible 3 billion – occurring in the wrong place. A simple spelling mistake! However, recent years have seen the first effective gene therapies being developed, based on supplementing patient cells with functional copies of the faulty genes, or using antisense oligonucleotides or small molecules to alter pre-mRNA processing. The challenges were how to deliver genes or alter gene expression in the diseased cells and how to do it safely, without negatively affecting other endogenous genes. The answers came in the form of viruses, small non-coding RNAs and bacterial proteins, which is somewhat ironic, because we are using infectious agents or mimetics of their products to help cure diseases. With old enemies turned allies, tool sets are now being assembled to tackle the most challenging of genetic diseases. This article explores some of those tool chests in further detail, using ataxia telangiectasia, spinal muscular atrophy and several other rare diseases to highlight progress and challenges.
想象一下,在一种疾病开始生活时,不太可能活到五岁生日,更不用说成年了,而且唯一可用的治疗方法充其量是姑息治疗。这是数百万天生患有罕见遗传病的人所面临的现实。有时,由于可能有30亿个DNA碱基中的一个碱基发生在错误的地方。一个简单的拼写错误!然而,近年来,基于向患者细胞补充缺陷基因的功能拷贝,或使用反义寡核苷酸或小分子来改变前信使核糖核酸的处理,已经开发出了第一种有效的基因疗法。挑战在于如何在患病细胞中传递基因或改变基因表达,以及如何在不对其他内源性基因产生负面影响的情况下安全地进行。答案是以病毒、小型非编码RNA和细菌蛋白的形式出现的,这有点讽刺,因为我们正在使用传染源或其产品的模拟物来帮助治愈疾病。随着宿敌变成盟友,现在正在组装工具集来应对最具挑战性的遗传疾病。本文进一步详细探讨了其中的一些工具箱,利用共济失调毛细血管扩张症、脊髓性肌萎缩和其他几种罕见疾病来强调进展和挑战。
{"title":"Foes to friends: do pathogens hold the key to gene therapy?","authors":"M. Pearson, Rafael J. Yáñez-Muñoz","doi":"10.1042/bio_2023_131","DOIUrl":"https://doi.org/10.1042/bio_2023_131","url":null,"abstract":"Imagine starting life with a disease where the chances of reaching a fifth birthday are unlikely, let alone making it to adulthood, and the only treatments available are palliative at best. This is the reality facing millions of individuals born with a rare genetic disease. Sometimes, due to a single DNA base change – out of a possible 3 billion – occurring in the wrong place. A simple spelling mistake! However, recent years have seen the first effective gene therapies being developed, based on supplementing patient cells with functional copies of the faulty genes, or using antisense oligonucleotides or small molecules to alter pre-mRNA processing. The challenges were how to deliver genes or alter gene expression in the diseased cells and how to do it safely, without negatively affecting other endogenous genes. The answers came in the form of viruses, small non-coding RNAs and bacterial proteins, which is somewhat ironic, because we are using infectious agents or mimetics of their products to help cure diseases. With old enemies turned allies, tool sets are now being assembled to tackle the most challenging of genetic diseases. This article explores some of those tool chests in further detail, using ataxia telangiectasia, spinal muscular atrophy and several other rare diseases to highlight progress and challenges.","PeriodicalId":35334,"journal":{"name":"Biochemist","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2023-08-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"49094162","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Meeting reports 会议报告
Q4 Biochemistry, Genetics and Molecular Biology Pub Date : 2023-08-14 DOI: 10.1042/bio_2023_133
Daniel Humphreys, Sarah Coulthurst
The 156 attendees from 20 countries, dominated by PhD students (53 attendees) and postdoctoral scientists (50), helped create a rich programme of cutting-edge science delivered through 100 posters, 39 offered talks (including mini, short and extended talks) and 19 invited speakers. Sponsorship from the Biochemical Society contributed to an enormously successful event.The programme began with keynote speaker Stefan Raunser (Max Planck Institute of Molecular Physiology, Germany) who summarized a decade of discoveries involving Tc toxin produced by the bacterial pathogen Photorhabdus luminescens. ETOX 2023 showcased two further keynotes who presented exciting advances in antibacterial effector proteins (Chris Hayes, University of California, USA) and Samonella genotoxins (Teresa Frisan, University of Umeå, Sweden).The impact of cryogenic electron microscopy emerged as a key point of interest, as the approach was exploited to reveal new atomic structures of toxins or their delivery systems by scientists such as Martin Pilhofer and David Albesa-Jové. Another key takeaway from ETOX 2023 was the pace of discoveries on bacterial type 6 secretion systems: how they are used by pathogens to deploy toxins that kill bacterial competitors or eukaryotic host cells or influence the surrounding microbiota for pathogen survival. Exciting mechanisms underlying innate immunity and subversion by pathogens were introduced by researchers such as Feng Shao, Neal Alto, Charlotte Odendall and Teresa Thurston. Early career researchers made an enormous contribution to the meeting and lit up the scientific programme with 21 talks including prize winners Rachel Prescott, Marila Torrado, Mohamed ElGhazaly, Tom Arrowsmith, Christy Cheung and Charles Ericson.After a final day of excursions in the Scottish Highlands, a conference dinner, Ceilidh dance and prize giving, the meeting closed with a final announcement – the location of ETOX 2025 in Bilbao, Spain!
来自20个国家的156名与会者,以博士生(53人)和博士后科学家(50人)为主,通过100张海报、39场讲座(包括迷你、简短和扩展讲座)和19名特邀演讲者,帮助创建了一个丰富的前沿科学项目。生化学会的赞助使这次活动取得了巨大的成功。会议由主讲人Stefan Raunser(德国马克斯·普朗克分子生理学研究所)开始,他总结了10年来有关病原菌光habdus luminesens产生的Tc毒素的发现。ETOX 2023还展示了另外两个主题演讲,他们介绍了抗菌效应蛋白(Chris Hayes,美国加州大学)和Samonella基因毒素(Teresa Frisan,瑞典尤梅夫大学)方面令人兴奋的进展。低温电子显微镜的影响成为了一个关键的兴趣点,因为这种方法被Martin Pilhofer和David albesa - jov等科学家用来揭示毒素或其传递系统的新原子结构。ETOX 2023的另一个关键收获是对细菌6型分泌系统的发现速度:病原体如何利用它们来部署毒素,杀死细菌竞争对手或真核宿主细胞,或影响周围的微生物群,以促进病原体的生存。Feng Shao, Neal Alto, Charlotte Odendall和Teresa Thurston等研究人员介绍了先天免疫和病原体颠覆的令人兴奋的机制。早期的职业研究人员为会议做出了巨大的贡献,并通过21场演讲点燃了科学计划,其中包括获奖者Rachel Prescott, Marila Torrado, Mohamed ElGhazaly, Tom Arrowsmith, Christy b张和Charles Ericson。在最后一天的苏格兰高地游览、会议晚宴、Ceilidh舞会和颁奖之后,会议以最后的宣布结束——ETOX 2025的地点在西班牙毕尔巴鄂!
{"title":"Meeting reports","authors":"Daniel Humphreys, Sarah Coulthurst","doi":"10.1042/bio_2023_133","DOIUrl":"https://doi.org/10.1042/bio_2023_133","url":null,"abstract":"The 156 attendees from 20 countries, dominated by PhD students (53 attendees) and postdoctoral scientists (50), helped create a rich programme of cutting-edge science delivered through 100 posters, 39 offered talks (including mini, short and extended talks) and 19 invited speakers. Sponsorship from the Biochemical Society contributed to an enormously successful event.The programme began with keynote speaker Stefan Raunser (Max Planck Institute of Molecular Physiology, Germany) who summarized a decade of discoveries involving Tc toxin produced by the bacterial pathogen Photorhabdus luminescens. ETOX 2023 showcased two further keynotes who presented exciting advances in antibacterial effector proteins (Chris Hayes, University of California, USA) and Samonella genotoxins (Teresa Frisan, University of Umeå, Sweden).The impact of cryogenic electron microscopy emerged as a key point of interest, as the approach was exploited to reveal new atomic structures of toxins or their delivery systems by scientists such as Martin Pilhofer and David Albesa-Jové. Another key takeaway from ETOX 2023 was the pace of discoveries on bacterial type 6 secretion systems: how they are used by pathogens to deploy toxins that kill bacterial competitors or eukaryotic host cells or influence the surrounding microbiota for pathogen survival. Exciting mechanisms underlying innate immunity and subversion by pathogens were introduced by researchers such as Feng Shao, Neal Alto, Charlotte Odendall and Teresa Thurston. Early career researchers made an enormous contribution to the meeting and lit up the scientific programme with 21 talks including prize winners Rachel Prescott, Marila Torrado, Mohamed ElGhazaly, Tom Arrowsmith, Christy Cheung and Charles Ericson.After a final day of excursions in the Scottish Highlands, a conference dinner, Ceilidh dance and prize giving, the meeting closed with a final announcement – the location of ETOX 2025 in Bilbao, Spain!","PeriodicalId":35334,"journal":{"name":"Biochemist","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2023-08-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"135262869","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Meeting Reports 会议报告
Q4 Biochemistry, Genetics and Molecular Biology Pub Date : 2023-08-14 DOI: 10.1042/bio_2023_134
Aga Gambus, Matthias Soller, Ferenc Mueller, Constanze Bonifer
The 6th BCGB Symposium on Genome Structure and Function, at the University of Birmingham, was a great success.The selling point of our meeting was to bring together researchers from across the region interested in various aspects of genome biology – from chromatin organization and gene expression to DNA replication and genome stability.The science was amazing, delegates enthusiastic and happy to network in person. We had over 150 participants, mainly early career researchers (ECRs), who enjoyed networking with our two keynote speakers: Ellen Rothenberg (CalTech) and Dan Tenen (Harvard University), who joined us from the USA. We had 11 invited speakers from all over the UK and EU and 13 elevated abstracts talks, which were delivered in majority by ECRs. We also had two flash poster sessions, which were excellent and acted as a great advertisement for the three poster sessions held.Most of the science presented was new and unpublished and we could appreciate the novel technologies and the emerging concepts in genome biology. The science presented and discussed also covered a wide spectrum of biomedical research – from structural protein biology to clinical trials. We all have learned a lot, and this variety of genome biology science certainly opened the horizon for many of the ECRs attending.Find out more about the event and programme here.
在伯明翰大学召开的第六届基因组结构与功能研讨会取得了圆满成功。我们会议的卖点是将来自该地区对基因组生物学各个方面感兴趣的研究人员聚集在一起——从染色质组织和基因表达到DNA复制和基因组稳定性。科学是惊人的,代表们热情并乐于亲自交流。我们有超过150名参与者,主要是早期职业研究人员(ecr),他们喜欢与我们的两位主题演讲者进行交流:来自美国的Ellen Rothenberg(加州理工学院)和Dan Tenen(哈佛大学)。我们邀请了来自英国和欧盟各地的11位发言人,以及13个高级摘要会谈,这些会谈主要由ecr提供。我们还举办了两场海报活动,非常出色,为三场海报活动做了很好的广告宣传。大多数展示的科学都是新的和未发表的,我们可以欣赏基因组生物学中的新技术和新兴概念。所介绍和讨论的科学也涵盖了广泛的生物医学研究——从结构蛋白质生物学到临床试验。我们都学到了很多,这种基因组生物学的多样性无疑为许多参加ecr的人打开了视野。点击这里了解更多有关活动和节目的信息。
{"title":"Meeting Reports","authors":"Aga Gambus, Matthias Soller, Ferenc Mueller, Constanze Bonifer","doi":"10.1042/bio_2023_134","DOIUrl":"https://doi.org/10.1042/bio_2023_134","url":null,"abstract":"The 6th BCGB Symposium on Genome Structure and Function, at the University of Birmingham, was a great success.The selling point of our meeting was to bring together researchers from across the region interested in various aspects of genome biology – from chromatin organization and gene expression to DNA replication and genome stability.The science was amazing, delegates enthusiastic and happy to network in person. We had over 150 participants, mainly early career researchers (ECRs), who enjoyed networking with our two keynote speakers: Ellen Rothenberg (CalTech) and Dan Tenen (Harvard University), who joined us from the USA. We had 11 invited speakers from all over the UK and EU and 13 elevated abstracts talks, which were delivered in majority by ECRs. We also had two flash poster sessions, which were excellent and acted as a great advertisement for the three poster sessions held.Most of the science presented was new and unpublished and we could appreciate the novel technologies and the emerging concepts in genome biology. The science presented and discussed also covered a wide spectrum of biomedical research – from structural protein biology to clinical trials. We all have learned a lot, and this variety of genome biology science certainly opened the horizon for many of the ECRs attending.Find out more about the event and programme here.","PeriodicalId":35334,"journal":{"name":"Biochemist","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2023-08-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"135263389","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
AAV-mediated gene therapy for rare metabolic disorders: turning a promise into a reality AAV介导的罕见代谢紊乱基因治疗:将希望变为现实
Q4 Biochemistry, Genetics and Molecular Biology Pub Date : 2023-08-14 DOI: 10.1042/bio_2023_128
B. Şeker Yılmaz, P. Gissen
Gene therapy is emerging as the realistic treatment option for inborn errors of metabolism (IEMs) and, with the promising safety and efficacy evidence from the proof-of-concept studies, adeno-associated virus (AAV) has become the frontrunner among viral vector candidates for these monogenic disorders. Different AAV capsids exhibit specific tissue tropisms, which can considerably increase the efficiency of gene transfer to particular organs. Here, we will discuss two distinct diseases: ornithine transcarbamylase (OTC) deficiency and Niemann–Pick disease type C, in which significant advances have been achieved in AAV-based gene therapy trials.
基因治疗正在成为先天性代谢错误(IEMs)的现实治疗选择,并且随着来自概念验证研究的有希望的安全性和有效性证据,腺相关病毒(AAV)已成为这些单基因疾病的候选病毒载体中的领跑者。不同的AAV衣壳表现出特定的组织趋向性,这可以大大提高基因转移到特定器官的效率。在这里,我们将讨论两种不同的疾病:鸟氨酸转氨基甲酰基酶(OTC)缺乏症和C型尼曼-皮克病,这两种疾病在基于aav的基因治疗试验中取得了重大进展。
{"title":"AAV-mediated gene therapy for rare metabolic disorders: turning a promise into a reality","authors":"B. Şeker Yılmaz, P. Gissen","doi":"10.1042/bio_2023_128","DOIUrl":"https://doi.org/10.1042/bio_2023_128","url":null,"abstract":"Gene therapy is emerging as the realistic treatment option for inborn errors of metabolism (IEMs) and, with the promising safety and efficacy evidence from the proof-of-concept studies, adeno-associated virus (AAV) has become the frontrunner among viral vector candidates for these monogenic disorders. Different AAV capsids exhibit specific tissue tropisms, which can considerably increase the efficiency of gene transfer to particular organs. Here, we will discuss two distinct diseases: ornithine transcarbamylase (OTC) deficiency and Niemann–Pick disease type C, in which significant advances have been achieved in AAV-based gene therapy trials.","PeriodicalId":35334,"journal":{"name":"Biochemist","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2023-08-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"43590198","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
The value of the ‘Science and Economic Development’ Parliamentary Links Day 2023 2023年“科学与经济发展”议会联系日的价值
Q4 Biochemistry, Genetics and Molecular Biology Pub Date : 2023-08-10 DOI: 10.1042/bio_2023_130
Calum Upton
Potential impact is an important driver in the career choices and journeys of many. There have even been organizations set up to help people make career decisions that can boost their global impact, such as ‘80000hours.org’. Impact has always been an important motivator for me in my decisions; however, I often find myself feeling helpless to make a significant impact through my research. Informing policymaking is an activity where scientists can use their expertise to have a beneficial impact, but how to engage with policymakers can be unclear. This is why I was so excited to have been invited to attend the Royal Society of Biology’s 2023 Parliamentary Links Day on Science and Economic Development. ‘The best way to make an impact is to join scientific advisory councils and panels” was the most important recommendation I took home with me following the event; advice which helped me walk away with more clarity on how I can be impactful.
在许多人的职业选择和职业生涯中,潜在的影响是一个重要的驱动因素。甚至还成立了一些组织,帮助人们做出可以提升其全球影响力的职业决定,比如“80000hours.org”。影响一直是我做决定的重要动力;然而,我经常发现自己无法通过我的研究产生重大影响。为政策制定提供信息是一项活动,科学家可以利用他们的专业知识产生有益的影响,但是如何与决策者接触可能不清楚。这就是为什么我被邀请参加英国皇家生物学会的2023年科学与经济发展议会联系日时如此兴奋的原因。“产生影响的最佳方式是加入科学咨询委员会和小组”是我在活动结束后带回家的最重要的建议;这些建议帮助我更清楚地知道如何才能有影响力。
{"title":"The value of the ‘Science and Economic Development’ Parliamentary Links Day 2023","authors":"Calum Upton","doi":"10.1042/bio_2023_130","DOIUrl":"https://doi.org/10.1042/bio_2023_130","url":null,"abstract":"Potential impact is an important driver in the career choices and journeys of many. There have even been organizations set up to help people make career decisions that can boost their global impact, such as ‘80000hours.org’. Impact has always been an important motivator for me in my decisions; however, I often find myself feeling helpless to make a significant impact through my research. Informing policymaking is an activity where scientists can use their expertise to have a beneficial impact, but how to engage with policymakers can be unclear. This is why I was so excited to have been invited to attend the Royal Society of Biology’s 2023 Parliamentary Links Day on Science and Economic Development. ‘The best way to make an impact is to join scientific advisory councils and panels” was the most important recommendation I took home with me following the event; advice which helped me walk away with more clarity on how I can be impactful.","PeriodicalId":35334,"journal":{"name":"Biochemist","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2023-08-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"48195386","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
International Sponsored Places: 47th FEBS Congress and 22nd Young Scientists’ Forum 2023 国际赞助地点:2023第47届FEBS大会和第22届青年科学家论坛
Q4 Biochemistry, Genetics and Molecular Biology Pub Date : 2023-08-10 DOI: 10.1042/bio_2023_136
Lucy Ollett
Each year, the Biochemical Society fully sponsors the attendance of two outstanding postgraduate or early career members to attend the Federation of European Biochemical Societies (FEBS) Congress and Young Scientists’ Forum (YSF). With a theme of ‘Together in bioscience for a better future’, the 47th FEBS Congress and 22nd YSF combined research presentations, general talks and practical career exercises to showcase the newest developments in biochemistry, molecular biology and related areas. Providing an incredible opportunity to make connections with European colleagues and discuss research with an international audience, we were delighted to offer International Sponsored Places to Dr Yang Li (University College London, UK) and Dr Ewa Blaszczak (Medical University of Lublin, Poland) in 2023. Here, Yang and Ewa discuss their experiences at this year’s meetings and highlight the valuable networking and interactions developed as a result of their participation.
每年,生化学会全额赞助两名优秀的研究生或早期职业成员参加欧洲生化学会联合会(FEBS)大会和青年科学家论坛(YSF)。第47届FEBS大会暨第22届YSF以“携手生物科学共创美好未来”为主题,结合研究报告、一般讲座及实际职业练习,展示生物化学、分子生物学及相关领域的最新发展。我们很高兴在2023年为Yang Li博士(英国伦敦大学学院)和Ewa Blaszczak博士(波兰卢布林医科大学)提供国际赞助名额,这是一个与欧洲同事建立联系并与国际观众讨论研究的绝佳机会。在这里,Yang和Ewa讨论了他们在今年会议上的经验,并强调了由于他们的参与而建立的宝贵的网络和互动。
{"title":"International Sponsored Places: 47th FEBS Congress and 22nd Young Scientists’ Forum 2023","authors":"Lucy Ollett","doi":"10.1042/bio_2023_136","DOIUrl":"https://doi.org/10.1042/bio_2023_136","url":null,"abstract":"Each year, the Biochemical Society fully sponsors the attendance of two outstanding postgraduate or early career members to attend the Federation of European Biochemical Societies (FEBS) Congress and Young Scientists’ Forum (YSF). With a theme of ‘Together in bioscience for a better future’, the 47th FEBS Congress and 22nd YSF combined research presentations, general talks and practical career exercises to showcase the newest developments in biochemistry, molecular biology and related areas. Providing an incredible opportunity to make connections with European colleagues and discuss research with an international audience, we were delighted to offer International Sponsored Places to Dr Yang Li (University College London, UK) and Dr Ewa Blaszczak (Medical University of Lublin, Poland) in 2023.\u0000 Here, Yang and Ewa discuss their experiences at this year’s meetings and highlight the valuable networking and interactions developed as a result of their participation.","PeriodicalId":35334,"journal":{"name":"Biochemist","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2023-08-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"44501125","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
期刊
Biochemist
全部 Acc. Chem. Res. ACS Applied Bio Materials ACS Appl. Electron. Mater. ACS Appl. Energy Mater. ACS Appl. Mater. Interfaces ACS Appl. Nano Mater. ACS Appl. Polym. Mater. ACS BIOMATER-SCI ENG ACS Catal. ACS Cent. Sci. ACS Chem. Biol. ACS Chemical Health & Safety ACS Chem. Neurosci. ACS Comb. Sci. ACS Earth Space Chem. ACS Energy Lett. ACS Infect. Dis. ACS Macro Lett. ACS Mater. Lett. ACS Med. Chem. Lett. ACS Nano ACS Omega ACS Photonics ACS Sens. ACS Sustainable Chem. Eng. ACS Synth. Biol. Anal. Chem. BIOCHEMISTRY-US Bioconjugate Chem. BIOMACROMOLECULES Chem. Res. Toxicol. Chem. Rev. Chem. Mater. CRYST GROWTH DES ENERG FUEL Environ. Sci. Technol. Environ. Sci. Technol. Lett. Eur. J. Inorg. Chem. IND ENG CHEM RES Inorg. Chem. J. Agric. Food. Chem. J. Chem. Eng. Data J. Chem. Educ. J. Chem. Inf. Model. J. Chem. Theory Comput. J. Med. Chem. J. Nat. Prod. J PROTEOME RES J. Am. Chem. Soc. LANGMUIR MACROMOLECULES Mol. Pharmaceutics Nano Lett. Org. Lett. ORG PROCESS RES DEV ORGANOMETALLICS J. Org. Chem. J. Phys. Chem. J. Phys. Chem. A J. Phys. Chem. B J. Phys. Chem. C J. Phys. Chem. Lett. Analyst Anal. Methods Biomater. Sci. Catal. Sci. Technol. Chem. Commun. Chem. Soc. Rev. CHEM EDUC RES PRACT CRYSTENGCOMM Dalton Trans. Energy Environ. Sci. ENVIRON SCI-NANO ENVIRON SCI-PROC IMP ENVIRON SCI-WAT RES Faraday Discuss. Food Funct. Green Chem. Inorg. Chem. Front. Integr. Biol. J. Anal. At. Spectrom. J. Mater. Chem. A J. Mater. Chem. B J. Mater. Chem. C Lab Chip Mater. Chem. Front. Mater. Horiz. MEDCHEMCOMM Metallomics Mol. Biosyst. Mol. Syst. Des. Eng. Nanoscale Nanoscale Horiz. Nat. Prod. Rep. New J. Chem. Org. Biomol. Chem. Org. Chem. Front. PHOTOCH PHOTOBIO SCI PCCP Polym. Chem.
×
引用
GB/T 7714-2015
复制
MLA
复制
APA
复制
导出至
BibTeX EndNote RefMan NoteFirst NoteExpress
×
0
微信
客服QQ
Book学术公众号 扫码关注我们
反馈
×
意见反馈
请填写您的意见或建议
请填写您的手机或邮箱
×
提示
您的信息不完整,为了账户安全,请先补充。
现在去补充
×
提示
您因"违规操作"
具体请查看互助需知
我知道了
×
提示
现在去查看 取消
×
提示
确定
Book学术官方微信
Book学术文献互助
Book学术文献互助群
群 号:481959085
Book学术
文献互助 智能选刊 最新文献 互助须知 联系我们:info@booksci.cn
Book学术提供免费学术资源搜索服务,方便国内外学者检索中英文文献。致力于提供最便捷和优质的服务体验。
Copyright © 2023 Book学术 All rights reserved.
ghs 京公网安备 11010802042870号 京ICP备2023020795号-1