Biochemist最新文献_第2页

Meeting Report 会议报告

Biochemist Pub Date : 2023-09-07 DOI: 10.1042/bio_2023_138

Daniel R. Dries

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

Biochemist Pub Date : 2023-08-24 DOI: 10.1042/bio_2023_127

Rodanthi Lyraki

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Gene therapy: yesterday, today and tomorrow 基因治疗:昨天，今天和明天

Biochemist Pub Date : 2023-08-24 DOI: 10.1042/bio_2023_137

Heather Doran

{"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":"66 1","pages":"0"},"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

Biochemist Pub Date : 2023-08-23 DOI: 10.1042/bio_2023_129

A. Beqqali, A. Baker

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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？

Biochemist Pub Date : 2023-08-21 DOI: 10.1042/bio_2023_131

M. Pearson, Rafael J. Yáñez-Muñoz

{"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":" ","pages":""},"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

Biochemist Pub Date : 2023-08-14 DOI: 10.1042/bio_2023_133

Daniel Humphreys, Sarah Coulthurst

{"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":"591 1","pages":"0"},"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

Biochemist Pub Date : 2023-08-14 DOI: 10.1042/bio_2023_134

Aga Gambus, Matthias Soller, Ferenc Mueller, Constanze Bonifer

{"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":"1 1","pages":"0"},"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

Biochemist Pub Date : 2023-08-14 DOI: 10.1042/bio_2023_128

B. Şeker Yılmaz, P. Gissen

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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

Biochemist Pub Date : 2023-08-10 DOI: 10.1042/bio_2023_130

Calum Upton

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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

Biochemist Pub Date : 2023-08-10 DOI: 10.1042/bio_2023_136

Lucy Ollett

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