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Drug discovery today. Therapeutic strategies最新文献

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Developing companion diagnostics for delivering personalised medicine: opportunities and challenges 开发伴随诊断以提供个体化药物:机遇与挑战
Drug discovery today. Therapeutic strategies Pub Date : 2013-12-01 Epub Date: 2013-07-11 DOI: 10.1016/j.ddstr.2013.05.002
Frank Desiere, Thorsten S. Gutjahr, Ulrich-Peter Rohr
{"title":"Developing companion diagnostics for delivering personalised medicine: opportunities and challenges","authors":"Frank Desiere,&nbsp;Thorsten S. Gutjahr,&nbsp;Ulrich-Peter Rohr","doi":"10.1016/j.ddstr.2013.05.002","DOIUrl":"10.1016/j.ddstr.2013.05.002","url":null,"abstract":"<div><p>A greater understanding of disease biology processes, the opportunity to develop targeted drugs leading to further improved patient outcomes and technology advances, as well as increasing pressure on healthcare budgets have led to a shift towards personalised healthcare solutions. Personalised medicine has tremendous potential benefits for patients and healthcare providers, as well as for regulatory agencies and pharmaceutical and diagnostic companies, but the advancement of this innovative therapeutic strategy depends on identifying biomarkers functioning as companion diagnostics for the targeted drug. However, considerable practical, methodological, regulatory and economic issues must be addressed to fully realise the potential of this approach.</p></div>","PeriodicalId":87599,"journal":{"name":"Drug discovery today. Therapeutic strategies","volume":"10 4","pages":"Pages e175-e181"},"PeriodicalIF":0.0,"publicationDate":"2013-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1016/j.ddstr.2013.05.002","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"77984500","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: 3
Key issues affecting the development and implementation of personalised medicine: a foresight exercise 影响个体化医疗发展和实施的关键问题:一项前瞻性工作
Drug discovery today. Therapeutic strategies Pub Date : 2013-12-01 Epub Date: 2015-03-20 DOI: 10.1016/j.ddstr.2015.01.001
Kirsten Steinhausen , Stephane Berghmans
{"title":"Key issues affecting the development and implementation of personalised medicine: a foresight exercise","authors":"Kirsten Steinhausen ,&nbsp;Stephane Berghmans","doi":"10.1016/j.ddstr.2015.01.001","DOIUrl":"10.1016/j.ddstr.2015.01.001","url":null,"abstract":"<div><p><span>A foresight exercise was undertaken by the European Science Foundation to identify the main challenges in the development and implementation of personalised medicine across Europe. A multi- and interdisciplinary group of key stakeholders, including patient groups, regulators, industry and academics, were consulted and invited to discuss and propose recommendations related to personalised medicine in four core areas: data handling; the introduction of new models and decision-making processes (from safety assessment to reimbursement procedures); the enhancement of interdisciplinarity and stakeholder participation; and the need for new infrastructure and funding resources. The results of these discussions were recently published in the Report ‘Personalised Medicine for the European Citizen: Towards more precise medicine for the diagnosis, treatment and prevention of disease (iPM)’ [</span><span>1</span>]. The aim of this review is to summarise and discuss the key messages of this report.</p></div>","PeriodicalId":87599,"journal":{"name":"Drug discovery today. Therapeutic strategies","volume":"10 4","pages":"Pages e189-e194"},"PeriodicalIF":0.0,"publicationDate":"2013-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1016/j.ddstr.2015.01.001","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"74748350","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: 2
Ethical issues in personalized medicine 个体化医疗中的伦理问题
Drug discovery today. Therapeutic strategies Pub Date : 2013-12-01 Epub Date: 2013-06-20 DOI: 10.1016/j.ddstr.2013.05.001
Ruth Chadwick
{"title":"Ethical issues in personalized medicine","authors":"Ruth Chadwick","doi":"10.1016/j.ddstr.2013.05.001","DOIUrl":"10.1016/j.ddstr.2013.05.001","url":null,"abstract":"<div><p>The search for personalized medicine is not a new phenomenon, but it is only with developments in genetic<span> knowledge – and pharmacogenomics in particular – that the ‘personalization project’ stands a realistic chance of improving health on a wide scale. Despite recent advances, the risk of hype is still present, as can be seen by expectations and claims surrounding direct-to-consumer genetic testing. The move towards personalized medicine may disadvantage some patients in some social contexts, by shifting the allocation of resources in the health care system. Risks also surround the increasing accumulation of health data that goes hand in hand with personalized medicine: a study has been able to identify individual research participants from stored genetic data.</span></p></div>","PeriodicalId":87599,"journal":{"name":"Drug discovery today. Therapeutic strategies","volume":"10 4","pages":"Pages e171-e174"},"PeriodicalIF":0.0,"publicationDate":"2013-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1016/j.ddstr.2013.05.001","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"81531903","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: 11
Personalized medicine in context: social science perspectives 情境下的个体化医疗:社会科学视角
Drug discovery today. Therapeutic strategies Pub Date : 2013-12-01 Epub Date: 2013-07-11 DOI: 10.1016/j.ddstr.2013.05.003
Richard Tutton , Kimberly Jamie
{"title":"Personalized medicine in context: social science perspectives","authors":"Richard Tutton ,&nbsp;Kimberly Jamie","doi":"10.1016/j.ddstr.2013.05.003","DOIUrl":"10.1016/j.ddstr.2013.05.003","url":null,"abstract":"<div><p>In the 1990s, the scientific and popular press heralded the emergence of a new paradigm in drug discovery<span> and development called pharmacogenomics<span> (pgmx). As well as capturing the interest of scientists, policymakers and journalists, the field of personalized medicine has also been of immense interest to social scientists who research new innovations in health and biomedicine. This article reviews existing social science research on pgmx. It considers work on mapping industry involvement in pgmx; the dynamics of clinical adoption and the challenges of pgmx testing becoming a standard healthcare service; and patient and public perspectives on pgmx. In conclusion, the article reflects on the future research agenda.</span></span></p></div>","PeriodicalId":87599,"journal":{"name":"Drug discovery today. Therapeutic strategies","volume":"10 4","pages":"Pages e183-e187"},"PeriodicalIF":0.0,"publicationDate":"2013-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1016/j.ddstr.2013.05.003","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"72660113","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: 14
Antisense therapies for cancer 癌症的反义疗法:弥合药物基因组学的鸿沟
Drug discovery today. Therapeutic strategies Pub Date : 2013-09-01 Epub Date: 2014-06-21 DOI: 10.1016/j.ddstr.2013.04.002
A. Robert MacLeod
{"title":"Antisense therapies for cancer","authors":"A. Robert MacLeod","doi":"10.1016/j.ddstr.2013.04.002","DOIUrl":"10.1016/j.ddstr.2013.04.002","url":null,"abstract":"<div><p><span><span>By contrast to other therapeutic approaches, the druggable universe is not limited with antisense technology as these inhibitors can be rationally designed based on sequence information alone. Recent clinical data have demonstrated proof of mechanism and clinical benefit for antisense drugs in many disease areas including cancer. Together with recent advances in antisense chemistry these findings suggest that this technology is now poised to emerge as a key therapeutic modality to bridge the ever growing </span>pharmacogenomic divide in cancer </span>drug discovery.</p></div>","PeriodicalId":87599,"journal":{"name":"Drug discovery today. Therapeutic strategies","volume":"10 3","pages":"Pages e157-e163"},"PeriodicalIF":0.0,"publicationDate":"2013-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1016/j.ddstr.2013.04.002","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"74510116","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: 5
Antisense modulation of RNA processing as a therapeutic approach in cancer therapy RNA加工的反义调控在癌症治疗中的应用
Drug discovery today. Therapeutic strategies Pub Date : 2013-09-01 Epub Date: 2013-09-04 DOI: 10.1016/j.ddstr.2013.06.002
Lee Spraggon, Luca Cartegni
{"title":"Antisense modulation of RNA processing as a therapeutic approach in cancer therapy","authors":"Lee Spraggon,&nbsp;Luca Cartegni","doi":"10.1016/j.ddstr.2013.06.002","DOIUrl":"10.1016/j.ddstr.2013.06.002","url":null,"abstract":"<div><p><span>Next-generation antisense technologies are re-emerging as viable and powerful approaches to the treatment of several genetic diseases. Similar strategies are also being applied to cancer therapy. Reprogramming of the expression of endogenous oncogenic products to replace them with functional antagonists, by interfering with alternative splicing (AS) or </span>polyadenylation, provides a promising novel approach to address acquired drug resistance and previously undruggable targets.</p></div>","PeriodicalId":87599,"journal":{"name":"Drug discovery today. Therapeutic strategies","volume":"10 3","pages":"Pages e139-e148"},"PeriodicalIF":0.0,"publicationDate":"2013-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1016/j.ddstr.2013.06.002","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"32976664","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: 11
Natural antisense transcripts as therapeutic targets 天然反义转录物作为治疗靶点
Drug discovery today. Therapeutic strategies Pub Date : 2013-09-01 Epub Date: 2013-05-10 DOI: 10.1016/j.ddstr.2013.03.001
Paul Halley , Olga Khorkova , Claes Wahlestedt
{"title":"Natural antisense transcripts as therapeutic targets","authors":"Paul Halley ,&nbsp;Olga Khorkova ,&nbsp;Claes Wahlestedt","doi":"10.1016/j.ddstr.2013.03.001","DOIUrl":"10.1016/j.ddstr.2013.03.001","url":null,"abstract":"","PeriodicalId":87599,"journal":{"name":"Drug discovery today. Therapeutic strategies","volume":"10 3","pages":"Pages e119-e125"},"PeriodicalIF":0.0,"publicationDate":"2013-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1016/j.ddstr.2013.03.001","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"32968075","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: 15
Antisense oligonucleotides as personalized medicine for Duchenne muscular dystrophy 反义寡核苷酸作为治疗杜氏肌营养不良症的个体化药物
Drug discovery today. Therapeutic strategies Pub Date : 2013-09-01 Epub Date: 2013-05-24 DOI: 10.1016/j.ddstr.2013.04.001
Judith C. van Deutekom, Sjef J. de Kimpe, Giles V. Campion
{"title":"Antisense oligonucleotides as personalized medicine for Duchenne muscular dystrophy","authors":"Judith C. van Deutekom,&nbsp;Sjef J. de Kimpe,&nbsp;Giles V. Campion","doi":"10.1016/j.ddstr.2013.04.001","DOIUrl":"10.1016/j.ddstr.2013.04.001","url":null,"abstract":"<div><p>In recent years, the use of antisense oligonucleotides (AONs) as RNA-modulating therapeutics has made significant progress. For the treatment of Duchenne muscular dystrophy (DMD), two chemically distinct oligonucleotides inducing specific exon 51 skipping from the DMD gene's pre-messenger RNA are currently being studied in phase II and III clinical trials. Depending on their efficacy, safety profile and approval by regulators, oligonucleotides targeting additional exons to address other mutations need to follow, through more tailor-made, shorter, development programs. This review provides an overview of the current developmental status of AONs for DMD and summarizes the challenges and discussions for such personalized mutation-specific therapeutics progressing through the development pipeline.</p></div>","PeriodicalId":87599,"journal":{"name":"Drug discovery today. Therapeutic strategies","volume":"10 3","pages":"Pages e149-e156"},"PeriodicalIF":0.0,"publicationDate":"2013-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1016/j.ddstr.2013.04.001","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"79168820","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: 9
Antisense therapies for cardiovascular/metabolic diseases 心血管/代谢疾病的反义治疗
Drug discovery today. Therapeutic strategies Pub Date : 2013-09-01 Epub Date: 2013-07-16 DOI: 10.1016/j.ddstr.2013.06.001
Richard S. Geary, Rosanne Crooke, Sanjay Bhanot, Walter Singleton
{"title":"Antisense therapies for cardiovascular/metabolic diseases","authors":"Richard S. Geary,&nbsp;Rosanne Crooke,&nbsp;Sanjay Bhanot,&nbsp;Walter Singleton","doi":"10.1016/j.ddstr.2013.06.001","DOIUrl":"10.1016/j.ddstr.2013.06.001","url":null,"abstract":"<div><p>RNA<span><span> targeted therapeutics are being developed in a broad array of therapeutic areas, and more recently a growing number of RNA targeted antisense approaches for cardiovascular and metabolic diseases have progressed into clinical development. Cardiovascular and metabolic diseases are growing health issues with significant associated morbidity and mortality. RNA represents a growing and accessible target space that has been shown to be specifically and selectively targeted utilizing short single strand antisense </span>oligonucleotides<span>. Antisense drugs are relatively small synthetic oligonucleotides with predictable safety and pharmacokinetics across a given chemical platform. Once identified, the remaining risk in development as a therapeutic is associated with selection of the right target, sufficient to affect a clinically meaningful change in the course of disease. This review focuses primarily on the translated mRNAs currently being targeted using antisense therapies in cardiovascular and metabolic disease.</span></span></p></div>","PeriodicalId":87599,"journal":{"name":"Drug discovery today. Therapeutic strategies","volume":"10 3","pages":"Pages e165-e170"},"PeriodicalIF":0.0,"publicationDate":"2013-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1016/j.ddstr.2013.06.001","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"90810816","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: 3
Clinical development of synthetic siRNA therapeutics 合成siRNA疗法的临床发展
Drug discovery today. Therapeutic strategies Pub Date : 2013-09-01 Epub Date: 2013-05-11 DOI: 10.1016/j.ddstr.2013.03.002
James D. Thompson
{"title":"Clinical development of synthetic siRNA therapeutics","authors":"James D. Thompson","doi":"10.1016/j.ddstr.2013.03.002","DOIUrl":"10.1016/j.ddstr.2013.03.002","url":null,"abstract":"<div><p>In 2001 the Tuschl laboratory reported that synthetic siRNAs<span><span><span> could be applied safely to mammalian cells to silence gene expression while avoiding induction of local and systemic innate immune responses to double-stranded RNA. Today there are 19 synthetic siRNAs in clinical studies in 14 different disease indications. Clinical activity has been demonstrated in liver, eye, lung, kidney, skin and </span>solid tumors. Synthetic </span>oligonucleotides, including siRNAs, are poised to become the next therapeutic platform in the pharmaceutical industry.</span></p></div>","PeriodicalId":87599,"journal":{"name":"Drug discovery today. Therapeutic strategies","volume":"10 3","pages":"Pages e133-e138"},"PeriodicalIF":0.0,"publicationDate":"2013-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1016/j.ddstr.2013.03.002","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"86171229","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: 8
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