Nature Reviews. Drug Discovery最新文献_第7页

Rebalancing calcium in Alzheimer disease 重新平衡阿尔茨海默病中的钙。

IF 122.7 1区医学

Nature Reviews. Drug Discovery Pub Date : 2024-07-05 DOI: 10.1038/d41573-024-00115-2

Katie Kingwell

引用次数: 0

The 60-year evolution of lipid nanoparticles for nucleic acid delivery 用于核酸递送的脂质纳米颗粒的 60 年发展历程。

IF 122.7 1区医学

Nature Reviews. Drug Discovery Pub Date : 2024-07-04 DOI: 10.1038/s41573-024-00977-6

P. R. Cullis, P. L. Felgner

{"title":"The 60-year evolution of lipid nanoparticles for nucleic acid delivery","authors":"P. R. Cullis, P. L. Felgner","doi":"10.1038/s41573-024-00977-6","DOIUrl":"10.1038/s41573-024-00977-6","url":null,"abstract":"Delivery of genetic information to the interior of target cells in vivo has been a major challenge facing gene therapies. This barrier is now being overcome, owing in part to dramatic advances made by lipid-based systems that have led to lipid nanoparticles (LNPs) that enable delivery of nucleic acid-based vaccines and therapeutics. Examples include the clinically approved COVID-19 LNP mRNA vaccines and Onpattro (patisiran), an LNP small interfering RNA therapeutic to treat transthyretin-induced amyloidosis (hATTR). In addition, a host of promising LNP-enabled vaccines and gene therapies are in clinical development. Here, we trace this success to two streams of research conducted over the past 60 years: the discovery of the transfection properties of lipoplexes composed of positively charged cationic lipids complexed with nucleic acid cargos and the development of lipid nanoparticles using ionizable cationic lipids. The fundamental insights gained from these two streams of research offer potential delivery solutions for most forms of gene therapies. Lipid nanoparticle-based systems are increasingly being utilized for the delivery of nucleic acid-based vaccines and therapeutics. In this Perspective, Cullis and Felgner trace the evolution of these systems over the past 60 years and discuss future prospects for LNP-enabled gene therapies.","PeriodicalId":19068,"journal":{"name":"Nature Reviews. Drug Discovery","volume":null,"pages":null},"PeriodicalIF":122.7,"publicationDate":"2024-07-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141534881","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Enhancing in situ cancer vaccines using delivery technologies 利用输送技术增强原位癌症疫苗。

IF 122.7 1区医学

Nature Reviews. Drug Discovery Pub Date : 2024-07-01 DOI: 10.1038/s41573-024-00974-9

Ningqiang Gong, Mohamad-Gabriel Alameh, Rakan El-Mayta, Lulu Xue, Drew Weissman, Michael J. Mitchell

{"title":"Enhancing in situ cancer vaccines using delivery technologies","authors":"Ningqiang Gong, Mohamad-Gabriel Alameh, Rakan El-Mayta, Lulu Xue, Drew Weissman, Michael J. Mitchell","doi":"10.1038/s41573-024-00974-9","DOIUrl":"10.1038/s41573-024-00974-9","url":null,"abstract":"In situ cancer vaccination refers to any approach that exploits tumour antigens available at a tumour site to induce tumour-specific adaptive immune responses. These approaches hold great promise for the treatment of many solid tumours, with numerous candidate drugs under preclinical or clinical evaluation and several products already approved. However, there are challenges in the development of effective in situ cancer vaccines. For example, inadequate release of tumour antigens from tumour cells limits antigen uptake by immune cells; insufficient antigen processing by antigen-presenting cells restricts the generation of antigen-specific T cell responses; and the suppressive immune microenvironment of the tumour leads to exhaustion and death of effector cells. Rationally designed delivery technologies such as lipid nanoparticles, hydrogels, scaffolds and polymeric nanoparticles are uniquely suited to overcome these challenges through the targeted delivery of therapeutics to tumour cells, immune cells or the extracellular matrix. Here, we discuss delivery technologies that have the potential to reduce various clinical barriers for in situ cancer vaccines. We also provide our perspective on this emerging field that lies at the interface of cancer vaccine biology and delivery technologies. In situ cancer vaccines generate tumour-specific T cell responses and hold promise for the treatment of solid tumours. This Review discusses drug delivery technologies such as lipid nanoparticles, hydrogels and scaffolds that are being developed to boost the antitumour immune effects of several types of in situ cancer vaccine.","PeriodicalId":19068,"journal":{"name":"Nature Reviews. Drug Discovery","volume":null,"pages":null},"PeriodicalIF":122.7,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141477027","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

A selective microbiome-sparing antibiotic 一种选择性微生物保护抗生素

IF 122.7 1区医学

Nature Reviews. Drug Discovery Pub Date : 2024-06-27 DOI: 10.1038/d41573-024-00113-4

Sarah Crunkhorn

引用次数: 0

ETS2 drives IBD ETS2 驱动 IBD

IF 122.7 1区医学

Nature Reviews. Drug Discovery Pub Date : 2024-06-27 DOI: 10.1038/d41573-024-00110-7

Sarah Crunkhorn

引用次数: 0

When chronic and rare disease worlds converge 当慢性病和罕见病的世界交汇在一起

IF 122.7 1区医学

Nature Reviews. Drug Discovery Pub Date : 2024-06-27 DOI: 10.1038/d41573-024-00114-3

Asher Mullard

引用次数: 0

Machine learning identifies AMPs 机器学习识别 AMP

IF 122.7 1区医学

Nature Reviews. Drug Discovery Pub Date : 2024-06-27 DOI: 10.1038/d41573-024-00111-6

Sarah Crunkhorn

引用次数: 0

Discovering CFTR modulators 发现 CFTR 调节剂

IF 122.7 1区医学

Nature Reviews. Drug Discovery Pub Date : 2024-06-27 DOI: 10.1038/d41573-024-00112-5

Sarah Crunkhorn

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Gene therapies start the climb from rare disease base camps to common disease peaks 基因疗法开始从罕见病大本营向常见病高峰攀登

IF 122.7 1区医学

Nature Reviews. Drug Discovery Pub Date : 2024-06-25 DOI: 10.1038/d41573-024-00109-0

Asher Mullard

引用次数: 0

FDA approves 21-valent pneumococcal vaccine 美国食品和药物管理局批准 21 价肺炎球菌疫苗

IF 122.7 1区医学

Nature Reviews. Drug Discovery Pub Date : 2024-06-21 DOI: 10.1038/d41573-024-00108-1

Asher Mullard

引用次数: 0