{"title":"Therapeutic applications of cell engineering using mRNA technology.","authors":"Yujia He, Angus P R Johnston, Colin W Pouton","doi":"10.1016/j.tibtech.2024.07.012","DOIUrl":null,"url":null,"abstract":"<p><p>Engineering and reprogramming cells has significant therapeutic potential to treat a wide range of diseases, by replacing missing or defective proteins, to provide transcription factors (TFs) to reprogram cell phenotypes, or to provide enzymes such as RNA-guided Cas9 derivatives for CRISPR-based cell engineering. While viral vector-mediated gene transfer has played an important role in this field, the use of mRNA avoids safety concerns associated with the integration of DNA into the host cell genome, making mRNA particularly attractive for in vivo applications. Widespread application of mRNA for cell engineering is limited by its instability in the biological environment and challenges involved in the delivery of mRNA to its target site. In this review, we examine challenges that must be overcome to develop effective therapeutics.</p>","PeriodicalId":23324,"journal":{"name":"Trends in biotechnology","volume":null,"pages":null},"PeriodicalIF":14.3000,"publicationDate":"2024-08-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Trends in biotechnology","FirstCategoryId":"5","ListUrlMain":"https://doi.org/10.1016/j.tibtech.2024.07.012","RegionNum":1,"RegionCategory":"工程技术","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"BIOTECHNOLOGY & APPLIED MICROBIOLOGY","Score":null,"Total":0}
引用次数: 0
Abstract
Engineering and reprogramming cells has significant therapeutic potential to treat a wide range of diseases, by replacing missing or defective proteins, to provide transcription factors (TFs) to reprogram cell phenotypes, or to provide enzymes such as RNA-guided Cas9 derivatives for CRISPR-based cell engineering. While viral vector-mediated gene transfer has played an important role in this field, the use of mRNA avoids safety concerns associated with the integration of DNA into the host cell genome, making mRNA particularly attractive for in vivo applications. Widespread application of mRNA for cell engineering is limited by its instability in the biological environment and challenges involved in the delivery of mRNA to its target site. In this review, we examine challenges that must be overcome to develop effective therapeutics.
期刊介绍：
Trends in Biotechnology publishes reviews and perspectives on the applied biological sciences, focusing on useful science applied to, derived from, or inspired by living systems.
The major themes that TIBTECH is interested in include:
Bioprocessing (biochemical engineering, applied enzymology, industrial biotechnology, biofuels, metabolic engineering)
Omics (genome editing, single-cell technologies, bioinformatics, synthetic biology)
Materials and devices (bionanotechnology, biomaterials, diagnostics/imaging/detection, soft robotics, biosensors/bioelectronics)
Therapeutics (biofabrication, stem cells, tissue engineering and regenerative medicine, antibodies and other protein drugs, drug delivery)
Agroenvironment (environmental engineering, bioremediation, genetically modified crops, sustainable development).
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