{"title":"使用 CRISPR/Cas9 系统纠正基因突变的好处","authors":"","doi":"10.3103/s0891416823030084","DOIUrl":null,"url":null,"abstract":"<h3>Abstract</h3> <p>The discovery of the CRISPR/Cas9 gene scissors technology was a breakthrough in the development of gene therapy methods and attracted the attention of the entire scientific community. This technology provides prospects for the treatment of many neurodegenerative diseases and cancers that were previously considered almost incurable. To date, the mechanisms of the development and progression of these groups of nosologies are becoming increasingly studied due to discoveries in the field of molecular biology and genetics. However, the application of the CRISPR/Cas9 technology has some limitations and difficulties that should be considered. The development of works on the creation and selection of specific gene carriers, a thorough safety assessment of modern genetic manipulations, and the integrated participation of specialists from different fields of science will be important to solve the problems associated with the use of the CRISPR/Cas9 technology and achieve the desired therapeutic effect.</p>","PeriodicalId":19005,"journal":{"name":"Molecular Genetics, Microbiology and Virology","volume":"30 1","pages":""},"PeriodicalIF":0.4000,"publicationDate":"2023-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Benefits of Using the CRISPR/Cas9 System for the Correction of Genetic Mutations\",\"authors\":\"\",\"doi\":\"10.3103/s0891416823030084\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<h3>Abstract</h3> <p>The discovery of the CRISPR/Cas9 gene scissors technology was a breakthrough in the development of gene therapy methods and attracted the attention of the entire scientific community. This technology provides prospects for the treatment of many neurodegenerative diseases and cancers that were previously considered almost incurable. To date, the mechanisms of the development and progression of these groups of nosologies are becoming increasingly studied due to discoveries in the field of molecular biology and genetics. However, the application of the CRISPR/Cas9 technology has some limitations and difficulties that should be considered. The development of works on the creation and selection of specific gene carriers, a thorough safety assessment of modern genetic manipulations, and the integrated participation of specialists from different fields of science will be important to solve the problems associated with the use of the CRISPR/Cas9 technology and achieve the desired therapeutic effect.</p>\",\"PeriodicalId\":19005,\"journal\":{\"name\":\"Molecular Genetics, Microbiology and Virology\",\"volume\":\"30 1\",\"pages\":\"\"},\"PeriodicalIF\":0.4000,\"publicationDate\":\"2023-09-01\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Molecular Genetics, Microbiology and Virology\",\"FirstCategoryId\":\"99\",\"ListUrlMain\":\"https://doi.org/10.3103/s0891416823030084\",\"RegionNum\":4,\"RegionCategory\":\"生物学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q4\",\"JCRName\":\"BIOCHEMISTRY & MOLECULAR BIOLOGY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Molecular Genetics, Microbiology and Virology","FirstCategoryId":"99","ListUrlMain":"https://doi.org/10.3103/s0891416823030084","RegionNum":4,"RegionCategory":"生物学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q4","JCRName":"BIOCHEMISTRY & MOLECULAR BIOLOGY","Score":null,"Total":0}
Benefits of Using the CRISPR/Cas9 System for the Correction of Genetic Mutations
Abstract
The discovery of the CRISPR/Cas9 gene scissors technology was a breakthrough in the development of gene therapy methods and attracted the attention of the entire scientific community. This technology provides prospects for the treatment of many neurodegenerative diseases and cancers that were previously considered almost incurable. To date, the mechanisms of the development and progression of these groups of nosologies are becoming increasingly studied due to discoveries in the field of molecular biology and genetics. However, the application of the CRISPR/Cas9 technology has some limitations and difficulties that should be considered. The development of works on the creation and selection of specific gene carriers, a thorough safety assessment of modern genetic manipulations, and the integrated participation of specialists from different fields of science will be important to solve the problems associated with the use of the CRISPR/Cas9 technology and achieve the desired therapeutic effect.
期刊介绍:
Molecular Genetics, Microbiology and Virology is a journal that covers most topical theoretical and applied problems of molecular genetics of pro- and eukaryotic organisms, molecular microbiology and molecular virology. An important part the journal assigns to investigations of the genetic apparatus of microorganisms, searching for forms of genetic exchange, genetic mapping of pathogenic causative agents, to ascertainment of the structure and functions of extrachromosomal factors of heredity and migratory genetic elements, to theoretical studies into the mechanisms of genetic regulation. The journal publishes results of research on molecular and genetic bases of an eukaryotic cell, functioning of chromosomes and chromatin, nature of genetic changes in malignization and a set of hereditary diseases. On the pages of the journal there is covered the formulation of molecular bases of virology including issues of integration of viral and cellular genomes, and issues of persistence. The journal plans to put materials on genetic engineering, envisaging synthesis and isolation of genes from natural reservoirs, creation of plasmid- and virus-based vector, production of recombinant DNA molecules, the creation of Gene Banks for Microbes, animals, and human; and also on biotechnological production of hormones, components of antiviral vaccines, diagnostic and therapeutic preparations.