{"title":"基因治疗:一项杰出的疾病治疗技术","authors":"Nayade Sarmiento, S. Tinoco","doi":"10.21931/RB/CS/2019.02.01.23","DOIUrl":null,"url":null,"abstract":"Nearly three decades ago, the first clinical trial based on gene therapy revolutionized the scientific field by demonstrating that it was possible to genetically modify harmful or defective cells and therapeutically improve patient conditions. Since then, this technique has been implemented for the study and treatment of different diseases, many of them fatal, with the hope of achieving a \"cure\". Several genetic editing tools have been developed and continue to be studied to improve the effectiveness of gene therapy, in addition to a more exhaustive analysis for choosing the type of vector, which is the main cause of adverse effects. In this review, we present characteristics of the gene therapy mechanism along with the types of vectors that are used in this procedure, followed by the most important applications in the medical field and briefly we describe some limitations and prospects to study in the future.","PeriodicalId":214615,"journal":{"name":"Humboldt kolleg Ibarra 2019","volume":"4 1","pages":"0"},"PeriodicalIF":0.0000,"publicationDate":"2019-02-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Gene Therapy: An Outstanding Technique For Diseases\",\"authors\":\"Nayade Sarmiento, S. Tinoco\",\"doi\":\"10.21931/RB/CS/2019.02.01.23\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"Nearly three decades ago, the first clinical trial based on gene therapy revolutionized the scientific field by demonstrating that it was possible to genetically modify harmful or defective cells and therapeutically improve patient conditions. Since then, this technique has been implemented for the study and treatment of different diseases, many of them fatal, with the hope of achieving a \\\"cure\\\". Several genetic editing tools have been developed and continue to be studied to improve the effectiveness of gene therapy, in addition to a more exhaustive analysis for choosing the type of vector, which is the main cause of adverse effects. In this review, we present characteristics of the gene therapy mechanism along with the types of vectors that are used in this procedure, followed by the most important applications in the medical field and briefly we describe some limitations and prospects to study in the future.\",\"PeriodicalId\":214615,\"journal\":{\"name\":\"Humboldt kolleg Ibarra 2019\",\"volume\":\"4 1\",\"pages\":\"0\"},\"PeriodicalIF\":0.0000,\"publicationDate\":\"2019-02-19\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Humboldt kolleg Ibarra 2019\",\"FirstCategoryId\":\"1085\",\"ListUrlMain\":\"https://doi.org/10.21931/RB/CS/2019.02.01.23\",\"RegionNum\":0,\"RegionCategory\":null,\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"\",\"JCRName\":\"\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Humboldt kolleg Ibarra 2019","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.21931/RB/CS/2019.02.01.23","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"","JCRName":"","Score":null,"Total":0}
Gene Therapy: An Outstanding Technique For Diseases
Nearly three decades ago, the first clinical trial based on gene therapy revolutionized the scientific field by demonstrating that it was possible to genetically modify harmful or defective cells and therapeutically improve patient conditions. Since then, this technique has been implemented for the study and treatment of different diseases, many of them fatal, with the hope of achieving a "cure". Several genetic editing tools have been developed and continue to be studied to improve the effectiveness of gene therapy, in addition to a more exhaustive analysis for choosing the type of vector, which is the main cause of adverse effects. In this review, we present characteristics of the gene therapy mechanism along with the types of vectors that are used in this procedure, followed by the most important applications in the medical field and briefly we describe some limitations and prospects to study in the future.