{"title":"眼科基因治疗策略的现状及展望。","authors":"Julia B Krajewska, Arleta Waszczykowska","doi":"10.1007/s13353-025-00973-5","DOIUrl":null,"url":null,"abstract":"<p><p>Gene therapies have recently emerged as promising strategies for treatment of previously incurable or poorly managed diseases. These hopes are particularly high in ophthalmology, as the eye is considered exceptionally suited for gene therapy. Expansion of gene therapy products may change the clinical course of treatment and give new chances to many patients. In this review, we address treatment possibilities and delivery methods as well as challenges and uncertainties related to gene therapy. We present inherited and acquired diseases which are subject to studies within this area, summarize current trends in ocular gene therapy, and indicate the future directions.</p>","PeriodicalId":14891,"journal":{"name":"Journal of Applied Genetics","volume":" ","pages":""},"PeriodicalIF":2.0000,"publicationDate":"2025-06-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Gene therapy strategies in ophthalmology-an overview of current developments and future prospects.\",\"authors\":\"Julia B Krajewska, Arleta Waszczykowska\",\"doi\":\"10.1007/s13353-025-00973-5\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><p>Gene therapies have recently emerged as promising strategies for treatment of previously incurable or poorly managed diseases. These hopes are particularly high in ophthalmology, as the eye is considered exceptionally suited for gene therapy. Expansion of gene therapy products may change the clinical course of treatment and give new chances to many patients. In this review, we address treatment possibilities and delivery methods as well as challenges and uncertainties related to gene therapy. We present inherited and acquired diseases which are subject to studies within this area, summarize current trends in ocular gene therapy, and indicate the future directions.</p>\",\"PeriodicalId\":14891,\"journal\":{\"name\":\"Journal of Applied Genetics\",\"volume\":\" \",\"pages\":\"\"},\"PeriodicalIF\":2.0000,\"publicationDate\":\"2025-06-05\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Journal of Applied Genetics\",\"FirstCategoryId\":\"99\",\"ListUrlMain\":\"https://doi.org/10.1007/s13353-025-00973-5\",\"RegionNum\":3,\"RegionCategory\":\"生物学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q3\",\"JCRName\":\"BIOTECHNOLOGY & APPLIED MICROBIOLOGY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Journal of Applied Genetics","FirstCategoryId":"99","ListUrlMain":"https://doi.org/10.1007/s13353-025-00973-5","RegionNum":3,"RegionCategory":"生物学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q3","JCRName":"BIOTECHNOLOGY & APPLIED MICROBIOLOGY","Score":null,"Total":0}
Gene therapy strategies in ophthalmology-an overview of current developments and future prospects.
Gene therapies have recently emerged as promising strategies for treatment of previously incurable or poorly managed diseases. These hopes are particularly high in ophthalmology, as the eye is considered exceptionally suited for gene therapy. Expansion of gene therapy products may change the clinical course of treatment and give new chances to many patients. In this review, we address treatment possibilities and delivery methods as well as challenges and uncertainties related to gene therapy. We present inherited and acquired diseases which are subject to studies within this area, summarize current trends in ocular gene therapy, and indicate the future directions.
期刊介绍:
The Journal of Applied Genetics is an international journal on genetics and genomics. It publishes peer-reviewed original papers, short communications (including case reports) and review articles focused on the research of applicative aspects of plant, human, animal and microbial genetics and genomics.