{"title":"[Gene therapy for hemophilia].","authors":"Yoshitaka Miyakawa","doi":"10.11406/rinketsu.66.465","DOIUrl":null,"url":null,"abstract":"<p><p>Gene therapies for hemophilia A and B were approved in the US and EU in 2022. A single infusion of adeno-associated virus vector containing FVIII and FIX gene increases clotting factor levels within a few weeks. Around 90% of hemophilia patients treated with gene therapy no longer need clotting factor injections and show a reduced annual bleeding rate. Gene therapy vectors for hemophilia have strong liver tropism and use liver-specific promoters. This review discusses the history of hemophilia gene therapy, phase 3 trials, and unmet needs.</p>","PeriodicalId":93844,"journal":{"name":"[Rinsho ketsueki] The Japanese journal of clinical hematology","volume":"66 6","pages":"465-472"},"PeriodicalIF":0.0000,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"[Rinsho ketsueki] The Japanese journal of clinical hematology","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.11406/rinketsu.66.465","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"","JCRName":"","Score":null,"Total":0}
引用次数: 0
Abstract
Gene therapies for hemophilia A and B were approved in the US and EU in 2022. A single infusion of adeno-associated virus vector containing FVIII and FIX gene increases clotting factor levels within a few weeks. Around 90% of hemophilia patients treated with gene therapy no longer need clotting factor injections and show a reduced annual bleeding rate. Gene therapy vectors for hemophilia have strong liver tropism and use liver-specific promoters. This review discusses the history of hemophilia gene therapy, phase 3 trials, and unmet needs.