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Prophylaxis with factor replacement has high efficacy in reducing bleeding but is limited by the need for frequent intravenous infusion and fluctuations in haemostasis between doses. Additional prophylaxis therapies are being developed which may overcome some of the current treatment barriers. Gene therapy (GT) is being developed to provide a functional cure such that there is sustained factor expression and minimal to no need for additional haemostatic therapy. There are now two approved gene therapies for haemophilia which may be transformative for many individuals. Benefits of GT should go beyond increasing factor activity and reducing bleeding as persons with haemophilia aim to achieve a \u2018haemophilia\u2010free mind\u2019 and health equity with optimal health and well\u2010being.<\/jats:p>","DOI":"10.1111\/jvh.13882","type":"journal-article","created":{"date-parts":[[2024,4,12]],"date-time":"2024-04-12T08:44:21Z","timestamp":1712911461000},"page":"4-8","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":6,"title":["The benefits of gene therapy in people with haemophilia"],"prefix":"10.1111","volume":"31","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-5665-8958","authenticated-orcid":false,"given":"Courtney D.","family":"Thornburg","sequence":"first","affiliation":[{"name":"Hemophilia and Thrombosis Treatment Center Rady Children's Hospital San Diego  San Diego California USA"},{"name":"Department of Pediatrics, Division of Hematology\/Oncology UC San Diego School of Medicine  La Jolla California USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2024,4,12]]},"reference":[{"key":"e_1_2_10_2_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.13862"},{"key":"e_1_2_10_3_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa2209226"},{"key":"e_1_2_10_4_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2018-08-872291"},{"key":"e_1_2_10_5_1","doi-asserted-by":"publisher","DOI":"10.1080\/14740338.2021.1893303"},{"key":"e_1_2_10_6_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood.2020009217"},{"key":"e_1_2_10_7_1","doi-asserted-by":"publisher","DOI":"10.1182\/bloodadvances.2021006403"},{"key":"e_1_2_10_8_1","doi-asserted-by":"publisher","DOI":"10.1146\/annurev-med-043021-033013"},{"key":"e_1_2_10_9_1","doi-asserted-by":"publisher","DOI":"10.1182\/hematology.2022000388"},{"key":"e_1_2_10_10_1","doi-asserted-by":"publisher","DOI":"10.1080\/14712598.2022.2002842"},{"key":"e_1_2_10_11_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa2113708"},{"key":"e_1_2_10_12_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.14391"},{"key":"e_1_2_10_13_1","doi-asserted-by":"publisher","DOI":"10.1182\/bloodadvances.2022008886"},{"key":"e_1_2_10_14_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa2211075"},{"key":"e_1_2_10_15_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa2211644"},{"key":"e_1_2_10_16_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.14807"},{"key":"e_1_2_10_17_1","doi-asserted-by":"publisher","DOI":"10.1002\/rth2.12567"},{"key":"e_1_2_10_18_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.13504"},{"key":"e_1_2_10_19_1","doi-asserted-by":"publisher","DOI":"10.1111\/jth.15023"},{"key":"e_1_2_10_20_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.14015"},{"key":"e_1_2_10_21_1","doi-asserted-by":"publisher","DOI":"10.1007\/s11136-011-9903-x"},{"key":"e_1_2_10_22_1","doi-asserted-by":"publisher","DOI":"10.1186\/s40814-018-0253-0"},{"key":"e_1_2_10_23_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.14190"},{"key":"e_1_2_10_24_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.14401"},{"key":"e_1_2_10_25_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.14383"},{"key":"e_1_2_10_26_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.14572"},{"key":"e_1_2_10_27_1","doi-asserted-by":"publisher","DOI":"10.2147\/PPA.S406894"},{"key":"e_1_2_10_28_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.14378"},{"key":"e_1_2_10_29_1","doi-asserted-by":"publisher","DOI":"10.1186\/s13023-022-02256-2"},{"key":"e_1_2_10_30_1","volume-title":"Roctaviain, INN\u2010valoctocogene roxaparvovec. 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