{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,8]],"date-time":"2026-09-08T23:38:19Z","timestamp":1788910699267,"version":"build-2803163510"},"reference-count":77,"publisher":"MDPI AG","issue":"13","license":[{"start":{"date-parts":[[2023,6,28]],"date-time":"2023-06-28T00:00:00Z","timestamp":1687910400000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/501100006769","name":"Russian Science Foundation","doi-asserted-by":"publisher","award":["23-64-00002"],"award-info":[{"award-number":["23-64-00002"]}],"id":[{"id":"10.13039\/501100006769","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["IJMS"],"abstract":"<jats:p>In contrast to the standard enzyme-replacement therapy, administered from once per 7\u201314 days to 2\u20133 times a week in patients with severe hemophilia B, as a result of a single injection, gene therapy can restore F9 gene expression and maintain it for a prolonged time. In clinical research, the approach of delivering a functional copy of a gene using adeno-associated viral (AAV) vectors is widely used. The scientific community is actively researching possible modifications to improve delivery efficiency and expression. In preclinical studies, the possibility of genome editing using CRISPR\/Cas9 technology for the treatment of hemophilia B is also being actively studied.<\/jats:p>","DOI":"10.3390\/ijms241310766","type":"journal-article","created":{"date-parts":[[2023,6,29]],"date-time":"2023-06-29T00:41:45Z","timestamp":1687999305000},"page":"10766","update-policy":"https:\/\/doi.org\/10.3390\/mdpi_crossmark_policy","source":"Crossref","is-referenced-by-count":28,"title":["Gene Therapy Approaches for the Treatment of Hemophilia B"],"prefix":"10.3390","volume":"24","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-9528-1575","authenticated-orcid":false,"given":"Anastasiia B.","family":"Soroka","sequence":"first","affiliation":[{"name":"Life Sciences Research Center, Moscow Institute of Physics and Technology, National Research University, 141700 Dolgoprudniy, Russia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-7608-439X","authenticated-orcid":false,"given":"Sofya G.","family":"Feoktistova","sequence":"additional","affiliation":[{"name":"Life Sciences Research Center, Moscow Institute of Physics and Technology, National Research University, 141700 Dolgoprudniy, Russia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Olga N.","family":"Mityaeva","sequence":"additional","affiliation":[{"name":"Life Sciences Research Center, Moscow Institute of Physics and Technology, National Research University, 141700 Dolgoprudniy, Russia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-9794-6297","authenticated-orcid":false,"given":"Pavel Y.","family":"Volchkov","sequence":"additional","affiliation":[{"name":"Life Sciences Research Center, Moscow Institute of Physics and Technology, National Research University, 141700 Dolgoprudniy, Russia"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"1968","published-online":{"date-parts":[[2023,6,28]]},"reference":[{"key":"ref_1","doi-asserted-by":"crossref","first-page":"1184","DOI":"10.1111\/jth.12958","article-title":"Hemophilia B: Molecular Pathogenesis and Mutation Analysis","volume":"13","author":"Goodeve","year":"2015","journal-title":"J. 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