{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,3,13]],"date-time":"2026-03-13T23:11:17Z","timestamp":1773443477725,"version":"3.50.1"},"reference-count":28,"publisher":"Wiley","issue":"2","license":[{"start":{"date-parts":[[2025,1,27]],"date-time":"2025-01-27T00:00:00Z","timestamp":1737936000000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"},{"start":{"date-parts":[[2025,1,27]],"date-time":"2025-01-27T00:00:00Z","timestamp":1737936000000},"content-version":"tdm","delay-in-days":0,"URL":"http:\/\/doi.wiley.com\/10.1002\/tdm_license_1.1"}],"funder":[{"DOI":"10.13039\/100009857","name":"Regeneron Pharmaceuticals","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100009857","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Haemophilia"],"published-print":{"date-parts":[[2025,3]]},"abstract":"<jats:title>ABSTRACT<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Introduction<\/jats:title>\n                    <jats:p>Gene editing therapies offer the possibility of substantial improvement in treatment and quality of life for people with haemophilia (PWH) in a landscape of dynamic therapeutic advancement. Developing a common and understandable language to discuss gene editing will be essential to ensure these treatments can be deployed in a safe and effective manner with fully informed and shared decision\u2010making between healthcare professionals (HCPs) and PWH. A lexicon explaining and clarifying key concepts is one potential tool to address these aims. Here we evaluate how a gene editing lexicon could be deployed to maximise impact and improve patient outcomes.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Aim<\/jats:title>\n                    <jats:p>To operationalise the gene editing lexicon for successful adoption by the haemophilia community.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>Through an innovative, iterative process, representatives from the haemophilia community, including multidisciplinary HCPs, PWH, and caregivers, with support from language strategy experts, developed a gene editing lexicon and evaluated operational aspects for real\u2010world adoption of this resource.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>A gene editing lexicon was developed, including infographics illustrating key concepts. Infographics were adapted from the lexicon to further clarify and communicate these concepts. Infographics were found to be a potentially vital tool for enhancing the practical use of the lexicon to promote shared decision\u2010making and attain informed consent for gene editing therapies.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusion<\/jats:title>\n                    <jats:p>A gene editing lexicon shows promise for improving the understanding of gene editing for all stakeholders in the haemophilia community. Ensuring the lexicon remains up to date with current therapies and appropriate strategies for adoption such as infographics will enable this resource to have maximum impact.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1111\/hae.15155","type":"journal-article","created":{"date-parts":[[2025,1,27]],"date-time":"2025-01-27T09:34:02Z","timestamp":1737970442000},"page":"207-213","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":1,"title":["Operationalising a Haemophilia Gene Editing Lexicon for Practical Use"],"prefix":"10.1111","volume":"31","author":[{"ORCID":"https:\/\/orcid.org\/0009-0006-1916-041X","authenticated-orcid":false,"given":"William","family":"McKeown","sequence":"first","affiliation":[{"name":"Care of Elderly Medicine Antrim Area Hospital  Antrim UK"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Cedric","family":"Hermans","sequence":"additional","affiliation":[{"name":"Cliniques universitaires Saint\u2010Luc Universit\u00e9 catholique de Louvain (UCLouvain)  Brussels Belgium"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Carmen","family":"Unzu","sequence":"additional","affiliation":[{"name":"DNA and RNA Medicine Division CIMA\u2010Universidad de Navarra, IdisNA  Pamplona Spain"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-2799-2615","authenticated-orcid":false,"given":"Mark A.","family":"Kay","sequence":"additional","affiliation":[{"name":"Pediatrics \u2010 Human Gene Therapy Stanford University  Palo Alto California USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-7423-9864","authenticated-orcid":false,"given":"Flora","family":"Peyvandi","sequence":"additional","affiliation":[{"name":"Department of Pathophysiology and Transplantation University of Milan  Milan Italy"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Penni","family":"Smith","sequence":"additional","affiliation":[{"name":"Utah Center for Bleeding and Clotting Disorders  Salt Lake City Utah USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-4506-0061","authenticated-orcid":false,"given":"Wolfgang","family":"Miesbach","sequence":"additional","affiliation":[{"name":"Department of Haemostaseology University Hospital Frankfurt  Frankfurt Germany"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-3310-328X","authenticated-orcid":false,"given":"Glenn F.","family":"Pierce","sequence":"additional","affiliation":[{"name":"World Federation of Hemophilia  Montreal Qu\u00e9bec Canada"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-2001-5958","authenticated-orcid":false,"given":"Kate","family":"Khair","sequence":"additional","affiliation":[{"name":"Haemnet  London UK"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Leonard A.","family":"Valentino","sequence":"additional","affiliation":[{"name":"National Bleeding Disorders Foundation  New York New York USA"},{"name":"Hemophilia and Thrombophilia Center Rush University  Chicago Illinois USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-2558-2089","authenticated-orcid":false,"given":"Steven W.","family":"Pipe","sequence":"additional","affiliation":[{"name":"Pediatric Hematology\u2010Oncology University of Michigan  Ann Arbor Michigan USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Monisha","family":"Pillai","sequence":"additional","affiliation":[{"name":"Maslansky and Partners  New York New York USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Micheala","family":"Jones","sequence":"additional","affiliation":[{"name":"Regeneron Pharmaceuticals, Inc.  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