{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,3,13]],"date-time":"2026-03-13T23:11:59Z","timestamp":1773443519825,"version":"3.50.1"},"reference-count":18,"publisher":"Wiley","issue":"6","license":[{"start":{"date-parts":[[2024,10,22]],"date-time":"2024-10-22T00:00:00Z","timestamp":1729555200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"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":[[2024,11]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Introduction<\/jats:title><jats:p>Despite the progress in gene editing platforms like CRISPR\/Cas9 with the\u00a0potential to transform the standard of care for haemophilia, the language used to explain and discuss gene editing is not aligned across the haemophilia community. Here, we present the objective and rationale for developing a clear, consistent, and globally aligned gene editing lexicon to address these communication gaps.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>Effectively communicating complex gene editing concepts requires a clear and consistent vocabulary. Through collaboration with a diversity of haemophilia stakeholders, our main goal is to develop an accurate, informative lexicon which avoids overpromising or highly technical terminology. Using an innovative process, representatives from several patient and scientific haemophilia organizations and select biotechnology companies will develop and refine language concepts to be tested with approximately seventy participants across the United States of America, United Kingdom, and Germany. Participants will include lived experience experts (LEEs) and haematologists. The process will be overseen by the Lexicon Steering Committee of global experts from leading scientific and patient organizations in the haemophilia and gene editing fields.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>Initial feedback provided a robust foundation and rationale for building clear, consistent language around gene editing. This lexicon development framework will allow for increased understanding across the haemophilia community, including the\u00a0development of valid informed consent and shared decision\u2010making materials.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusion<\/jats:title><jats:p>Results provide important building blocks for stimuli development and highlight the need for a novel gene editing lexicon. In the next phase, language stimuli will be tested with LEEs and haematologists to better understand audience preferences and help shape the final lexicon.<\/jats:p><\/jats:sec>","DOI":"10.1111\/hae.15108","type":"journal-article","created":{"date-parts":[[2024,10,22]],"date-time":"2024-10-22T18:54:10Z","timestamp":1729623250000},"page":"1272-1280","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":5,"title":["A novel gene editing lexicon strategy for the haemophilia community: Research plan for development and preliminary results"],"prefix":"10.1111","volume":"30","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-5429-8437","authenticated-orcid":false,"given":"Cedric","family":"Hermans","sequence":"first","affiliation":[{"name":"Cliniques universitaires saint\u2010Luc Universit\u00e9 catholique de Louvain (UCLOuvain)  Brussels Belgium"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-7927-6418","authenticated-orcid":false,"given":"Leonard A.","family":"Valentino","sequence":"additional","affiliation":[{"name":"National Bleeding Disorders Foundation  New York City New York USA"},{"name":"Hemophilia and Thrombophilia Center Rush University  Chicago Illinois USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Courtney D.","family":"Thornburg","sequence":"additional","affiliation":[{"name":"Department of Pediatrics University of California San Diego  La Jolla California USA"},{"name":"Hemophilia and Thrombosis Treatment Cente Rady Children's Hospital San Diego  San Diego California USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Carmen","family":"Unzu","sequence":"additional","affiliation":[{"name":"DNA&amp;RNA Medicine Division, CIMA\u2010Universidad de Navarra IdisNA  Pamplona Spain"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Mark A.","family":"Kay","sequence":"additional","affiliation":[{"name":"Pediatrics \u2010 Human Gene Therapy Stanford University  Stanford 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\/0009-0006-1916-041X","authenticated-orcid":false,"given":"William","family":"McKeown","sequence":"additional","affiliation":[{"name":"Care of Elderly Medicine Antrim Area Hospital  Antrim Northern Ireland"}],"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 Quebec 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"}]},{"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":"Katarina","family":"Starcevic","sequence":"additional","affiliation":[{"name":"Language Strategy Maslansky and Partners  New York City New York USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Monisha","family":"Pillai","sequence":"additional","affiliation":[{"name":"Language Strategy Maslansky and Partners  New York City New York USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Micheala","family":"Jones","sequence":"additional","affiliation":[{"name":"Regeneron Pharmaceuticals, Inc.  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