Operationalising a Haemophilia Gene Editing Lexicon for Practical Use.

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Title: Operationalising a Haemophilia Gene Editing Lexicon for Practical Use.
Authors: McKeown W; Care of Elderly Medicine, Antrim Area Hospital, Antrim, UK., Hermans C; Cliniques universitaires Saint-Luc, Université catholique de Louvain (UCLouvain), Brussels, Belgium., Unzu C; DNA and RNA Medicine Division, CIMA-Universidad de Navarra, IdisNA, Pamplona, Spain., Kay MA; Pediatrics - Human Gene Therapy, Stanford University, Palo Alto, California, USA., Peyvandi F; Department of Pathophysiology and Transplantation, University of Milan, Milan, Italy., Smith P; Utah Center for Bleeding and Clotting Disorders, Salt Lake City, Utah, USA., Miesbach W; Department of Haemostaseology, University Hospital Frankfurt, Frankfurt, Germany., Pierce GF; World Federation of Hemophilia, Montreal, Québec, Canada., Khair K; Haemnet, London, UK., Valentino LA; National Bleeding Disorders Foundation, New York, New York, USA.; Hemophilia and Thrombophilia Center, Rush University, Chicago, Illinois, USA., Pipe SW; Pediatric Hematology-Oncology, University of Michigan, Ann Arbor, Michigan, USA., Pillai M; Maslansky and Partners, New York, New York, USA., Jones M; Regeneron Pharmaceuticals, Inc., Tarrytown, New York, USA., Delwart V; Regeneron Pharmaceuticals, Inc., Tarrytown, New York, USA., Sindhurakar A; Regeneron Pharmaceuticals, Inc., Tarrytown, New York, USA., Gutstein DE; Regeneron Pharmaceuticals, Inc., Tarrytown, New York, USA., Kessler CM; Medicine and Pathology, Georgetown University, Washington, District of Columbia, USA.
Source: Haemophilia : the official journal of the World Federation of Hemophilia [Haemophilia] 2025 Mar; Vol. 31 (2), pp. 207-213. Date of Electronic Publication: 2025 Jan 27.
Publication Type: Journal Article
Journal Info: Publisher: Blackwell Science Country of Publication: England NLM ID: 9442916 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1365-2516 (Electronic) Linking ISSN: 13518216 NLM ISO Abbreviation: Haemophilia Subsets: MEDLINE
Database: MEDLINE Ultimate
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  Data: <searchLink fieldCode="AU" term="%22McKeown+W%22">McKeown W</searchLink>; Care of Elderly Medicine, Antrim Area Hospital, Antrim, UK.<br /><searchLink fieldCode="AU" term="%22Hermans+C%22">Hermans C</searchLink>; Cliniques universitaires Saint-Luc, Université catholique de Louvain (UCLouvain), Brussels, Belgium.<br /><searchLink fieldCode="AU" term="%22Unzu+C%22">Unzu C</searchLink>; DNA and RNA Medicine Division, CIMA-Universidad de Navarra, IdisNA, Pamplona, Spain.<br /><searchLink fieldCode="AU" term="%22Kay+MA%22">Kay MA</searchLink>; Pediatrics - Human Gene Therapy, Stanford University, Palo Alto, California, USA.<br /><searchLink fieldCode="AU" term="%22Peyvandi+F%22">Peyvandi F</searchLink>; Department of Pathophysiology and Transplantation, University of Milan, Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Smith+P%22">Smith P</searchLink>; Utah Center for Bleeding and Clotting Disorders, Salt Lake City, Utah, USA.<br /><searchLink fieldCode="AU" term="%22Miesbach+W%22">Miesbach W</searchLink>; Department of Haemostaseology, University Hospital Frankfurt, Frankfurt, Germany.<br /><searchLink fieldCode="AU" term="%22Pierce+GF%22">Pierce GF</searchLink>; World Federation of Hemophilia, Montreal, Québec, Canada.<br /><searchLink fieldCode="AU" term="%22Khair+K%22">Khair K</searchLink>; Haemnet, London, UK.<br /><searchLink fieldCode="AU" term="%22Valentino+LA%22">Valentino LA</searchLink>; National Bleeding Disorders Foundation, New York, New York, USA.; Hemophilia and Thrombophilia Center, Rush University, Chicago, Illinois, USA.<br /><searchLink fieldCode="AU" term="%22Pipe+SW%22">Pipe SW</searchLink>; Pediatric Hematology-Oncology, University of Michigan, Ann Arbor, Michigan, USA.<br /><searchLink fieldCode="AU" term="%22Pillai+M%22">Pillai M</searchLink>; Maslansky and Partners, New York, New York, USA.<br /><searchLink fieldCode="AU" term="%22Jones+M%22">Jones M</searchLink>; Regeneron Pharmaceuticals, Inc., Tarrytown, New York, USA.<br /><searchLink fieldCode="AU" term="%22Delwart+V%22">Delwart V</searchLink>; Regeneron Pharmaceuticals, Inc., Tarrytown, New York, USA.<br /><searchLink fieldCode="AU" term="%22Sindhurakar+A%22">Sindhurakar A</searchLink>; Regeneron Pharmaceuticals, Inc., Tarrytown, New York, USA.<br /><searchLink fieldCode="AU" term="%22Gutstein+DE%22">Gutstein DE</searchLink>; Regeneron Pharmaceuticals, Inc., Tarrytown, New York, USA.<br /><searchLink fieldCode="AU" term="%22Kessler+CM%22">Kessler CM</searchLink>; Medicine and Pathology, Georgetown University, Washington, District of Columbia, USA.
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  Data: <i>Publisher: </i><searchLink fieldCode="PB" term="%22Blackwell+Science%22">Blackwell Science </searchLink><i>Country of Publication: </i>England <i>NLM ID: </i>9442916 <i>Publication Model: </i>Print-Electronic <i>Cited Medium: </i>Internet <i>ISSN: </i>1365-2516 (Electronic) <i>Linking ISSN: </i><searchLink fieldCode="IS" term="%2213518216%22">13518216 </searchLink><i>NLM ISO Abbreviation: </i>Haemophilia <i>Subsets: </i>MEDLINE
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              Text: 2025 Mar
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