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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Description
ISSN:1365-2516
DOI:10.1111/hae.15155