Development of a novel gene editing lexicon for hemophilia: methodology and results
This collaborative approach ensures broad adoption of the lexicon within the hemophilia community and readiness for beta testing.
Authors
- William McKeown (ORCID: https://orcid.org/0009-0006-1916-041X)
- Cedric R.J.R. Hermans (ORCID: https://orcid.org/0000-0001-5429-8437)
- David E. Gutstein (ORCID: https://orcid.org/0000-0001-7776-3477)
- Steven W. Pipe (ORCID: https://orcid.org/0000-0003-2558-2089)
- Mark A. Kay (ORCID: https://orcid.org/0000-0002-2799-2615)
- Courtney D. Thornburg (ORCID: https://orcid.org/0000-0002-5665-8958)
- Craig M. Kessler (ORCID: https://orcid.org/0000-0003-0599-5724)
- Paul Smith (ORCID: https://orcid.org/0000-0002-7850-8440)
- Flora Peyvandi (ORCID: https://orcid.org/0000-0001-7423-9864)
- Leonard A. Valentino (ORCID: https://orcid.org/0000-0002-7927-6418)
- Carmen Unzu (ORCID: https://orcid.org/0000-0002-1582-7196)
- Lauren S. Whyte (ORCID: https://orcid.org/0000-0001-7939-656X)
- Kate Khair (ORCID: https://orcid.org/0000-0003-2001-5958)
- Glenn F. Pierce (ORCID: https://orcid.org/0000-0002-3310-328X)
- Wolfgang Miesbach (ORCID: https://orcid.org/0000-0002-4506-0061)
- Virginie Delwart
- Micheala Jones
- Katarina Starcevic
- Monisha Pillai
- Megan Chiao
- Ilia Antonino
- Anil Sindhurakar
Institutions
- Goethe University Frankfurt (DE)
- Cliniques Universitaires Saint-Luc (BE)
- Palo Alto University (US)
- Georgetown University (US)
- Regeneron (United States) (US)
- University of Milan (IT)
- University of Michigan (US)
- University of California San Diego (US)
- Rady Children's Hospital-San Diego (US)
- Bleeding & Clotting Disorders Institute (US)
- Intellia Therapeutics (United States) (US)
- University Hospital Frankfurt (DE)
- Rush University (US)
- Stanford University (US)
Publication Details
- Journal
- Research and Practice in Thrombosis and Haemostasis
- Published
- 2025-02-01
- DOI
- https://doi.org/10.1016/j.rpth.2025.102710
- Citations
- 2
- Primary Topic
- Hemophilia Treatment and Research
- Type
- article
- Field-Weighted Citation Impact
- 3.54