Gene-based therapies for hemophilia
Gene therapy is a transformative approach to treating genetic disorders in order to improve disease outcomes for patients. Hemophilia A and B are inherited genetic disorders caused by mutations in the FVIII and FIX genes, respectively. Traditional treatments for hemophilia have included intravenous...
Saved in:
| Main Authors: | Melissa F. Glasner, Steven Pipe, Wolfgang Miesbach |
|---|---|
| Format: | Article |
| Language: | English |
| Published: |
Elsevier
2025-05-01
|
| Series: | Research and Practice in Thrombosis and Haemostasis |
| Subjects: | |
| Online Access: | http://www.sciencedirect.com/science/article/pii/S2475037925001943 |
| Tags: |
Add Tag
No Tags, Be the first to tag this record!
|
Similar Items
-
POLYMORPHISM OF THROMBOPHILIA GENES AND THEIR ROLE IN DEVELOPMENT OF DIFFERENT DISEASE PHENOTYPES AND THROMBOTIC COMPLICATIONS IN HEMOPHILIA PATIENTS
by: T. Yu. Polyanskaya, et al.
Published: (2019-03-01) -
CRISPR/Cas9‐mediated somatic correction of a novel coagulator factor IX gene mutation ameliorates hemophilia in mouse
by: Yuting Guan, et al.
Published: (2016-03-01) -
Single‐domain antibodies targeting antithrombin reduce bleeding in hemophilic mice with or without inhibitors
by: Elena Barbon, et al.
Published: (2020-03-01) -
Oral post-surgical complications in patients with hemophilia and von Willebrand disease
by: Luisa Catarina Porfirio de Sousa, et al.
Published: (2025-07-01) -
Non-accidental trauma in the setting of hemophilia A – A case report
by: Kameron Williamson, et al.
Published: (2024-12-01)