Evaluating Gene Therapy as a Potential Paradigm Shift in Treating Severe Hemophilia

Hemophilia is characterized by a deficiency in coagulation factors VIII or IX. The general standard of care for severe hemophilia is frequent intravenous recombinant or plasma-derived factor replacement to prevent bleeding. While this treatment is effective in preventing bleeding, frequent infusions...

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Detalles Bibliográficos
Autores principales: Thornburg, Courtney D., Simmons, Dana H., von Drygalski, Annette
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Springer International Publishing 2023
Materias:
Review Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10432364/
https://www.ncbi.nlm.nih.gov/pubmed/37490225
http://dx.doi.org/10.1007/s40259-023-00615-4

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