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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...
Autores principales: | , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Springer International Publishing
2023
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Materias: | |
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 |