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Gene Therapy in Hemophilia: Recent Advances
Hemophilia is a monogenic mutational disease affecting coagulation factor VIII or factor IX genes. The palliative treatment of choice is based on the use of safe and effective recombinant clotting factors. Advanced therapies will be curative, ensuring stable and durable concentrations of the defecti...
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| Publicado no: | Int J Mol Sci |
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| Main Authors: | , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
MDPI
2021
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC8306493/ https://ncbi.nlm.nih.gov/pubmed/34299267 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3390/ijms22147647 |
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