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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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Detalles Bibliográficos
Autores principales: Rodríguez-Merchán, E. Carlos, De Pablo-Moreno, Juan Andres, Liras, Antonio
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8306493/
https://www.ncbi.nlm.nih.gov/pubmed/34299267
http://dx.doi.org/10.3390/ijms22147647