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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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Bibliographic Details
Published in:Int J Mol Sci
Main Authors: Rodríguez-Merchán, E. Carlos, De Pablo-Moreno, Juan Andres, Liras, Antonio
Format: Artigo
Language:Inglês
Published: MDPI 2021
Subjects:
Online Access: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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