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The gene therapy journey for hemophilia: are we there yet?

Since the isolation and characterization of the genes for FVIII and FIX some 30 years ago, a longstanding goal of the field has been development of successful gene therapy for the hemophilias. In a landmark study published in 2011, Nathwani et al demonstrated successful conversion of severe hemophil...

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Detalles Bibliográficos
Autor Principal: High, Katherine A.
Formato: Artigo
Idioma:Inglês
Publicado: American Society of Hematology 2012
Assuntos:
Acceso en liña:https://ncbi.nlm.nih.gov/pmc/articles/PMC3512227/
https://ncbi.nlm.nih.gov/pubmed/22829631
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/blood-2012-05-423210
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