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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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| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
American Society of Hematology
2012
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| Assuntos: | |
| Acesso em linha: | 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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