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A new “FIX” for hemophilia B gene therapy
In this issue of Blood, Nair et al(1) demonstrate that AAV-directed gene therapy using a new bioengineered FIX transgene provides higher FIX activity and superior hemostatic efficacy than other FIX variants and may allow for lower and potentially safer vector doses in future human clinical trials.
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| Publicado en: | Blood |
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| Autor principal: | |
| Formato: | Artigo |
| Lenguaje: | Inglês |
| Publicado: |
American Society of Hematology
2021
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| Materias: | |
| Acceso en línea: | https://ncbi.nlm.nih.gov/pmc/articles/PMC8160502/ https://ncbi.nlm.nih.gov/pubmed/34042982 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/blood.2021011753 |
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