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FVIII expression by its native promoter sustains long-term correction avoiding immune response in hemophilic mice
Here we describe a successful gene therapy approach for hemophilia A (HA), using the natural F8 promoter (pF8) to direct gene replacement to factor VIII (FVIII)–secreting cells. The promoter sequence and the regulatory elements involved in the modulation of F8 expression are still poorly characteriz...
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| Publicado no: | Blood Adv |
|---|---|
| Main Authors: | , , , , , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
American Society of Hematology
2019
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC6418497/ https://ncbi.nlm.nih.gov/pubmed/30862611 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/bloodadvances.2018027979 |
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