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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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Publicat a:Blood Adv
Autors principals: Merlin, Simone, Famà, Rosella, Borroni, Ester, Zanolini, Diego, Bruscaggin, Valentina, Zucchelli, Silvia, Follenzi, Antonia
Format: Artigo
Idioma:Inglês
Publicat: American Society of Hematology 2019
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Accés en línia: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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