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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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Detalhes bibliográficos
Publicado no:Blood Adv
Main Authors: Merlin, Simone, Famà, Rosella, Borroni, Ester, Zanolini, Diego, Bruscaggin, Valentina, Zucchelli, Silvia, Follenzi, Antonia
Formato: Artigo
Idioma:Inglês
Publicado em: American Society of Hematology 2019
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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