Optimized liver-specific expression systems for fviii and fix

The present invention relates to nucleic acid expression cassettes and vectors containing liver- specific regulatory elements and codon-optimized factor IX or factor VIII transgenes, methods employing these expression cassettes and vectors and uses thereof. The present invention is particularly useful for applications using liver-directed gene therapy, in particular for the treatment of hemophilia A and B.

Octrooi-publicatienummer: EP3270944

Jaar aanvraag: 2019

Jaar toekenning: 2019

Gevalideerd voor IOF-sleutel: Ja

Toegewezen aan: Universitaire Associatie Brussel