Vectors for liver-directed gene therapy of hemophilia and methods and use thereof

The present invention relates to vectors containing liver-specific regulatory sequences and codon-optimized factor IX or factor VIII genes, methods employing these vectors and uses of these vectors. Expression cassettes and vectors containing these liver-specific regulatory elements and codon-optimized factor IX or factor VIII genes are also disclosed. The present invention is particularly useful for applications using gene therapy, in particular for the treatment of hemophilia A and B.

Octrooi-publicatienummer: US10398787

Jaar aanvraag: 2018

Jaar toekenning: 2018

Gevalideerd voor IOF-sleutel: Ja

Toegewezen aan: Universitaire Associatie Brussel