Optimised coding sequence and promoter
View Patent ↗An optimized coding sequence of human blood clotting factor eight (VIII) and a promoter may be used in vectors, such as rAAV, for introduction of factor VIII, and/or other blood clotting factors and transgenes. Exemplary of these factors and transgenes are alpha-1-antitrypsin, as well as those involved in the coagulation cascade, hepatocye biology, lysosomal storage, urea cycle disorders, and lipid storage diseases. Cells, vectors, proteins, and glycoproteins produced by cells transformed by the vectors and sequence, may be used in treatment.
1. A vector comprising a promoter, wherein the promoter comprises the nucleotide sequence of SEQ ID NO: 3.
2. The vector of claim 1 which is a recombinant adeno-associated virus (rAAV) vector.
3. An isolated host cell comprising the vector of claim 1 .