Myers Bigel, P.A.
Optimized human clotting factor VIII gene expression cassettes and their use
The invention relates to synthetic liver-specific promoters and expression constructs for producing polypeptides and functional nucleic acids in the liver of a subject. The invention further relates to Factor VIII proteins containing modifications in the amino acid sequence of the Factor VIII protein, as well as nucleic acid constructs encoding the Factor VIII proteins and methods of using these compositions to treat a bleeding disorder.
The University of North Carolina at Chapel Hill
A C A C C
A61P C12P A61K C07K C12N
inventional inventional additional inventional inventional
Xiao Juan Zhenhua
Xiao Li Yuan