Optimized factor VIII gene
AssigneeBioverativ Therapeutics Inc.
InventorsSiyuan Tan, Robert T. Peters
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Abstract
The present invention provides codon optimized Factor VIII sequences, vectors and host cells comprising codon optimized Factor VIII sequences, polypeptides encoded by codon optimized Factor VIII sequences, and methods of producing such polypeptides. The present invention also provides methods of treating bleeding disorders such as hemophilia comprising administering to the subject a codon optimized Factor VIII nucleic acid sequence or the polypeptide encoded thereby.
Claims17
A method of increasing expression of a polypeptide with Factor VIII activity in a subject comprising administering an isolated nucleic acid molecule to a subject in need thereof,
- wherein the isolated nucleic acid molecule comprises a nucleotide sequence encoding a polypeptide with Factor VIII activity and has at least 96% sequence identity to SEQ ID NO:
- 2,
- wherein the isolated nucleic acid molecule is comprised in a vector, and
- wherein the expression of the polypeptide is increased relative to a reference nucleic acid molecule comprising SEQ ID NO:
- 3 or the vector comprising the reference nucleic acid molecule.
A method of treating a bleeding disorder characterized by a deficiency in Factor VIII, comprising administering to a subject in need thereof an isolated nucleic acid molecule,
- wherein the isolated nucleic acid molecule is comprised in a vector, and
- wherein the isolated nucleic acid molecule comprises a nucleotide sequence encoding a polypeptide with Factor VIII activity and has at least 96% sequence identity to SEQ ID NO:
- 2.
Dependencies are inferred from the claim text.