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US 2019/0194295 A1Published application
Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a
AssigneeBaxalta Incorporated
InventorsFalko-Günter Falkner, Franziska Horling, Johannes Lengler, Hanspeter Rottensteiner, Friedrich Scheiflinger
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Abstract
The present disclosure provides, among other aspects, codon-altered polynucleotides encoding Factor VIII variants for expression in mammalian cells. In some embodiments, the disclosure also provides mammalian gene therapy vectors and methods for treating hemophilia A.
Claims11
A polynucleotide comprising the nucleotide sequence of SEQ ID NO:
- 13, wherein the polynucleotide encodes a Factor VIII polypeptide.
2-65. (canceled)
Dependencies are inferred from the claim text.