VIRAL VECTORS ENCODING RECOMBINANT FVIII VARIANTS WITH INCREASED EXPRESSION FOR GENE THERAPY OF HEMOPHILIA A

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专利权人:

BAXALTA INCORPORATED

发明人:

FALKNER, FALKO-GUNTER,HORLING, FRANZISKA,LENGLER, JOHANNES,ROTTENSTEINER, HANSPETER,SCHEIFLINGER, FRIEDRICH

申请号：

CA3005565

公开号：

CA3005565A1

申请日:

2016.11.11

申请国别(地区):

CA

年份:

2017

代理人:

摘要：

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.

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