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;Baxalta GmbH

发明人:

Falkner, Falko-Günter,Horling, Franziska,Lengler, Johannes,Rottensteiner, Hanspeter,Scheiflinger, Friedrich

申请号：

AU2016354550

公开号：

AU2016354550A1

申请日:

2016.11.11

申请国别(地区):

AU

年份:

2018

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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http://www.ckcest.cn/home/