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:

Hanspeter Rottensteiner,Friedrich Scheiflinger

申请号：

US16743850

公开号：

US20200289672A1

申请日:

2020.01.15

申请国别(地区):

US

年份:

2020

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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