Horling, Franziska,Lengler, Johannes,Falkner, Falko-Gunther,Rottensteiner, Hanspeter,Scheiflinger, Friedrich
申请号：
AU2018272831
公开号：
AU2018272831A1
申请日:
2018.05.22
申请国别(地区):
AU
年份:
2019
代理人:
摘要：
The present disclosure provides, among other aspects, codon-altered polynucleotides encoding Factor IX variants for expression in mammalian cells. In some embodiments, the disclosure also provides mammalian gene therapy vectors and methods for treating hemophilia B.
