Falkner, Falko-Günter,Horling, Franziska,Rottensteiner, Hanspeter,Scheiflinger, Friedrich,Lengler, Johannes
申请号：
AU2020203761
公开号：
AU2020203761A1
申请日:
2020.06.05
申请国别(地区):
AU
年份:
2020
代理人:
摘要：
#$%^&*AU2020203761A120200716.pdf#####Abstract The present disclosure provides, among other aspects, codon-alteredpolynucleotides encoding Factor VIII variants for expression in mammalian cells. In some embodiments the disclosure also provides mammalian gene therapy vectors, infected host cells and methods for treating hemophilia A.WO 2017/083762 PCT/US2016/061684 1/89 LLJ L 0~0
