HORLING, FRANZISKA,LENGLER, JOHANNES,FALKNER, FALKO-GUNTHER,ROTTENSTEINER, HANSPETER,SCHEIFLINGER, FRIEDRICH
申请号:
CA3064730
公开号:
CA3064730A1
申请日:
2018.05.22
申请国别(地区):
CA
年份:
2018
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.