BUNTING, Stuart,COLOSI, Peter Cameron,PUNGOR, Erno
申请号:
PE2018000419
公开号:
PE20181338A1
申请日:
2016.09.23
申请国别(地区):
PE
年份:
2018
代理人:
摘要:
The vectors and viruses encoding / expressing factor VIII of adenosine virus (VAA): aav5-fvii-sq. in addition, they also include fviivavectors with high activity and fviivavectors with complete or broken functional protein. It also refers to the preparation method of this vector, that is, FVII, which contains or represents this vector, and the pharmacological formula containing this vector, which is useful in the treatment of hemophilia A a.Referido a vectores y virus que codifican/expresan un Factor VIII (FVIII) de virus adenoasociado (VAA): AAV5-FVIII-SQ. Ademas, incluye vectores FVIIIVAA con una actividad de expresion elevada y vectores FVIII VAA que expresan la proteina FVIII funcional de longitud completa o truncada. Tambien se refiere a los metodos de preparacion de dichos vectores, a las particulas viricas FVIII VAA recombinantes que comprenden o expresan dichos vectores y formulaciones farmaceuticas que los comprenden, siendo utiles en el tratamiento de la hemofilia A.