BUNTING, Stuart,COLOSI, Peter Cameron,PUNGOR, Erno
申请号：
PE0004192018
公开号：
PE13382018A1
申请日:
2016.09.23
申请国别(地区):
PE
年份:
2018
代理人:
摘要：
Referred to Vectors and viruses that encode \/ Express factor VIII (FVIII), adeno-associated virus (AAV): AAV5 - FVIII sq.It also includes a fviiivaa vector with high expression and activity of FVIII Protein expressing vector to FVIII Functional full-length or truncated.It also refers to the methods of preparation of these vectors,The Viral particles which comprise or recombinant FVIII to express such Vectors and Pharmaceutical formulations containing Same, being useful in the treatment of Hemophilia 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.
