The Children's Hospital of Philadelphia;The Childrens Hospital of Philadelphia
发明人:
High, Katherine A.,Mingozzi, Federico,Sun, Junwei,Johnson, Philip
申请号：
AU2013221212
公开号：
AU2013221212A1
申请日:
2013.02.19
申请国别(地区):
AU
年份:
2014
代理人:
摘要：
The invention relates to adeno-associated virus (AAV) serotype AAV-Rh74 and related AAV vectors, and AAV-Rh74 and related AAV vector mediated gene transfer methods and uses. In particular, AAV-Rh74 targets polynucleotides to cells, tissues or organs for expression (transcription) of genes encoding therapeutic proteins and peptides, and polynucleotides that function as or are transcribed into inhibitory nucleic acid sequences.
