THE CHILDREN'S HOSPITAL OF PHILADELPHIA;THE CHILDRENS HOSPITAL OF PHILADELPHIA
发明人:
YAZICIOGLU, MUSTAFA, N.,MINGOZZI, FEDERICO,ANGUELA, XAVIER,HIGH, KATHERINE, A.
申请号:
SG11201406776T
公开号:
SG11201406776TA
申请日:
2013.04.18
申请国别(地区):
SG
年份:
2015
代理人:
摘要:
Compositions and methods for AAV mediated gene therapy are disclosed. AAV vectors comprise protein capsid variants comprising therapeutically beneficial transgenes. AAV variants are provided which exhibit increased transduction efficiency when compared to AAV serotypes (e.g., AAV1, AAV2, AAV8, AAV-rh74), which lack the modifications disclosed herein. Such improved vectors are useful for transduction of a variety of tissues.