Mustafa N. Yazicioglu,Federico Mingozzi,Xavier Aaguela,Katherine A. High
申请号:
US15887641
公开号:
US20180245098A1
申请日:
2018.02.02
申请国别(地区):
US
年份:
2018
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.