SEYMOUR, Albert Barnes,AHMED, Seemin Seher,WRIGHT, Jason Boke,DOLLIVE, Serena Nicole,RUBIN, Hillard
申请号：
AU2019215150
公开号：
AU2019215150A1
申请日:
2019.02.01
申请国别(地区):
AU
年份:
2020
代理人:
摘要：
Provided herein are adeno-associated virus (AAV) compositions that can express a phenylalanine hydroxylase (PAH) polypeptide in a cell, thereby restoring the PAH gene function. Also provided are methods of use of the AAV compositions, and packaging systems for making the AAV compositions.
