Albert Barnes Seymour,Seemin Seher Ahmed,Jason Boke Wright,Serena Nicole Dollive,Hillard Rubin
申请号：
US16798890
公开号：
US20200289674A1
申请日:
2020.02.24
申请国别(地区):
US
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.
