SEYMOUR, ALBERT BARNES,AHMED, SEEMIN SEHER,WRIGHT, JASON BOKE,DOLLIVE, SERENA NICOLE,RUBIN, HILLARD
申请号：
CA3089824
公开号：
CA3089824A1
申请日:
2019.02.01
申请国别(地区):
CA
年份:
2019
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.
