Peter Keller,Takashi Kei Kishimoto,Andres Muro,Giulia De Sabbata
申请号：
US16513566
公开号：
US20200038462A1
申请日:
2019.07.16
申请国别(地区):
US
年份:
2020
代理人:
摘要：
Provided herein are methods and compositions related to nucleic acids encoding ornithine transcarbamylase (OTC), such as nucleic acids comprising an OTC codon-optimized sequence, as well as related vectors, such as AAV vectors. Also, provided are methods for administering AAV vectors that comprise a sequence that encodes an enzyme associated with an urea cycle disorder and an expression control sequence, in combination with synthetic nanocarriers coupled to an immunosuppressant.
