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METHODS AND COMPOSITIONS OF MMA CONSTRUCTS AND VECTORS
专利权人:
Selecta Biosciences; Inc.
发明人:
Peter Keller,Takashi Kei Kishimoto
申请号:
US16513576
公开号:
US20200038463A1
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 methylmalonyl-CoA mutase (MUT) as well as related vectors, such as AAV vectors and Anc80 vectors. Also, provided are methods for administering viral vectors that comprise a sequence that encodes an enzyme associated with an organic acidemia and an expression control sequence, in combination with synthetic nanocarriers coupled to an immunosuppressant.
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