专利名称 | METHODS AND COMPOSITIONS OF MMA CONSTRUCTS AND VECTORS | ||
申请号 | WOUS19042073 | 申请日 | |
公开(公告)号 | WO2020018587A1 | 公开(公告)日 | |
申请(专利权)人 | SELECTA BIOSCIENCES INC | 发明人 | KELLER Peter; KISHIMOTO Takashi Kei |
专利来源 | 国家知识产权局 | 转化方式 | |
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. |