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scAAV9.U7.ACCA is a non-replicating recombinant adeno-associated virus vector containing 4 antisense sequences designed to target exon 2 in the human DMD gene under the control of U7 small nuclear non-coding RNA. Developed by Nationwide Children’s Hospital, it is being investigated for the treatment of Duchenne muscular dystrophy.
scAAV9.U7.ACCA is a non-replicating recombinant adeno-associated virus vector containing 4 antisense sequences designed to target exon 2 in the human DMD gene under the control of U7 small nuclear non-coding RNA. Developed by Nationwide Children’s Hospital, it is being investigated for the treatment of Duchenne muscular dystrophy.
每项发明一条，涵盖美国、欧洲、PCT 与中国的同族专利。
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专利文献通常使用化学名或研发代号指代药物，而非通用名；且专利申请在提交约 18 个月后才公开，较新的药物可能尚未出现在这里。
