CPC C07K 14/4702 (2013.01) [C07K 14/47 (2013.01); C12N 15/86 (2013.01); C12Q 1/6883 (2013.01); A61K 38/00 (2013.01); C12N 2710/10343 (2013.01); C12N 2840/44 (2013.01); C12Q 2600/118 (2013.01); C12Q 2600/156 (2013.01)] | 10 Claims |
1. An adeno-associated virus or vector that further comprises a non-native nucleotide sequence capable of expressing a chimeric protein comprising an N-terminal nucleotide binding domain of transactivation response element DNA-binding protein (TDP-43), a C-terminal domain derived from a splicing repressor, and an autoregulatory element, wherein the autoregulatory element comprises SEQ ID NO: 3.
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