| CPC C12N 15/102 (2013.01) [C12N 15/86 (2013.01); C12N 15/907 (2013.01); C12N 2310/20 (2017.05); C12N 2800/80 (2013.01)] | 19 Claims |
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1. A vector comprising a transgene comprising in 5′ to 3′ orientation: a first splice acceptor, a first coding sequence, a first terminator, a second terminator reverse complement, a second coding sequence reverse complement, and a second splice acceptor reverse complement,
wherein the first coding sequence is operably linked to the first splice acceptor and first terminator, and the second coding sequence is operably linked to the second splice acceptor and second terminator, wherein the first terminator is selected from an SV40 poly (A) or BGH poly (A),
wherein the first and second coding sequences differ in nucleic acid sequence but each encode the same amino acid sequence, wherein said amino acid sequences encoded by the first and second coding sequences correspond to amino acid sequences having at least 80% identity to the amino acid sequence of an endogenous human ATXN3 gene, and wherein the transgene is equal to or less than 4.7 kb.
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