CPC C12N 15/86 (2013.01) [A61K 9/0085 (2013.01); A61K 38/1787 (2013.01); A61K 48/005 (2013.01); A61K 48/0083 (2013.01); C07K 14/70571 (2013.01); C12N 7/00 (2013.01); C12N 2750/14143 (2013.01); C12N 2800/22 (2013.01); C12N 2830/50 (2013.01)] | 21 Claims |
1. A recombinant adeno-associated virus (rAAV) vector comprising in 5′ to 3′ direction:
(a) a first AAV inverted terminal repeat (ITR) sequence;
(b) a promoter sequence;
(c) a transgene nucleic acid molecule of SEQ ID NO: 3 or SEQ ID NO: 6
(d) a polyA sequence; and
(e) a second AAV ITR sequence.
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