US 12,077,772 B2
Transgene cassettes, AAV vectors and AAV viral vectors for the expression of human codon-optimized SLC6A1
Steven J. Gray, Southlake, TX (US); and Frances C. Shaffo, Dallas, TX (US)
Assigned to THE BOARD OF REGENTS OF THE UNIVERSITY OF TEXAS SYSTEM, Austin, TX (US)
Filed by THE BOARD OF REGENTS OF THE UNIVERSITY OF TEXAS SYSTEM, Austin, TX (US)
Filed on Nov. 7, 2020, as Appl. No. 17/092,240.
Claims priority of provisional application 62/932,824, filed on Nov. 8, 2019.
Prior Publication US 2021/0139934 A1, May 13, 2021
Int. Cl. C12N 15/86 (2006.01); A61K 9/00 (2006.01); A61K 38/17 (2006.01); A61K 48/00 (2006.01); C07K 14/705 (2006.01); C12N 7/00 (2006.01)
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.