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US 12,077,772 B2Granted
Transgene cassettes, AAV vectors and AAV viral vectors for the expression of human codon-optimized SLC6A1
AssigneeBoard of Regents, The University of Texas System
InventorsSteven J. Gray, Frances C. Shaffo
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Abstract
The present disclosure provides methods and compositions for the treatment of diseases and genetic disorders linked to SLC6A1 loss and/or misfunction. The methods and compositions of the present disclosure comprise rAAV vectors and rAAV viral vectors comprising transgene nucleic acid molecules comprising nucleic acid sequences encoding for a GAT1 polypeptide.
Claims21
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.
5 more elements
Dependencies are inferred from the claim text.