Recombinant viral vector, recombinant adeno-associated virus comprising the same, and uses thereof in treating sialidosis
AssigneeNational Taiwan University
InventorWuh-Liang HWU
Life of this patent
Estimating the term…
Abstract
Disclosed herein is a recombinant viral vector comprising an AAV 5′ inverted terminal repeat (ITR) sequence, an AAV 3′ ITR sequence, and an expression cassette flanked by the AAV 5′ ITR and 3′ ITR sequences. According to the embodiments of the present disclosure, the expression cassette comprises a first and a second coding sequences respectively encoding a human neuraminidase 1 (NEU1) and a human protective protein/cathepsin A (PPCA). Also disclosed herein are a recombinant adeno-associated virus (AAV) comprising the recombinant viral vector, a pharmaceutical composition comprising the recombinant AAV, and uses thereof in treating sialidosis.
Claims21
A recombinant viral vector comprising an AAV 5′ inverted terminal repeat (ITR) sequence, an AAV 3′ ITR sequence, and an expression cassette, wherein the expression cassette is flanked by the AAV 5′ ITR and 3′ ITR sequences, and comprises,
- (a) a promoter;
- (b) a first coding sequence operably linked to the promoter encoding a human neuraminidase 1 (NEU1);
- and
- (c) a second coding sequence operably linked to the promoter encoding a human protective protein/cathepsin A (PPCA).
(canceled)
Dependencies are inferred from the claim text.