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US 2026/0049336 A1Published application
Bicistronic aav vectors encoding hexosaminidase alpha and beta-subunits and uses thereof
AssigneeUniversity of Massachusetts
InventorsMiguel Sena Esteves, Diane Golebiowski
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Abstract
Aspects of the disclosure relate to bicistronic AAV nucleic acid constructs comprising a transgene encoding hexosaminidase A (HEXA) and hexosaminidase (HEXB) proteins. In some embodiments, the disclosure provides methods for treating or preventing lysosomal storage disorders, such as Tay-Sachs disease and Sandhoff disease, using bicistronic nucleic acid constructs described by the disclosure.
Claims35
An isolated nucleic acid construct, comprising
- (i) a first expression cassette, comprising a nucleic acid encoding a hexosaminidase alpha-subunit (HEXA) under the control of a first promoter, and
- (ii) a second expression cassette, comprising a nucleic acid encoding a hexosaminidase beta-subunit (HEXB) under the control of a second promoter,
- wherein the first expression cassette and the second expression cassette are flanked by adeno-associated virus (AAV) inverted terminal repeats (ITRs).
An isolated nucleic acid construct, comprising
- (i) a first expression cassette, comprising a nucleic acid encoding a hexosaminidase alpha-subunit, and
- (ii) a second expression cassette, comprising a nucleic acid encoding a hexosaminidase beta-subunit,
- wherein the first expression cassette and the second expression cassette are operably linked by a bidirectional promoter, and wherein the first expression cassette and the second expression cassette are flanked by adeno-associated virus (AAV) inverted terminal repeats (ITRs).
An isolated nucleic acid comprising the sequence set forth in any one of SEQ ID NOs:
- 3-9.
Dependencies are inferred from the claim text.