Raav vectors encoding of lysosomal beta-galactosidase (glb1) and cathepsin a
AssigneeUniversity of Massachusetts
InventorMiguel Sena Esteves
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Abstract
Aspects of the disclosure relate to methods for co-administration of recombinant AAVs (rAAVs). In some embodiments, the methods comprise administering one or more rAAVs engineered to express a transgene encoding β-galactosidase (GLB1), Cathepsin A (CTSA), and/or β-galactosidase (GLB1) and Cathepsin A (CTSA). In some embodiments, the disclosure provides methods for treating lysosomal storage disorders, such as GM-1 gangliosidosis, using compositions described by the disclosure.
Claims57
A method comprising administering to a cell:
- (i) a first isolated nucleic acid comprising a transgene engineered to express a β-galactosidase (GLB) protein;
- and
- (ii) a second isolated nucleic acid comprising a transgene engineered to express a cathepsin protein.
An isolated nucleic acid comprising the sequence set forth in any one of SEQ ID NOs:
- 1-4.
An isolated nucleic acid comprising the sequence set forth in any one of SEQ ID NO:
- 9-12.
A composition comprising:
- (i) a first recombinant adeno-associated virus (rAAV) comprising a transgene engineered to express a β-galactosidase (GLB) protein;
- and
- (ii) a second rAAV, comprising a transgene engineered to express a cathepsin protein.
A recombinant adeno-associated virus (rAAV) comprising a transgene engineered to express:
- (i) a β-galactosidase (GLB) protein, or a portion thereof;
- and
- (ii) a cathepsin protein or a portion thereof.
A method for treating a lysosomal storage disease, the method comprising administering to a subject having a lysosomal storage disease:
- (i) a first isolated nucleic acid comprising a transgene engineered to express a β-galactosidase (GLB) protein;
- and
- (ii) a second isolated nucleic acid comprising a transgene engineered to express a cathepsin protein.
Dependencies are inferred from the claim text.