Methods of treating metachromatic leukodystrophy
AssigneeGenzyme Corporation
InventorShyam RAMACHANDRAN
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Abstract
Provided herein are expression cassettes for expressing a transgene in a liver cell, wherein the transgene encodes an ARSA polypeptide. Also provided are methods to treat metachromatic leukodystrophy (MLD). Further provided herein are vectors (e.g., rAAV vectors), viral particles, pharmaceutical compositions and kits for expressing an ARSA polypeptide in an individual in need thereof.
Claims23
A recombinant adeno-associated virus (rAAV) particle comprising (1) a rAAV vector comprising an expression cassette for expressing an arylsulfatase A (ARSA) polypeptide, wherein the expression cassette comprises a gene encoding the ARSA polypeptide operably linked to a promoter and optionally an enhancer, and (2) a modified AAV9 capsid protein comprising a targeting peptide that has SEQ ID NO:
- 10.
21-24. (canceled)
26-49. (canceled)
Dependencies are inferred from the claim text.