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US 2026/0048146 A1Published application
Methods and compositions for treating leukodystrophies
OwnerGenevieve LAFORET, Adam SHAYWITZ +1inventors
InventorsGenevieve LAFORET, Adam SHAYWITZ, Kathleen M. KIRBY
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Abstract
Disclosed herein are recombinant adeno-associated viral vectors expressing aspartoacylase (ASPA) protein and related uses for treating leukodystrophies.
Claims35
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A method of treating Canavan disease in a subject in need thereof, comprising:
- administering to the subject a therapeutically effective amount of a recombinant adeno-associated virus (rAAV) vector, wherein the rAAV vector comprises:
- (i) a nucleic acid molecule comprising at least one AAV inverted terminal repeat (ITR) and
- (ii) a non-AAV nucleotide sequence encoding aspartoacylase (ASPA), wherein the non-AAV nucleotide sequence is operably linked to a promoter;
- and
- wherein the therapeutically effective amount is in the range of about 1013 vg/kg to about 1015 vg/kg,
- thereby treating Canavan disease in the subject.
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Dependencies are inferred from the claim text.