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US 2026/0078407 A1Published application
RAAV Vectors for the Treatment of GM1 and GM2 Gangliosidosis
AssigneeUniversity of Massachusetts
InventorMiguel Sena Esteves
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Abstract
Aspects of the disclosure relate to compositions and methods for the treatment of lysosomal storage disorders, such as GM1 gangliosidosis, Tay Sachs disease, and Sandhoff disease. In some embodiments, the compositions comprise viral vectors encoding beta-galactosidase. In some embodiments, the compositions comprise viral vectors encoding beta-hexosaminidase subunits (e.g. HEXA, HEXB, or combinations thereof).
Claims13
1-60. (canceled)
An isolated nucleic acid comprising a sequence as set forth by SEQ ID NO:
- 16, 18, or 19.
A kit comprising:
- a container housing a recombinant AAV (rAAV), wherein the rAAV comprises a capsid containing a nucleic acid, wherein the nucleic acid comprises a sequence as set forth by SEQ ID NO:
- 16, 18, or 19.
A kit comprising:
- (i) a first container housing a first rAAV comprising a capsid containing a first nucleic acid comprising a sequence as set forth by SEQ ID NO:
- 18;
- and
- (ii) a second container housing a second rAAV comprising a capsid containing a second nucleic acid comprising a sequence as set forth by SEQ ID NO:
- 19;
- and, optionally,
- (iii) a third container comprising a pharmaceutically acceptable carrier.
4 more elements
Dependencies are inferred from the claim text.