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US 2026/0297618 A1Published application
Gm2 activator vectors and methods for use thereof
AssigneeGencorrex Therapies Inc.
InventorJagdeep WALIA
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Abstract
This disclosure relates to nucleic acid constructs for expression of GM2 activator (GM2A) protein, and viral vectors comprising said constructs useful for the treatment of AB-variant GM2 Gangliosidosis (ABGM2). Also provided are methods and uses of the vectors disclosed herein for the treatment of ABGM2.
Claims21
A nucleic acid construct comprising a nucleotide sequence encoding a GM2 activator (GM2A) protein operably linked to a promoter and a transcription termination site.
15-19. (canceled)
Dependencies are inferred from the claim text.