Optimized aga genes and expression cassettes and their use
AssigneeThe University of North Carolina at Chapel Hill
InventorSteven James Gray
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Abstract
This invention relates to polynucleotides comprising optimized aspartylglucosaminidase (AGA) open reading frames and vectors and cells comprising the same. The invention further relates to methods of using the same for delivery of the open reading frame to a cell or a subject and methods for treating aspartylglucosaminuria (AGU) in a subject.
Claims33
A polynucleotide comprising a human AGA open reading frame, wherein the nucleotide sequence has been codon-optimized for expression in human cells.
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