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US 12,516,301 B2Granted
Optimized SUMF1 genes and expression cassettes and their use
AssigneeThe University of North Carolina at Chapel Hill
InventorsSteven James Gray, Rachel Bailey
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Abstract
This invention relates to polynucleotides comprising optimized SUMF1 open reading frame (ORF) sequences, vectors comprising the same, and methods of using the same for deliver) of the ORF to a cell or a subject and to treat disorders associated with aberrant expression of a SUMF1 gene or aberrant activity of a SUMF1 gene product in the subject, such as SUMF1 disease.
Claims19
A polynucleotide comprising a human SUMF1 open reading frame, wherein the human SUMF1 open reading frame is codon-optimized for expression in a human cell and comprises the nucleotide sequence of SEQ ID NO:
- 1 or a nucleotide sequence having at least about 90% identity thereto.
Dependencies are inferred from the claim text.