Optimized FIG4 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 FIG. 4 open reading frame (ORF) sequences, vectors comprising the same, and methods of using the same for delivery of the ORF to a cell or a subject and to treat disorders associated with aberrant expression of a FIG. 4 gene or aberrant activity of a FIG. 4 gene product in the subject, such as CMT4J.
Claims23
A polynucleotide comprising a human FIG4 open reading frame, wherein the human FIG4 open reading frame is codon-optimized for expression in a human cell, wherein said human FIG4 open reading frame 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.