Optimized fig4 genes and expression cassettes and their use
AssigneeThe University of North Carolina at Chapel Hill
InventorsSteven James Gray, Rachel Bailey
Life of this patent
Estimating the term…
Abstract
This invention relates to polynucleotides comprising optimized FIG4 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 FIG4 gene or aberrant activity of a FIG4 gene product in the subject, such as CMT4J.
Claims35
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.
(canceled)
7-10. (canceled)
13-14. (canceled)
(canceled)
28-30. (canceled)
(canceled)
35-38. (canceled)
40-42. (canceled)
46-48. (canceled)
Dependencies are inferred from the claim text.