Compositions and Methods for the Treatment of Genetic Diseases
AssigneeDuke University
InventorsBaodong Sun, Baodong Sun, Jeong-A Lim
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Abstract
This disclosure provides isolated nucleic acid molecules comprising nucleic acid sequences encoding microbial polypeptides that are codon optimized for expression in mammalian cells, vectors comprising an immunotolerant dual promoter system, and methods using these polynucleotides and polypeptides to treat glycogen storage diseases and other inherited diseases.
Claims31
A isolated nucleic acid molecule comprising a nucleic acid sequence encoding a microbial polypeptide for degrading glycogen, wherein the nucleic acid sequence is codon-optimized for expression in a mammalian cell.
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A method of treating a deficiency of a polypeptide for degrading glycogen in a subject, comprising administering to the subject an isolated nucleic acid molecule comprising a nucleic acid sequence encoding a microbial polypeptide for degrading glycogen.
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Dependencies are inferred from the claim text.