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US 12,582,724 B2Granted
Compositions comprising microbial polypeptides for degrading glycogen and methods for the treatment of glycogen storage diseases
AssigneeDuke University
InventorsPriya Kishnani, 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.
Claims14
A 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;
- wherein the microbial polypeptide is a type I Pullulanase from Bacillus subtilis or a limit dextrin alpha-1,6-glucohydrolase (GlgX) from Escherichia coli,
- wherein the type I Pullulanase is encoded by SEQ ID NOs:
- 3 and 8-16 and the GlgX is encoded by SEQ ID NOs:
- 6 and 17-25.
3 more elements
Dependencies are inferred from the claim text.