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US 2025/0186618 A1Published application
Compositions and methods for treating and/or preventing glycogen storage diseases
AssigneeDuke University
InventorsBaodong Sun, Priya Kishnani
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Abstract
Disclosed herein are compositions for and gene therapy methods of treating and/or preventing glycogen storage disease progression including the progression of GSD IV and/or APBD. Also disclosed herein are compositions for and gene therapy methods of preventing glycogen accumulation and/or degrading accumulated glycogen.
Claims29
An isolated nucleic acid molecule, comprising:
- a nucleic acid sequence encoding a polypeptide capable of preventing glycogen accumulation and/or degrading accumulated glycogen, wherein the nucleic acid sequence is a codon-optimized for expression in a human or mammalian cell, and wherein the polypeptide capable of preventing glycogen accumulation and/or degrading accumulated glycogen comprises a glycogen branching enzyme (GBE).
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Dependencies are inferred from the claim text.