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/Mini-gde For The Treatment Of Glycogen Storage Disease Iii
Abstract

The present invention relates to a mini-glycogen debranching enzyme ((GDE); mini-GDE) for the treatment of glycogen storage disease III (GSDIII (Cori disease)). Also disclosed are functional truncated human GDE polypeptides, functional non-human GDE polypeptides, nucleic acid molecules, nucleic acid constructs, or vectors encoding functional truncated human GDE or non-human GDE polypeptides as well as host cells expressing functional truncated human GDE or non-human GDE polypeptides. In a further aspect, the disclosure provides functional truncated human GDE polypeptides, functional non-human GDE polypeptides, nucleic acid molecules, nucleic acid constructs, vectors, or cells of the invention for use in a method for treating GSDIII (Cori disease).

Full Text

What is claimed is:

The present invention relates to a mini-glycogen debranching enzyme ((GDE); mini-GDE) for the treatment of glycogen storage disease III (GSDIII (Cori disease)). Also disclosed are functional truncated human GDE polypeptides, functional non-human GDE polypeptides, nucleic acid molecules, nucleic acid constructs, or vectors encoding functional truncated human GDE or non-human GDE polypeptides as well as host cells expressing functional truncated human GDE or non-human GDE polypeptides. In a further aspect, the disclosure provides functional truncated human GDE polypeptides, functional non-human GDE polypeptides, nucleic acid molecules, nucleic acid constructs, vectors, or cells of the invention for use in a method for treating GSDIII (Cori disease).
Timeline
Filed
05/19/2026
Published
09/10/2026
Granted
Not Available
IPC Codes(4)
C12N 9/44:acting on alpha-1, 6-glucosidic bonds, e.g. isoamylase, pullulanase
A61K 38/00:Medicinal preparations containing peptides (peptides containing beta-lactam rings A61K 31/00; cyclic dipeptides not having in their molecule any other peptide link than those which form their ring, e.g. piperazine-2,5-diones, A61K 31/00; ergoline-based peptides A61K 31/48; containing macromolecular compounds having statistically distributed amino acid units A61K 31/74; medicinal preparations containing antigens or antibodies A61K 39/00; medicinal preparations characterised by the non-active ingredients, e.g. peptides as drug carriers, A61K 47/00)
C12N 9/10:Transferases (2.) (ribonucleases C12N 9/22)