Therapeutics for Glycogen Accumulation Disease Type III

摘要

The present invention provides various translatable polynucleotide and oligomer molecules expressing human amylo-alpha-1, 6-glucosidase, 4-alpha-glucanotransferase (AGL), or fragments thereof, having AGL activity. . The present polynucleotides and oligomeric molecules are expressible to provide human AGL or fragments thereof having AGL activity. The molecule can be used as an active agent that expresses an active polypeptide or protein in a cell or subject. The agent can improve, prevent, delay the onset of a disease or condition associated with reduced activity of amylo-alpha-1, 6-glucosidase, 4-alpha-glucanotransferase (AGL) in a subject. Or in a method for treatment.