Compositions comprising microbial polypeptides for degrading glycogen and methods for the treatment of glycogen storage diseases

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.

CPC Classifications