Ongoing research is exploring several therapeutic approaches for APBD, including small molecules, miRNA, and CRISPR/Cas9 techniques aimed at reducing glycogen synthase 1 to decrease GBE1 substrate levels [9–11], gene therapy to enhance GBE1 enzyme activity [12], and small molecules to promote lysosomal degradation of PBs [13]. The gene discussed is GBE1; the disease is adult polyglucosan body disease.