Accordingly, lysosomal pathways and their molecular components are emerging as promising pharmacological targets.39 Enzyme replacement therapy, successfully applied in Gaucher disease with recombinant glucocerebrosidase, involves administering functional enzymes to restore lysosomal degradation capacity.40,41 Additionally, clinical trials of adeno-associated viral vector-based gene therapy for patients with Hurler syndrome (Mucopolysaccharidosis Type I, or MPS I), a neurotypical lysosomal storage disorder, are currently ongoing (NCT03580083). Here, GBA1 is linked to Gaucher disease.