abstract |
In general, the present invention provides a method for treating synucleinopathies in a subject who has not been clinically diagnosed with lysosomal storage disease. In addition, related pharmaceutical manufacturing methods and screening methods are also provided. One or both of an acid β-glucocerebrosidase (GBA) polypeptide and a polynucleotide encoding an acid β-glucocerebrosidase (GBA) polypeptide are expressed in the subject's nervous system or in a subject. A pharmaceutical composition is administered comprising an amount effective to reduce the level of α-synuclein in the lysosomal compartment. [Selection] Figure 5 |