| abstract |
The present invention relates to gene therapy for lysosomal disorders. In particular, the present disclosure relates, in certain aspects, to compositions and methods for treating diseases associated with abnormal lysosomal function, such as Parkinson's disease (PD) and Gaucher's disease. In certain embodiments, the present disclosure provides expression constructs comprising, alone or in combination with one or more PD-associated genes, an infusion encoding β-glucocerebrosidase (GBA) or a portion thereof Gene. In certain embodiments, the present disclosure provides methods of treating Parkinson's disease by administering these expression constructs to a subject in need thereof. |