abstract |
In some aspects, the present disclosure relates to compositions and methods for the treatment of diseases associated with aberrant lysosomal function, such as Parkinson's disease (PD) and Gaucher's disease. In some embodiments, the present disclosure provides expression constructs comprising a transgene encoding beta-glucocerebrosidase (GBA), or a portion thereof, alone or in combination with one or more PD associated genes. In some embodiments, the present disclosure provides a method of Parkinson's disease by administering said expression construct to a subject in need thereof. |