abstract |
In some aspects, the present disclosure relates to compositions and methods for the treatment of diseases associated with abnormal lysosomal function, such as Parkinson's disease and Gauche disease. In some embodiments, the present disclosure is an expression comprising a transgene encoding beta-glucocerebrosidase (GBA) or a portion thereof, lysosomal membrane protein 2 (LIMP2), prosaposin, or any combination of the above. Provide constructs. In some embodiments, the present disclosure provides a method of Parkinson's disease by administering the expression construct to a subject in need thereof. |