Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency

Abstract

The disclosure relates to compositions and methods for altering, e.g., enhancing, the expression of GCase proteins, whether in vitro and / or in vivo. Such compositions include delivery of an adeno-associated viral (AAV) particle. The compositions and methods of the present disclosure are useful in the treatment of subjects diagnosed with, or suspected of having Parkinson Disease or related condition resulting from a deficiency in the quantity and / or function of GBA gene product or associated with decreased expression or protein levels of GCase protein.