| abstract |
The present invention relates to expression constructs and vectors for the treatment and/or prevention of diseases that are associated with a loss of GBA1 function, such as lysosomal storage disorders including Gaucher disease or Niemann-Pick type C (NPC) disease, and synucleinopathies including Parkinson's disease, dementia with Lewy bodies, multi-system atrophy (MSA) or pure autonomic failure (PAF). |