These diseases include Fabry disease (FLT190 and 4D-310), late onset Pompe disease (SPK-3006, AAV2/8SPhGAA, and AT845), Mucopolysaccharidosis Type I (SB-318), Mucopolysaccharidosis Type IIIA (LYS-SAF302), Mucopolysaccharidosis Type IIIB (rAAV2/5-hNAGLU), Mucopolysaccharidosis Type VI (AAV2/8.TBG.hARSB), Gangliosidosis (Tay Sachs and/or Sandhoff disease) (AAV-GLB1, AXO-AAV-GM2, TSHA-101, and LYS-GM101), and Gaucher disease Type II (PR001). The gene discussed is CGA; the disease is Sandhoff disease.