Our studies focus on the isolation and purification of intact endosomes and lysosomes. These organelles are then characterized for their membrane composition to identify novel, membrane proteins. We have established a novel method for the isolation and characterization of endosomes and lysosomes. A long-term goal is to identify all the components of the endosomal/lysosomal apparatus using 2D electrophoresis and tandem mass spectroscopy. Therapy for Diseases that Affect the CNS. Effective gene therapy strategies for the treatment of human disease still remain highly experimental due to difficulties encountered in the actual application of many gene therapy schemes. Thus, we are developing novel approaches to address these limitations. Our strategies focus on "Bypass Therapy" an approach aiming at stimulating alternate pathways to bypass a cellular block caused by a specific gene defect.