Neuronal ceroid lipofuscinosis, also called Batten diseuse (BD) are closely related autosomal recessive neurodegenerative disorders characterized hi storage ol" lipofuscin/ceroid in the lysosomes of neural tissues. ATP synthase subunit c has been shown to bo stored in the lysosomes o t neural tissues and ribroblasts oflate infantile and juvenile BD patients. To understand whether the detect in the BD patients is in lysosomal hydrolases or abberration in the subunit e. we have sequestered subunit c and ubiquitin in the lipid membrane and terri t in in the interlamellar space of liposomes, coated with heat aggregated autologous IgG or apolipoprotein I; and delivered to normal and BD polymorphonucleocytes (PMN) and fibroblasts. respectively. The liposomes were endocyli/ed by cells and accumulated in the lysosomes as evidenced by Electron microscopic studies. Although the rate of degradation was slower by the BD PMN. both normal and BD PMN degraded '""I-ubiquHin. These studies indicate that a Hydrophobie molecule such as subunit c can be delivered to BD PMN and ilbroblasts and the sequestered proteins are accessible ID lysosomal hydroiases and that BD patients the lysosomal hydrolases for the degradation of Hydrophobie proteins remain functional.
|Original language||English (US)|
|State||Published - Dec 1 1996|
ASJC Scopus subject areas
- Molecular Biology