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Master gene may hold key to treating lysosomal storage disorders
HOUSTON -- (September 7, 2011) --... researchers showed that a master gene called transcription factor EB or TFEB controls both the mechanism by which debris is gathered up in cell and that which destroys it.

In ...the journal Developmental Cell, Dr. Andrea Ballabio... along with his colleagues have found that TFEB also regulates exocytosis of lysosomes – a process in which these intracellular machines act as a dump truck, taking the debris to the inner surface of the cell and fusing with its membrane to dump debris outside the cells.

Getting rid of abnormal buildup
Cells need to be able to rid themselves of the debris that collects after important activities. When this debris piles up in the cell, it can clog the cell,... When the lysosomes that carry out the destruction process fail, diseases such as Tay Sachs, Batten and Fabry result... In adults, the failure of this system may lead to neurodegenerative diseases such as Alzheimer’s or Parkinson’s.

In this study, the researchers found that increasing the level TFEB draws more lysosomes to the cell membrane and also improves their ability to fuse there. When the researchers increased the amount of TFEB in cell models of diseases associated with defects in debris removal (lysosomal storage disorders), they were "rescued." In other words, the increased number of lysosomes rid the cells of the abnormal buildup of debris...


The findings will have to be verified ...to determine if this ... can be used as a treatment.

New way to treat neurodegenerative disorders?
"These findings do suggest an alternative strategy to treat LSDs (lysosomal storage disorders) and common neurodegenerative disorders," said Ballabio.

http://www.bcm.edu/news/item.cfm?newsID=4399