Master gene may shed new light on lysosomal and neurodegenerative disorders

Cells, like ordinary households, produce "garbage" ? debris and dysfunctional elements - that need disposal. When the mechanism for taking out this garbage fails, rare genetic diseases called lysosomal storage disorders (including Tay-Sachs, Batten and Fabry disease) can disable and even kill the children they affect. Researchers have now discovered a master gene that controls not only the lysosomes, which destroy the debris, but also cellular compartments called autophagosomes that encapsulate the material and fuse with the lysosomes to achieve the ultimate clearance of the cell's "garbage."

Molecular Biology Genetics Genes Biology