Batten Disease Researcher Published in Cell Reports

Venkatachalam and Wong

Dr. Venkatachalam and Dr. Wong

Lysosomes have traditionally been thought of as the cell’s trash cans. However, recent evidence suggest that a more appropriate analogy for lysosomes would be arecycling plants – proteins degraded in lysosomes are broken down into components that can be used for other purposes. Recycling of superfluous or toxic material allows cells to remain efficient and ensure normal function during cellular stress. A recent study by the laboratory of Dr. Kartik Venkatachalam and colleagues at the University of Texas Health Sciences Center in Houston, Texas, shows a loss of lysosomal function in fly and mouse models of juvenile Batten and Mucolipidosis type IV, another lysosomal storage disease with similarities to Batten.  Consequently, neurons (brain cells) in these animal models suffered a scarcity of components generated via lysosomal protein degradation (recycling) and diminished activation of critical cellular pathways responsible for brain function and prevention of neuronal loss. Remarkably, the authors have found that administration of a high-protein diet along with a drug currently in phase II clinical trials for treating cancer significantly inhibited neuronal dysfunction in animal models. These findings suggest that this or a similar pharmacotherapeutic strategy could inhibit neurodegeneration in children with juvenile Batten and other lysosomal storage diseases. Read the Cell Reports article.

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