The NGI Center funded a pilot project to Drs. Dickson, Cooper and Sardiello to Monitoring Lysosomal Content and Signaling in Lysosomal Storage Disorders. This funding led to a larger collaborative NIH funding for more than $8 million.
This collaborative effort, which combines the areas of pediatrics, genetics, bioinformatics, and psychiatry will lead scientists to understand why cells die in lysosomal storage disorders (LSD). At this time, LSDs, which include over 50 genetic diseases, lead very young children to lose intellectual and cognitive function over time, and eventually die. No treatments exist to cure brain cell death, but only delay the progress.
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