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Beyond Batten Disease Foundation awards $1.75M grant to Drs. Ballabio and Sardiello

Sep. 30, 2014

Beyond Batten Disease Foundation awards a $1.75 million grant to the Jan and Dan Duncan Neurological Research Institute at Texas Children’s Hospital in Houston, Texas to explore whether the pharmacological activation of TFEB can be used to activate the lysosomal system and to promote cellular clearance in JNCL, which in turn will slow disease progression. Drs Ballabio and Sardiello will use these funds to: 1) validate these drug targets, 2) screen thousands of compounds for their ability to reach and activate these targets, 3) explore the effects of the most promising compounds on the health of juvenile Batten mouse models, 4) explore the effects of the most promising compounds on juvenile Batten iPS cells, the first human brain cell model for juvenile Batten disease, and 5) continue their search for the reason why the loss of CLN3 protein causes juvenile Batten disease. Read more