Chemical chaperone could open door to treatment of neurological disorder

Armen Hareyan's picture
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An unexpected finding turned out to be a clue leading researchers at Washington University School of Medicine in St. Louis to propose a new treatment approach for Niemann-Pick disease, a rare, deadly neurodegenerative disorder.

To overcome the genetic defect in Niemann-Pick disease, the researchers suggest that chemical compounds could potentially "chaperone" mutant protein molecules through the cell's quality control machinery. And they believe the approach also could be useful for more common diseases

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