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October 11, 2021

Small-Molecule Suppression of Calpastatin Degradation Reduces Neuropathology in Models of Huntington’s Disease

Huntington’s Disease (HD) is a fatal neurological disorder caused by a trinucleotide CAG repeat expansion in the N-terminal exon 1 of the huntingtin (Htt) gene. HD manifests as uncontrolled involuntary movements accompanied by progressive motor and cognitive deficits, psychiatric disturbance, and dementia. There is no therapy to slow or prevent disease progression.

Mitochondria are the “the powerhouse of the cell”. Disease can occur when mitochondria are unable to generate energy for the needs of cells, and mitochondrial dysfunction is a common hallmark of neurological disorders. Because mitochondrial dysfunction plays a critical role in the pathogenesis of HD, Dr. Qi and her team seek to identify chemical compounds that enhance mitochondrial function as a potential strategy for treating HD.

With the support from the Harrington Discovery Institute, Dr. Qi and her team are conducting medicinal chemistry modification in further search of molecules that could become a novel therapeutic for HD, and potentially a range of neurological disorders.

“I want to thank the funding and intelligent support from the Harrington Discovery Institute, specifically the Therapeutics Development Center and the Project Manager, in our project.”
- Xin Qi, PhD

Click here to read the full article in Nature