At the moment, Huntington’s disease is not curable. But researchers like Philip Mitchell, a Scientific Director at Charles River, who works with Diana Miszczuk, Associate Director in CNS Pharmacology at Charles River, and oversees work on Huntington’s disease, are working hard to change this.

The Huntington’s disease research community is very much focused on delaying the onset of the disease or slowing its progression by targeting pathological proteins through multiple approaches.
Philip Mitchell

Therapeutic development for Huntington’s disease has largely focused on small molecules and antisense oligonucleotides (ASO). The latter are small strands of RNA or DNA that can be designed to target the HTT mRNA transcript and prevent the mutant protein from being made.

Working in collaboration with the Cure Huntington’s Disease Initiative (CHDI), a privately-funded, not-for-profit biomedical research organization devoted to developing therapeutics against Huntington’s disease, the team at Charles River recently published an article in the Journal of Medicinal Chemistry describing the optimization of a new class of small-molecule inhibitors that could mitigate Huntington’s disease.

 

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Nature