Huntington’s Disease
This image shows the brain of a mouse model of Huntington's disease.
What is Hungtington's disease?
Huntington’s disease is a neurodegenerative disorder caused by the expansion of CAG repeats in the Huntingtin (HTT) gene. Accumulation of abnormal HTT protein in neurons is toxic and induces neuronal dysfunction and death. Clinically this leads to chorea initially, followed by difficulties speaking and swallowing, decrease in cognitive abilities, and ultimately neuropsychiatric complications.
Our therapeutic strategy: silencing HTT
We aim to silence HTT using an AAV9 or AAVrh.10-delivered artificial miRNA targeting HTT. For this research program we are partnering with Dr. Neil Aronin, who is a Professor of Medicine at UMass, specialized in Endocrinology. You can visit his lab website here. Dr. Aronin's lab is focused on Huntington's disease, and he collaborates with several groups to develop therapeutics. Our joint program is based on using gene therapy to achieve silencing.
Let's have the next breakthrough together!