Huntington’s disease belongs to a group of diseases called repeat associated diseases. We believe that the malfunction of the translation machinery is the underlying cause of this disease as well as for other repeat associated diseases.
Read more about Repeat associated diseases
Huntington’s disease (HD) is a dominantly transmitted neurodegenerative disorder with an average age at onset of 40 years. It is associated with extended repeats of glutamine (repeat of the trinucleotide CAG) in a protein called Huntingtin (HTT). Normally, the CAG segment is repeated 10 to 35 times within HTT, but when repeats are higher than 100, disease occurs. The exact mechanism which causes disease due to extended CAG repeats is unclear. Currently, there is no treatment that can stop or reverse the course of HD.
Using Anima’s PSM technology we can see that there is a general translation defect in cells expressing mutant HTT. We have developed three different assays towards the discovery of molecules which will alleviate translation arrest in extended-HTT expressing cells.
Anima’s discovery program uses our own novel technology to discover compounds that correct translation malfunctions.