The challenges facing scientists and clinicians developing treatments for Huntington’s disease (HD) are considerable, and will not stop when a treatment has been found. Any useful therapy must also be delivered safely and effectively. Delivering drugs or other treatments, such as gene therapies, that need to be delivered directly into the brain, is going to be particularly challenging. The brains of mice, in which many therapies are developed, are very small and easy to access. By contrast, the human brain is large, and so good distribution of drugs or gene therapy is not guaranteed. An intermediate species suitable for testing therapies is needed. Candidate species are non-human primates and farm animals, such as sheep and pigs.
Sheep are an unusual species for studying HD, but they have a particular advantage as a stepping stone species for ‘scaling up’ therapies – they have large brains. They also have a major disadvantage. They have rarely been used for studying brain function and behaviour, so there are no established methods for testing effectiveness of treatment.
In this presentation, I will discuss the challenges of using a new species for studying HD. I will give an overview of ongoing studies, to show how sheep can be used a large animal model for testing efficacy and delivery of therapies for HD.
- Sheep Huntington’s disease behaviour