Development of a Non-Human Primate Model for the Identification and Development of Novel Therapeutics for Dystonia
Abstract
During the research period we have developed a quantitative assay (Western blot) to measure torsinA in macaque in the deep cerebellar nuclei and cerebellar tissue and have also synthesized the AAV vector that we will use to knockdown torsinA and demonstrated that it efficiently knocks down torsinA in vitro. Therefore, we successfully developed the tools that we require to progress the project.We have gained IACUC approval for the non-human primate (NHP) studies and purchased 12 macaques. By injecting gadolinium followed by MRI scans, we have shown that we can successfully target the deep cerebellar nuclei. All 12 NHPs have now been injected with AAV vector, the animals left 2 months, and behavioral data collected over this period. Animals have now been killed and deep cerebellar nuclei tissue and cerebellar tissue successfully collected. This tissue is currently being analyzed to determine the degree of torsinA knockdown. The behaviour of the NHPs is also being analyzed.The project is currently on track with the agreed Statement of Work and data on the degree of torsinA knockdown will be available by month 14 of the project.
Document Details
- Document Type
- Technical Report
- Publication Date
- Oct 01, 2023
- Accession Number
- AD1220639
Entities
People
- James Koprich
- Jonathan Brotchie
- Patrick Howson
Organizations
- Atuka (Canada)