Principal Investigator: Dr. Willeke van Roon-Mom
Location: Leiden University Medical Centre, Leiden, The Netherlands
Year Founded: 1995
What disease areas do you research?
- SCA1
- SCA3
- Huntington’s Disease
- Dutch – Cerebral Amyloid Angiopathy
- Alzheimer Disease
What models and techniques do you use?
- Mouse Models
- hiPSC-derived stem cells models
- Post-mortem patient tissue
- Antisense oligonucleotides
- RNA sequencing
- Behavioural assays
- Imaging and molecular biology
Research Focus
What is your research about?
The Neuro-D research group studies how diseases develop and progress at the molecular level in several neurodegenerative diseases. They focus on diseases that have protein aggregation, where the disease proteins clump up into bundles in the brain and don’t work correctly.
We focus strongly on translational research, meaning we try to bridge the gap between research happening in the laboratory to what is happening in medical clinics. To do this we use more “traditional” research models like animal and cell models. But we also use donated patient tissues and induced pluripotent stem cell (iPSC) models, which is closer to what is seen in medical clinics.
Our aim is to unravel what is going wrong in these diseases, then discover and test potential novel drug targets and therapies.
One thing we are doing to work towards this goal is identifying biomarkers to measure how diseases progress over time. To do this, we use sequencing technology and other techniques to look at new and past data from patients.
Why do you do this research?
So far there are no therapies to stop the progression of ataxia. If we can understand what is happening in diseases in individual cells, we can develop therapies that can halt or maybe even reverse disease progression.
Identifying biomarkers is also important, because it will help us figure out the best time to treat patients when we eventually have a therapy to test.
Are you recruiting human participants for research?
Yes, we are! We are looking for participants for a SCA1 natural history study and biomarker study. More information can be found here. Please note that information about this study is only available in Dutch.
Fun Fact
All our fridges and freezers have funny names like walrus, seal, snow grouse and snowflake.
For More Information, check out the Neuro-D lab Leiden website!
Written by Dr. Ronald Buijsen, Edited by Celeste Suart
