Willeke M.C. van Roon-Mom, PhD

I am a molecular biologist at Leiden University Medical Center (LUMC). I focus on rare hereditary brain disorders that have aberrant protein aggregation as hallmark pathological feature. My research is translational and I work closely together with clinical departments, biotechnology and pharmaceutical companies as well as patient representatives. I am interested in understanding disease mechanisms that can reveal novel targets that can then be used for therapy development.

I completed my study and training as a biologist at the University of Groningen, specialising in Medical Biology.I initially worked on cell transplant therapies for Parkinson’s disease at the University Medical Centre Groningen (UMCG). In 1995, I moved to New Zealand where I did my PhD at the University of Auckland on protein aggregation in Huntington disease, mainly working with post mortem human brain material. After a postdoc of 2 years in Auckland, I moved back to The Netherlands in 2005 to start my own research group at the department of Human Genetics at LUMC. Now, my group is studying different diseases, many of which belong to the group of polyglutamine disorders. We use post mortem human brain tissue, animal models and induced pluripotent stem cell (iPSC) models to unravel disease pathology and develop RNA targeting therapies.  

In addition to my own research group, I am co-director of the Dutch Center of RNA Therapeutics where we want to apply our expertise in the development of RNA targeting therapies to patients with unique mutations that have no outlook on therapies developed through the regular clinical trial paradigm. To this end, we work closely together with European and global partners to overcome hurdles in regards to practical, legal and ethical issues.

I am one of the co-applicants of the CUREQ consortium and I lead WP1. My team is responsible for making the isogenic iPSC lines, standardising the iPSC work within the consortium and implementing our 2D and 3D iPSC assays to CureQ.  I am co-leading WP3 together with Mayke Oosterloo. I will supervise one PostDoc that will study the SCA1 disease landscape in the isogenic iPSC, a technician that is responsible for making the isogenic iPSC lines, and a technician that will coordinate the iPSC lab work. Furthermore, I am co-supervising Angela Santiago Aranda, the CureQ PhD student from the Reits lab.