Kia ora everyone,

 

I am Jamie, a nerdy biomedical scientist from Auckland, New Zealand. In high school I decided that I wanted to pursue a career curing diseases. Six years and 18,000 km of flying later I am hoping to use antisense oligonucleotides to make that dream a reality! Both my undergraduate and postgraduate studies were done at the University of Auckland. My postgraduate thesis was completed in the lab of Professor Russell Snell, where I investigated a hypothesis that aberrant ammonia and urea metabolism contributes to the progression of HD.

 

After graduating, the Snell lab had to endure me for another year working as a research assistant where I was able to work on several interesting projects. These included continuing my thesis work, developing transgenic sheep models for retinitis pigmentosa (in true New Zealand fashion, we like to involve sheep wherever possible) and exploring the effects of various environmental factors on telomere length. Finally, I have uprooted my life in New Zealand to pursue my PhD as a part of the EFFecT network in the NeuroD lab group at the LUMC in the Netherlands. Having started on October 16th (three days after I arrived in the Netherlands), I will be working towards developing allele-specific antisense oligonucleotides to treat trinucleotide repeat expansion neurodegenerative disorders.

 

On the few occasions when I am not tinkering away in the lab, I am a massive sports fan. In particular, I am overly invested in the NBA team the Houston Rockets and I also enjoy cricket and rugby. Moreover, I am very excited to travel around Europe and soak up the food and history on offer!

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DC7: Development and evaluation of allele-specific ASOs to treat neurodegenerative repeat expansion disorders

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Supervisor: Dr. R. Buijsen 

External mentor: Dr. S. Alves

Host Institute: Leiden University Medical Center, The Netherlands (www.lumc.nl)

Secondments planned: Biobizkaia Health Research Institute, Spain; Institut de Recherches Servier, France

Doctoral program: PhD Program of the Leiden University Medical Center

Starting date: October 16, 2025

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Project description:

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DC7 will be involved in the research of the NeuroD-group at the LUMC (Neuro-D Lab Leiden). Within our group we focus on the development of ASOs for repeat expansion neurodegenerative diseases. DC7 will design and evaluate allele-specific ASO strategies to target these repeat expansions that result in toxic products. These ASOs will be tested initially in cell-based model systems. Once the best candidates have been identified, they will be tested on in patient-derived 2D neuronal cultures and functional readouts will be measured to evaluate phenotype correction. Finally, the best performing ASO will be optimised and functionally assessed in 3D brain organoids. Altogether, we expect to set up a pipeline/roadmap to develop therapies for these devastating diseases.

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