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

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Supervisor: Dr. R. Buijsen 

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

Anticipated starting date: October 1st, 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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