**Job Description**
This Ph.D project focuses on developing gene-targeted therapies for newly identified ALS-associated genes, building upon recent breakthroughs in SOD1 gene mutation therapies. The doctoral candidate will acquire diverse skills, including trans-differentiation of fibroblasts into neurons (iNeurons), development and screening of antisense oligonucleotides (AONs), live-cell imaging, and transcriptomic analyses. The work involves optimizing protocols for converting patient-derived skin cells into neurons, screening new AONs, and using advanced techniques to select effective AONs with the ultimate goal of developing a treatable AON for ALS patients. The candidate will collaborate with a multidisciplinary team within UMC Utrecht and external partners like the Dutch Center for RNA Therapeutics and Charles River Laboratories.
**Skills & Abilities**
• Trans-differentiation of fibroblasts into neurons (iNeurons)
• Development of antisense oligonucleotides (AONs)
• Screening of antisense oligonucleotides (AONs)
• Live-cell imaging
• Transcriptomic analyses
• Electrophysiology
• Biomarker analyses
• Collaboration and teamwork
**Qualifications**
Required Degree(s) in:
• Relevant Master’s degree (e.g., Biomedical Sciences, Medicine, Bioinformatics, Statistics)
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