**Job Description**
This Ph.D. project focuses on developing gene-targeted therapies for newly identified ALS-associated genes, building on the recent success of therapies for specific SOD1 gene mutations. The doctoral candidate will acquire a broad skill set, including trans-differentiation of fibroblasts into neurons (iNeurons), development and screening of antisense oligonucleotides (AONs), live-cell imaging, and transcriptomic analyses. The ultimate goal is to translate an effective AON into a treatment for ALS patients.
**Skills & Abilities**
• Trans-differentiation of fibroblasts into neurons (iNeurons)
• Development and screening of antisense oligonucleotides (AONs)
• Live-cell imaging
• Transcriptomic analyses
• Reporter assays
• RNA sequencing
• Automated live-cell imaging
• Electrophysiology
• Biomarker analyses
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