Rate My Professor Younbok Lee

As Principal Investigator, Lee leads multiple funded projects, including 'Developing AAV Gene therapy for C9orf72 ALS/FTD' (My Name'5 Doddie Foundation, 2024-2027), 'Accelerating the proof-of-concept study for FUS AAV gene therapy' (My Name'5 Doddie Foundation, 2025), 'Developing AAV gene therapy for Motor Neuron Disease' (MNDA, 2023-2026), and 'AAV gene therapy for mutant FUS Amyotrophic Lateral Sclerosis' (LifeArc, 2022-2024). He co-founded AviadoBio, a King's College London spin-out in 2021 with Professor Christopher Shaw and Dr. Do Young Lee, where he serves as Vice President and Head of Discovery, advancing therapies like AVB-101 for FTD, optioned exclusively to Astellas in 2024. Key publications include 'Hexanucleotide repeats in ALS/FTD form length-dependent RNA foci, sequester RNA binding proteins, and are neurotoxic' (Cell Reports, 2013), 'Differential roles of the ubiquitin proteasome system and autophagy in the clearance of soluble and aggregated TDP-43 species' (Journal of Cell Science, 2014), 'C9ORF72 repeat expansion causes vulnerability of motor neurons to Ca2+-permeable AMPA receptor-mediated excitotoxicity' (Nature Communications, 2018), 'Disruption of ER-mitochondria tethering and signalling in C9orf72-associated amyotrophic lateral sclerosis and frontotemporal dementia' (Aging Cell, 2022), and 'Stimulating VAPB-PTPIP51 ER-mitochondria tethering corrects FTD/ALS mutant TDP43 linked Ca2+ and synaptic defects' (Acta Neuropathologica Communications, 2024). With over 3700 citations, his contributions advance therapeutic development in neurodegeneration.