Rate My Professor Younbok Lee

Dr. Younbok Lee is a Senior Lecturer in the Department of Basic and Clinical Neuroscience within the School of Neuroscience at King's College London, part of the Institute of Psychiatry, Psychology and Neuroscience. His research focuses on noncoding RNAs and RNA binding proteins, including heterogeneous nuclear ribonucleoproteins (hnRNPs), and their roles in neurodegenerative disease progression, particularly frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS). He investigates post-transcriptional gene expression regulation and disruptions in mRNA processing that contribute to these disorders. Lee develops gene therapy strategies targeting mutations such as C9orf72 and FUS, employing AAV vectors for delivery in preclinical models. His work addresses RNA misprocessing, protein aggregation, ER-mitochondria tethering defects, synaptic dysregulation, and excitotoxicity in motor neurons.

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.