Gene therapy is a powerful and innovative approach for patients using molecular biology tools to engineer genetic materials. This revolutionary therapy holds promise for treating a wide range of diseases, from rare genetic disorders to neurodegenerative diseases. Our team is developing gene therapy trials for FTD/ALS and multiple types of gene therapy vectors and delivery strategies. We are also involved in regulatory and operational challenges of gene therapy trials, including early discovery, vector science, and pre-clinical studies. We have a strong Industry partnership with King’s College London spinout company AviadoBio. As I am a co-founder of the AviadoBio (vice president and Head of discovery), our lab is developing transformative medicines with industry partners for patients with debilitating and life-threatening neurodegenerative disorders.

Please see my Research Staff Profile for more detail.

 

Key Publications:

• Youn-Bok Lee, Emma L. Scotter, Do-Young Lee, Claire Troakes, Jackie Mitchell, Boris Rogelj, Jean-Marc Gallo, Christopher E. Shaw, 2021, Cytoplasmic TDP-43 is involved in cell fate during stress recovery, Human Molecular Genetics

• Keith Mayl, Christopher E. Shaw, Youn-Bok Lee, 2021, Disease mechanisms and therapeutic approaches in c9orf72 als-ftd, Biomedicines

• Lee et al., 2017. C9orf72 poly GA RAN-translated protein plays a key role in amyotrophic lateral sclerosis via aggregation and toxicity Human Molecular Genetics.

• Lee et al., 2013. Hexanucleotide Repeats in ALS/FTD Form Length-Dependent RNA Foci, Sequester RNA Binding Proteins, and Are Neurotoxic. Cell Reports.

• Lee et al., 2009.Twist-1 regulates the miR-199a/214 cluster during development. Nucleic Acids Research.

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Key collaborators:

• Professor Amit Nathwani, University College London
• Professor Jernej Ule, University College London
• Professor Christopher Shaw, King's College London
• Professor James Uney, University of Bristol