ICKSMCB 2021 / 2021 International Conference of the Korean Society for Molecular and Cellular Biology / November 3 - 5, 2021 / ICC JEJU

Award Lecture

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KSMCB Presidential Lecture Award

November 4 (Thu), 17:30-18:10, Tamna Hall (5F)

Chair: Yong Tae Kwon, Ph.D. (Seoul National University, Korea)

Toward a cure for Parkinson¡¯s disease: from bench to bed

Kwang-Soo Kim, Ph.D.
McLean Hospital, Harvard Medical School, USA

Parkinson¡¯s disease (PD) is a neurodegenerative disorder associated with age-dependent loss of midbrain dopamine (mDA) neurons in the substantia nigra. Based on his >30 years¡¯ investigation of the transcriptional cascade of mDA neurons, Prof. Kim¡¯s lab is focusing his efforts on translational research to develop novel therapeutic avenues for PD. First, he identified the orphan nuclear receptor Nurr1 as a promising drug target for mechanism-based neuroprotective therapeutics. Although Nurr1 was viewed as a ligand-independent, constitutively active transcription factor, Prof. Kim identified both synthetic and native ligands that directly bind to Nurr1 to robustly activate its transcriptional functions as both activator and repressor. Using both in vitro and in vivo studies, Prof. Kim showed that these ligands can prominently ameliorate motor defects in rodent models of PD in a neuroprotective manner without dyskinesia-like side effects, rendering them to be promising drug candidates. Second, the unique loss of mDA neurons observed in PD makes it a promising disease for cell replacement therapy. As a first step towards personalized cell therapy for PD, Prof. Kim established a platform of core techniques to successfully treat a PD patient using his own skin cell-derived dopamine cells. In this lecture, Prof. Kim will discuss how basic molecular studies can be translated into novel therapeutic approaches for PD, demonstrating a proof-of-principle of ¡°bench to bed side¡± approach.