Asian Alliance for Stem Cells and Regenerative Medicine (AASCRM) Webinar Series March 2026

2026.3.3

Events

Asian Alliance for Stem Cells and Regenerative Medicine (AASCRM) Webinar Series March 2026

March Session

Stem Cell Research Using Genome Editing Technologies

Date: 24 March 2026 (Tuesday)
Time: 11:00-12:30(SGT, CST)/ 12:00-13:30 (JST, KST)/ 14:00-15:30 (AEDT)
Format: Virtual (Zoom Meeting)
Hosts: TBD (KSSCR)
Zeng Li, National Neuroscience Institute, Singapore (SCSS)

Speaker’s Information

Hyuk-Jin Cha

Seoul National University, Korea

“Mutation-Agnostic Base Editing of the Progerin Farnesylation Site Rescues Hutchinson-Gilford Progeria Syndrome Phenotypes in Neuromuscular Organoids”

Abstract
Hutchinson-Gilford Progeria Syndrome (HGPS) is a rare, fatal premature aging disorder caused by a de novo mutation in the LMNA gene that leads to the production of progerin, a farnesylated, pathogenic form of lamin A. Treatment with farnesyltransferase inhibitors achieves significant yet limited life extension, highlighting progerin farnesylation as a key pathogenic driver of HGPS. In this study, rather than correcting the single pathogenic point mutation, we introduce Farnesylation Amino acid Targeted Editing (FATE), a novel, mutation-agnostic precision genome editing strategy that selectively disrupts the farnesylation site of LMNA. Next-generation sequencing confirmed that FATE exclusively edits the LMNA locus without inducing off-target mutations or affecting other genes encoding farnesylated proteins. Using neuromuscular organoids (NMOs) derived from two isogenic pairs of human pluripotent stem cells (hPSCs) carrying the HGPS mutation (HGPS-hPSCs), we found perinuclear progerin accumulation exclusive to the muscular compartment to be associated with defective formation of DNA damage foci and loss of perinuclear heterochromatin. Notably, applying FATE to HGPS-hPSCs successfully abolished these muscle-specific pathologies in subsequently-derived NMOs. Direct delivery of FATE mRNA into HGPS-NMOs likewise effectively inhibited perinuclear accumulation of progerin and rescued the formation of DNA damage repair foci. These findings demonstrate FATE as a broadly applicable, mutation-agnostic editing approach that targets a fundamental pathogenic mechanism in HGPS and therefore has feasible utility in clinical application.

Bio
Hyuk-Jin Cha, Ph.D., is a Professor at the Seoul National University College of Pharmacy and an expert in cellular signaling and human pluripotent stem cell (hPSC)-based disease modeling. He received his B.S. from Seoul National University (1993–1999) and earned his Ph.D. in Pharmaceutical Sciences from the University of Maryland, Baltimore (1999–2004). Following his doctoral training, he conducted postdoctoral and research appointments at the National Cancer Institute, Harvard University, and Georgetown University. He later served as faculty at Sogang University before joining Seoul National University, where he currently leads research on genome editing, pluripotency regulation, early embryonic development, and partial in vivo reprogramming for tissue regeneration. Dr. Cha has published extensively in leading international journals and serves on the editorial boards of several prominent scientific journals in the fields of stem cell biology and molecular medicine.

Richard She

Nanyang Technological University, Singapore

“Decoding the Genetic Landscape of Human Evolution”

Abstract
The 3-fold expansion of the cerebral cortex is the defining feature of human evolution. This extraordinary transformation occurred over a remarkably short evolutionary time span of ~7 million years. However, the causal mechanisms and pathways that govern size control of the developing brain have not yet been systematically investigated. The focus of my research program will be to use comparative studies of humans and great apes to pinpoint the cellular and molecular principles that led to the emergence of our unique human features. Our approach draws inspiration from classical developmental genetics paradigms such as the Heidelberg screens, which identified many of the key signaling pathways that govern morphogenesis. However, while the core elements of these pathways are highly conserved from fruit flies to humans, unique aspects of human development arise from altered timing, regulation, and complexity. Performing parallel screens in closely related species allows us to identify key species differences in development. We plan to observe the balance of self-renewal and differentiation in neural progenitor cells, the key cell type that gives rise to virtually all neurons. By making perturbations to neuronal progenitor cells at genome-wide scale, we will systematically identify the key pathways that regulate human brain expansion.

Bio
Richard is an Assistant Professor at Nanyang Technological University. As an undergraduate at Princeton, he worked with Eric Wieschaus on cell cycle control in the early Drosophila embryo. At Stanford, he earned a Ph.D. in Chemical and Systems Biology and trained in yeast genetics with Dan Jarosz, using large inbred crosses to fine‑map the DNA variants that give rise to complex traits. He then joined Jonathan Weissman’s lab for his postdoc, where he built CRISPR tools to identify unique species‑specific vulnerabilities in human and chimpanzee stem cells. Richard is a recipient of the Helen Hay Whitney Fellowship and NTU’s Nanyang Assistant Professorship. In his free time, he plays music, tries not to get injured while playing basketball and badminton, and enjoys cooking for friends.

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