KAIST GSMSE Synthetic & Programmable Advanced Cell Engineering Lab
SPACE Lab

Our Question

How can we cure genetic diseases?

Diagram showing two paths from a disease-causing mutation to a cure: correcting the mutation in vivo or ex vivo, and investigating the mutation to reveal its mechanism and develop mutation-specific drugs.
Figure 1Two paths from a disease-causing mutation to a cure — correcting it directly, or investigating it to guide mutation-specific treatment.
01

Cell & Gene Therapy

Context-aware genome editing tools — tailored to each cellular context for greater precision, efficiency, and safety.

Cell Context × Genome Editing × Artificial Intelligence

We investigate how cellular states and cell-type-specific biology shape the efficiency, precision, and outcomes of genome editing. We use generative AIs to control cellular factors that determine genome editing outcomes. AI-designed de novo proteins are integrated with next-generation editors to create genome editing systems optimized for specific cellular contexts.

Cell & Gene Therapy

We translate context-aware genome editing into therapeutic strategies. From long-DNA replacement in patient-derived iPSCs to organ-specific genome editing, we aim to develop next-generation ex vivo and in vivo therapies capable of correcting diverse disease-causing mutations.

Roadmap of context-aware genome editing research: Step 1, understanding hPSC characteristics in base editors and prime editors; Step 2, developing hPSC-specialized base and prime editors; Step 3, AI-driven development of precise genome editing tools, with supporting figures from JC Park et al., Molecular Therapy Nucleic Acids 2022 and 2023, Nature Communications 2024, and Cell 2025.
Figure 2Development of context-aware genome editing — from characterizing hPSC-specific editing outcomes to AI-driven design of precision editing tools. J.C. Park et al., Molecular Therapy – Nucleic Acids, 2022 & 2023; Nature Communications, 2024; Cell, 2025.
02

Disease Modeling & Drug Discovery

A variomics platform built on human pluripotent stem cells — modeling genetic disease and powering mutation-specific drug discovery.

hPSC Variomics

We develop scalable genome engineering platforms that introduce large libraries of genetic variants into human pluripotent stem cells. This enables systematic and quantitative analysis of disease-associated variants in genetically controlled human models.

Mutation-Specific Drug Discovery

Single-cell multi-omics and phenotypic screening connect individual genetic variants with disease mechanisms and therapeutic responses. By resolving how different mutations within the same disease gene respond to treatment, we aim to enable mutation-specific therapeutic discovery and precision medicine.

Diagram of the variomics platform: introducing barcoded gene-of-interest mutant libraries into hPSCs via precise genome editing, differentiating the edited cells into neurons, then analyzing them by single-cell multi-omics and drug screening.
Figure 3Development of a variomics platform for mutation-specific disease mechanisms and drug discovery — from barcoded variant libraries in hPSCs, through differentiation, to single-cell multi-omics analysis and drug screening.