Embedded Files
RNA Cargo Platforms
RNA therapeutics span an exceptionally broad biophysical space, ranging from short double-stranded siRNA (~20 base pairs) to long and structurally complex mRNA exceeding 10,000 nucleotides. Beyond canonical mRNA and siRNA, a growing repertoire of RNA modalities — including guide RNA (gRNA), transfer RNA (tRNA), long non-coding RNA (lncRNA), and self-amplifying mRNA (saRNA) — has emerged as a powerful foundation for next-generation RNA therapeutics.
At the ART Lab, we investigate the intrinsic properties of diverse RNA cargoes and their interactions with delivery systems to enable precise and effective therapeutic deployment.
Selected Research Highlights:
"Development of mRNA vaccines and their prophylactic and therapeutic applications" Kyuri Lee#, Minjeong Kim#, Yunmi Seo, Hyukjin Lee* Nano Res. 11, 5173–5192 (2018).
"Emergence of synthetic mRNA: In vitro synthesis of mRNA and its applications in regenerative medicine" Hyokyoung Kwon#, Minjeong Kim#, Yunmi Seo, Yae Seul Moon, Hwa Jeong Lee, Kyuri Lee, Hyukjin Lee* Biomaterials. 156, 172-193 (2018).
Programmable Lipid Nanoparticles
Programmable lipid nanoparticles (LNPs) provide a versatile platform for controlling the delivery and biological behavior of RNA therapeutics. At the ART Lab, we design and synthesize novel ionizable lipids and integrate lipid chemistry with formulation engineering to create LNP systems with tunable delivery properties across tissues and cell types.
Through systematic investigation of structure–activity relationships (SAR) and mechanisms of action (MOA), we elucidate how variations in lipid composition and formulation parameters influence cargo-specific delivery, intracellular trafficking, and in vivo performance. These insights enable the rational design of LNPs optimized for diverse RNA modalities, ranging from siRNA-mediated gene silencing to genome editing and protein expression.
By combining chemical innovation with a cargo-centric framework, we aim to establish programmable LNP platforms that expand the therapeutic reach of RNA medicines across organs, tissues, and disease contexts.
Selected Research Highlights:
"Novel piperazine-based ionizable lipid nanoparticles allow the repeated dose of mRNA to fibrotic lungs with improved potency and safety" Minjeong Kim#, Michaela Jeong#, Gyeongseok Lee, Yeji Lee, Jeongeun Park, Hyein Jung, Seongeun Im, Joo-Sung Yang, Kyungjin Kim, Hyukjin Lee * Bioeng Transl Med. 8, e10556 (2023).
"Engineered ionizable lipid nanoparticles for targeted delivery of RNA therapeutics into different types of cells in the liver" Minjeong Kim, Michaela Jeong, S. Hur, Y. Cho, J. Park, H. Jung, Y. Seo, H. A. Woo, K. T. Nam, K. Lee, Hyukjin Lee* Sci. Adv. 7, abf4398 (2021).
Disease Modeling & Translational RNA Therapeutics
Many rare and genetic diseases remain inadequately treated due to the intrinsic limitations of conventional small-molecule drugs, which often fail to modulate complex or previously “undruggable” molecular targets. RNA therapeutics offer a fundamentally different paradigm, enabling direct and precise regulation of disease-causing genes and pathways.
At the ART Lab, we develop RNA therapeutic strategies grounded in disease-relevant models, with a particular focus on rare and genetic disorders where unmet medical needs are greatest. By leveraging advanced delivery platforms and cargo-specific design principles, our research aims to translate molecular insights into therapeutic modalities capable of meaningful clinical impact.
Situated within a medical school environment, the ART Lab works in close collaboration with outstanding physician-scientists and clinical researchers at the Catholic Medical Center. These partnerships enable the integration of clinically informed disease models, patient-relevant endpoints, and translational perspectives early in the research process. Through this bench-to-bedside approach, we strive to develop RNA therapeutics that move beyond proof-of-concept studies and bring innovative treatments closer to patients.
Selected Research Highlights:
"Dual SORT LNPs for multi-organ base editing" Minjeong Kim, Eunice S. Song, Joseph C. Chen, Sumanta Chatterjee, Yehui Sun, Sang M. Lee, Shiying Wu, Priyanka Patel, Zeru Tian, Ariel Kantor, Brandon A. Wustman, David J. Lockhart & Daniel J. Siegwart* Nat. Biotechnol. (2025). 10.1038/s41587-025-02675-z
Nature Biotechnology Research Briefing highlight article entitled "Delivering base editors to the liver and lungs in alpha-1 antitrypsin deficiency" Nat. Biotechnol. (2025). https://doi.org/10.1038/s41587-025-02705-w
The Dallas Morning News article entitled "Rescued by fat bubbles: UTSW scientists treat rare genetic disease with designer molecule" Jul. 10, 2025
"In vivo delivery of CRISPR-Cas9 using lipid nanoparticles enables antithrombin gene editing for sustainable hemophilia A and B therapy" Jeong Pil Han#, MinJeong Kim#, Beom Seok Choi#, Jeong Hyeon Lee, Geon Seong Lee, Michaela Jeong, Yeji Lee, Eun-Ah Kim, Hye-Kyung Oh, Nanyeong Go, Hyerim Lee, Kyu Jun Lee, Un Gi Kim, Jae Young Lee, Seokjoong Kim, Jun Chang, Hyukjin Lee*, Dong Woo Song*, and Su Cheong Yeom* Sci. Adv. 8, abj6901 (2022).
Page updated
Google Sites
Report abuse