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Research

Our research focuses on the development of novel RNA technology and their related therapeutic and basic biological applications. 

Innovating mRNA Delivery for Therapies and Vaccines 

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     â€‹The exploration of mRNA in developing novel therapeutic approaches has garnered increasing attention in recent years. Our efforts are primarily focused on enhancing mRNA-based treatments, encompassing a wide array of applications such as vaccines for infectious diseases and cancer, therapeutic protein replacement, and addressing genetic disorders.

A significant challenge in this field is the absence of an effective and precisely targeted intracellular delivery system for mRNA molecules. This gap hinders the successful conversion of mRNA into clinically viable drugs. In response to this, our lab is dedicated to creating the next generation of intracellular delivery systems. Utilizing the advancements in nanotechnology, we are working towards systems that not only exhibit high efficiency but also specificity in the delivery of mRNA in vivo. This endeavor is pivotal in supporting and propelling the development of groundbreaking mRNA-based gene therapies and vaccines, potentially revolutionizing the future of medical treatments.

​Innovation in mRNA/circRNA Manufacturing 

Vaccine Production Line

     In the rapidly evolving field of RNA therapeutics, innovation in mRNA and circRNA manufacturing stands as a critical frontier. Our lab focuses on revolutionizing the production of these RNA molecules, essential for next-generation medical treatments. For mRNA, the challenge lies in achieving high-yield, high-purity synthesis while maintaining the integrity and functionality of the molecule. We employ cutting-edge techniques to streamline the transcription process, ensuring a consistent and scalable production. This is crucial for mRNA vaccines and gene therapies, where the demand for large-scale, high-quality mRNA is ever-increasing. CircRNA manufacturing, on the other hand, requires innovative approaches to achieve its unique circular structure. Our research delves into novel circularization methods that enhance the stability and efficiency of circRNA, making it a viable tool for gene regulation therapies and biomarker discovery. By advancing these manufacturing techniques, we aim to lower production costs, increase accessibility, and improve the efficacy of RNA-based therapies. This commitment to innovation not only propels the field of RNA therapeutics forward but also holds the potential to transform patient care, opening new avenues for treatment in various diseases and conditions.

​Understanding mRNA/circRNA molecular structure for optimizing mRNA/circRNA sequence design
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     In our lab, we're dedicated to understanding the molecular structures of mRNA (messenger RNA) and circRNA (circular RNA), essential for advancing RNA-based therapies and research. The linear structure of mRNA, crucial in transferring genetic information from DNA to protein synthesis machinery, includes key elements like the 5' cap, coding sequence, and poly(A) tail. Optimizing these components is vital for improving mRNA's stability and translational efficiency, enhancing its therapeutic potential as seen in mRNA vaccines and gene editing techniques.

Meanwhile, circRNA, with its unique closed-loop structure, is significant in gene regulation and offers promising prospects in diagnostics and therapeutics. This circular form provides greater stability and resistance to degradation. Our research focuses on unraveling circRNA's structure-function relationship to develop molecules with precise regulatory functions.

These endeavors in understanding mRNA and circRNA structures not only deepen our insight into RNA biology but also drive innovations in medical applications. Through our work, we're contributing to the development of tailored RNA-based treatments and diagnostic tools, marking a significant stride in personalized medicine and targeted therapies.

Developing novel applications for RNA  technology (in vivo CART)

Image by National Cancer Institute

      We are pioneering RNA-based strategies to enable in vivo generation of CAR-T cells, eliminating the need for complex and costly ex vivo cell engineering. By combining circular RNA design with targeted lipid nanoparticle (LNP) delivery, our approach allows direct reprogramming of T cells inside the body to express chimeric antigen receptors. This platform offers a safer, faster, and more accessible path toward next-generation immunotherapies for cancer and autoimmune diseases.

©2025 by Yuhong Cao Research.

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