Center for RNA Biology and Therapeutics
The Center for RNA Biology and Therapeutics at City of Hope® is a premier research center dedicated specifically to RNA biology and RNA-based therapeutics. It serves as a dynamic hub for multidisciplinary collaboration, bringing together scientists and physicians to drive breakthroughs in RNA science and pioneering innovative treatments for cancer, diabetes, and other human diseases.
Led by Jianjun Chen, Ph.D., Simms/Mann Family Foundation Chair, Professor and Chair of the Department of Systems Biology, the Center is dedicated to advancing scientific excellence in RNA biology through discovery, innovation, and collaboration. Our mission is to uncover fundamental molecular mechanisms that govern RNA biology, including RNA biogenesis, modification, regulation, and function, and harness these insights to develop groundbreaking diagnostics and therapies. By fostering a multidisciplinary and collaborative environment, the Center seeks not only to expand the frontiers of RNA science but also to translate these discoveries into transformative treatments that improve patient lives worldwide.
Research Focus
The Center’s research spans the full spectrum of RNA biology and translational applications, including:
- RNA biology: spanning the full life cycle of RNA, from transcription, splicing, editing, and transport to translation, degradation, and localization. Our research investigates multiple RNA species, including mRNA, tRNA, rRNA, and diverse non-coding RNAs (lncRNAs, miRNAs, snoRNAs, and circRNAs), with particular emphasis on chemical modifications (e.g., m⁶A, Ψ, m⁵C, m⁷G, Ac⁴C), RNA structural dynamics, and interactions with RNA-binding proteins and ribonucleoprotein complexes that regulate gene expression, cellular functions, and disease pathogenesis.
- RNA-based diagnostics: Leveraging RNA modification profiles and non-coding RNA signatures as sensitive and specific biomarkers to inform disease prognosis, predict therapeutic response, monitor residual disease, and guide personalized treatment strategies. These RNA signatures, detectable in patient samples such as blood, bone marrow, and circulating cell-free RNA, offer minimally invasive approaches for early detection, real-time monitoring, and outcome prediction across cancers and other diseases.
- RNA-based therapeutics: represent a transformative frontier in medicine, harnessing the versatility of RNA to modulate gene expression, reprogram immune responses, and correct disease-driving pathways. From small molecules and degraders that target dysregulated RNA machineries, to mRNA vaccines and protein replacement therapies that enable rapid, customizable treatments, RNA is reshaping how we approach both cancer and genetic disease. Engineered RNA platforms are also empowering next-generation cell therapies, including CAR-T, CAR-NK, and CAR-macrophage therapies, while RNA editing technologies such as ADAR- and CRISPR–Cas13-based platforms open the door to precise, reversible correction of pathogenic transcripts without altering the genome. Complementary modalities such as siRNAs, antisense oligonucleotides (ASOs), and small activating RNAs (saRNAs) further expand the therapeutic toolbox, collectively positioning RNA as a versatile and dynamic foundation for personalized and precision medicine.
The Power and Potential of RNA Research
While DNA encodes the blueprint of life, RNA executes and regulates these instructions through complex and dynamic processes. Beyond messenger RNAs (mRNAs), non-coding RNAs (ncRNAs) vastly outnumber protein-coding RNAs and perform critical but not yet fully understood functions in gene regulation and cellular biology.
Over 170 chemical RNA modifications have been identified in mRNA, tRNA, rRNA, and ncRNA. Aberrations in these modifications are increasingly recognized as drivers of cancer, diabetes, metabolic disorders, and therapeutic resistance. Understanding these processes opens opportunities for both fundamental discoveries and translational breakthroughs.
RNA research at City of Hope holds tremendous promise because of RNA’s central role in biology, its dynamic and reversible regulation, and its unique potential as a therapeutic target or tool. RNA signatures may enable early disease detection and prediction of therapeutic response, while RNA machineries themselves represent novel drug targets.
City of Hope investigators are advancing knowledge of RNA’s role in cancer initiation, progression, metabolism, metastasis, immune response, therapeutic resistance, and relapse, and are leveraging the knowledge to develop cutting-edge RNA-based therapies. Current clinical efforts already include RNA-based therapeutic strategies for liver, breast, lung, and brain cancers.
The Center for RNA Biology and Therapeutics at City of Hope is committed to building a collaborative and innovative research community that pushes the boundaries of RNA science, accelerates the development of RNA-based/targeted diagnostics and therapies, and transforms patient care.