Synthetic RNA Therapeutics and Ribodiagnostics

Learning Outcomes

This course explores novel approaches in synthetic RNA therapeutics and ribodiagnostics, including RNA antagonists, chemically modified RNA, and synthetic circular RNA. It covers machine learning approaches for synthetic RNA signatures as diagnostic or therapeutic biomarkers, advanced delivery strategies such as nanoparticles and liposomes, as well as design principles that enhance stability, specificity, and efficacy. Students gain a foundational understanding of the technologies and strategies enabling the development of next-generation RNA-based medicines, with a view towards synthetic translational ribomedicine.

Upon completion the students will be able to:

• Interpret diagnostic molecular signatures and link them to biological pathways.
• Critically assess challenges and limitations of ML-driven ribodiagnostics and propose improvements.
• Explain the biological functions and advantages of circRNAs in gene regulation and disease treatment.
• Compare strategies for constructing circRNAs and promoting back-splicing.
• Compare and contrast different synthetic drug delivery systems, including viral, lipid-based, polymeric, and hybrid platforms.
• Evaluate strategies for organ- and cell-specific targeting in therapeutic delivery.
• Apply principles of synthetic biology and translational nanomedicine to design or optimize delivery systems for specific therapeutic applications.
• Explain the molecular principles and design strategies behind mRNA vaccines, RNA aptamers, and gene replacement therapies.
• Describe the mechanisms and applications of cell reprogramming for regenerative medicine.
• Analyze real-world case studies of advanced therapeutics in clinical and preclinical contexts.
• Identify challenges and propose solutions for improving efficacy, safety, and scalability of RNA- and gene-based therapies.

Module Syllabus

• Machine Learning Analysis of Transcriptome Data for synthetic ribodiagnostic signatures
• Synthetic circular RNAs and ribotherapeutics
• Synthetic Drug Delivery Systems: From Viruses to Liposomes and Nanosomes
• Advanced RNA and Gene-Based Therapeutics: from mRNA vaccines to gene replacement and cell therapy
• Synthetic Therapeutics for Neurodegenerative Diseases

Suggested Bibliography

• Relevant literature per lecture, including scientific publications and reviews from international journals, which is available in the course e-class.
• Principles and techniques of Biochemistry and Molecular Biology, 7th edition, Edited by Keith Wilson and John Walker, electronic source
• Laboratory techniques in biochemistry and molecular biology, Elsevier, ScienceDirect, electronic source