tFNA + miRNA: A Breakthrough in Ophthalmic Treatment

In 2024, the Nobel Prize in Physiology or Medicine was awarded to American scientists Victor Ambros and Gary Ruvkun for their groundbreaking discoveries in the field of microRNA (miRNA). miRNA, as a small molecule RNA, has revolutionized our understanding of gene regulation. Its critical role in various biological processes has been recognized, opening new directions for disease treatments, including cancer, cardiovascular diseases, and eye conditions. This award not only acknowledges the discovery of miRNA but also highlights its immense potential in precision medicine.

What is microRNA?

microRNA (miRNA) is a small, non-coding RNA molecule, typically around 22 nucleotides in length, and is present in a wide range of organisms, from viruses to humans. Its main function is to regulate gene expression. miRNAs achieve this by binding to messenger RNA (mRNA), selectively controlling the expression levels of mRNA, which directly affects protein production within the cell. This regulatory mechanism allows miRNA to influence various physiological processes, such as cell differentiation, proliferation, apoptosis, and immune responses. When miRNA function is disrupted, it can lead to diseases such as cancer, neurodegenerative disorders, and retinal diseases. Research has shown that miRNAs regulate about one-third of human genes, with the human genome encoding more than 1,000 types of miRNAs, making them key players in many biological processes.

The Power of microRNA: How It Works in Biology

The remarkable feature of miRNA is its ability to precisely regulate gene expression. Each miRNA can target multiple mRNAs, and a single mRNA can be regulated by different miRNAs. This regulatory mechanism ensures the precision of biological responses, playing a crucial role in processes such as organism development, cell differentiation, immune responses, and even tumorigenesis. By regulating gene expression at the post-transcriptional level, miRNA can act as a biological switch, halting disease progression or enhancing therapeutic effects.

Yunhai’s Tetrahedral Framework Nucleic Acids (tFNA) Loaded with microRNA

In the field of ophthalmic treatment, Yunhai Bio is developing an innovative drug that combines microRNA with tetrahedral framework nucleic acids (tFNA). This tFNA-loaded miRNA drug has shown remarkable efficacy in inhibiting retinal neovascularization and protecting the optic nerve, surpassing even aflibercept, a drug considered the gold standard for this condition. Extensive trials have demonstrated that when a specific miRNA is loaded onto a tFNA structure, it effectively inhibits the formation of abnormal blood vessels in the retina, showing great potential for treating retinal diseases such as diabetic retinopathy. The use of tFNA technology to deliver specific miRNAs has also demonstrated therapeutic potential in other ophthalmic areas, including optic nerve damage, glaucoma, and uveal melanoma. Additionally, this technology shows promise in fields such as orthopedics (e.g., osteoarthritis, bone regeneration), dermatology (e.g., psoriasis, skin fibrosis, infectious wound healing), as well as in acute ischemic stroke and cancer therapy.

Compared to traditional drugs, this tFNA-based delivery system offers higher targeting accuracy and lower side effects. The unique structure of tFNA not only enhances the bioavailability of miRNA but also enables sustained release and improved tissue penetration in the eye. This breakthrough offers a safer and more effective alternative for treating retinal diseases.

The awarding of the 2024 Nobel Prize in Physiology or Medicine highlights the importance of microRNA in advancing our understanding of life sciences. The combination of microRNA and tFNA showcases how cutting-edge biotechnology can reshape the future of medicine. Yunhai Bio believes that as the biomedical community continues to explore these fields, these discoveries will lead to more breakthroughs and improvements in human health. Research into miRNA and tFNA not only provides new insights into the complexity of life but also offers new possibilities for treating a wide range of diseases.