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Neville E. Sanjana's team at New York University published a research paper titled "Transcriptome-scale RNA-targeting CRISPR screens reveal essential lncRNAs in human cells" in the international academic journal Cell. The study developed a transcriptome-scale CRISPR screening technology based on CRISPR-Cas13 targeting RNA, and used this technology to screen and identify 778 essential lncRNAs in 5 human cells from different tissues, indicating that many lncRNAs are not junk, but play an essential and important role in human cancer and development.
In October 2024, Victor Ambros and Gary Ruvkun were awarded the Nobel Prize in Physiology or Medicine for their discovery of the central role of microRNA (miRNA) in gene expression. This discovery in 1993 revealed that miRNA regulates gene expression by binding to target mRNA and inhibiting its translation. Since then, the important role of miRNA in multiple gene expression pathways such as cell differentiation, proliferation and survival has gradually been recognized. However, although the biological functions of miRNA have been well studied, the road to applying miRNA in clinical treatment is still long and challenging.
The team led by Yin Hao from Wuhan University in China published a research paper titled "Rapid generation of long, chemically modified pegRNAs for prime editing" in the journal Nature Biotechnology. The study used RNA ligation technology to successfully break through the length barrier of chemically synthesized RNA and efficiently prepared high-purity chemically modified pegRNA (125-145 nt) and engineered pegRNA (epegRNA) (170-190 nt). The L-epegRNA prepared by this method showed excellent editing efficiency in multiple cell lines and two primary cells.
In 2005, Inagaki et al. reported for the first time that a large number of mRNA-like long non-coding RNAs were expressed in a tissue- and cell-specific manner in model species. In 2008, Mercer et al. used in situ hybridization to identify the expression of a large number of long non-coding RNAs in mouse brain tissues, and confirmed that the expression levels of these long non-coding RNAs were related to specific neuroanatomical locations, cell types, and subcellular locations. After 2010, as the academic community continued to heat up its research on IncRNA, the level of related basic research has advanced by leaps and bounds, and has gradually begun to shift from academia to industry. However, due to the short history of lncRNA research, lncRNA drug development is still in its early stages.
Recently, a research team from Zhejiang University in China published a research paper titled "Compact RNA editors with natural miniature Cas13j nucleases" in the journal Nature Chemical Biology. Data-driven protease mining can use the diversity of species resources to mine "new proteins". This study used BT-IT fusion technology to conduct large-scale mining of microbial metagenomic data through intelligent mining algorithms, discovered extremely small Cas13j proteins and performed efficient in vivo RNA editing.
Cancer remains the leading cause of death worldwide, and traditional treatment modalities such as chemotherapy and radiotherapy are often associated with recurrence risk and severe side effects. Gene therapy for cancer has gained increasing prominence due to its personalized capabilities. However, in order to effectively treat cancer through gene therapy, it is crucial to accurately target tumor cells, deliver tumor suppressor genes, or disrupt oncogenes using tools such as RNAi or CRISPR, as imprecise targeting may increase the risk of recurrence. Recent advances have shown that lipid nanoparticles (LNPs) are powerful carriers of siRNA therapies, showing potential in the management of genetic diseases.
Researchers from the U.S. Army Medical Research Institute of Infectious Diseases and Moderna recently published a research paper titled "Comparison of protection against mpox following mRNA or modified vaccinia Ankara vaccination in nonhuman primates" in the journal Cell. The study compared the effectiveness of modified vaccinia Ankara (MVA) and mRNA-1769 vaccines in non-human primates. The results showed that, similar to MVA, mRNA-1769 produced a protective effect against monkeypox virus attack and further reduced symptoms and course of the disease. Compared with MVA, mRNA-1769 enhanced viral control and disease reduction, highlighting the potential of mRNA vaccines in reducing the threat of future pandemics.
Niren Murthy from the University of California, Berkeley, Aijun Wang from the University of California, Davis, and others published a research paper titled "Acid-degradable lipid nanoparticles enhance the delivery of mRNA" in Nature Nanotechnology. The study developed an acid-degradable linker called "azido-acetal", which was used to synthesize degradable lipids composed of polyethylene glycol lipids, anionic lipids, and cationic lipids, and based on this, LNPs (RD-LNPs) that hydrolyze rapidly in endosomes were synthesized. In in vitro and in vivo experiments, RD-LNP significantly improved the performance of LNP-mRNA complexes. Compared with traditional LNPs, mRNA is more effectively delivered to the liver, lungs, spleen, and brain of mice, as well as hematopoietic stem/progenitor cells in vitro.
Hyperuricemia (HU) is a metabolic disease caused by high serum uric acid (SUA) levels due to insufficient renal excretion, excessive production, or insufficient intestinal excretion. Various conventional treatments are commonly used to treat HU, such as valproic acid and allopurinol (xanthine oxidase inhibitor). Valproic acid increases urination, thereby enhancing the excretion of urate crystals, while allopurinol relieves symptoms by reducing uric acid production by inhibiting xanthine oxidase. However, increased urination increases the excretion of urate crystals, leading to kidney damage.
Breast cancer remains one of the most challenging cancers, mainly due to its heterogeneity and metastatic propensity. The Notch signaling pathway is required for multiple cellular processes and is involved in the development and progression of breast cancer. Noncoding RNAs (ncRNAs) have now become key regulators of gene expression, which can affect the biology of cancer by interacting with the Notch signaling pathway. Noncoding RNAs include microRNAs, long noncoding RNAs (lncRNAs), and circular RNAs (circRNAs). Recently, in a review report published in the international journal Gene Expression, entitled "Non-coding RNAs Affect Breast Cancer Development Through the Notch Signaling Pathway: An Overview", researchers from Tabriz University outlined the importance of ncRNAs in human breast cancer and their potential as therapeutic targets, paving the way for the development of innovative therapies to significantly impact patient care and prognosis.
