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Current lipid nanoparticle (LNP) drug delivery technologies are formulated with ingredients such as cholesterol that are destined to accumulate in the liver, which is why many established genetic medicines (such as gene therapy, mRNA vaccines and gene editing technologies) are excreted before they reach their targets. If you want to treat brain diseases or lung diseases, you don't want the delivered drugs to go to the liver. This requires the development of solutions that can target drugs to the right tissues and cells.
The 2024 Nobel Prize in Physiology or Medicine was first announced on October 7. The winners are Victor Ambros and Gary Ruvkun. They won the prize for their discovery of microRNA and its role in post-transcriptional gene regulation.
Recently, researchers from Central South University in China and other institutions published a research paper titled "Human papillomavirus-encoded circular RNA circE7 promotes immune evasion in head and neck squamous cell carcinoma" in the journal Nature Communications. The study found that human HPV virus downregulates the expression of the immune checkpoint molecule Galectin-9 by encoding circular RNA, circE7, thereby promoting immune escape in head and neck squamous cell carcinoma (HNSCC). Based on this discovery, a new idea of combining TIM-3 (Galectin-9 receptor on T cells) monoclonal antibody with existing PD-1 monoclonal antibody to improve the immunotherapy effect of head and neck squamous cell carcinoma was proposed.
Recently, researchers from the Broad Institute of MIT and Harvard University published a research paper titled "Systematic multi-trait AAV capsid engineering for efficient gene delivery" in Nature Communications. The study developed a general machine learning method for systematically designing multi-feature AAV capsids, Fit4Function, which generates reproducible screening data by utilizing AAV capsid libraries that uniformly sample the manufacturable sequence space to train accurate sequence-to-function models, thereby helping to design AAV protein shells (capsids) with multiple ideal features, such as the ability to deliver genes to specific organs or achieve gene delivery in multiple species, thereby helping to accelerate the engineering of AAVs for gene therapy.
Recombinant adeno-associated virus (rAAV) vector is one of the most promising viral vectors in gene therapy. Currently, three-plasmid transfection based on human embryonic kidney 293 (HEK293) cells is the most commonly used rAAV vector production system, but its low production efficiency has become one of the challenges faced by rAAV gene therapy drugs on the road to commercialization. In view of this challenge, researchers are working to develop improved methods to increase the production of rAAV in HEK293 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.
Hematopoietic stem and progenitor cells (HSPCs) spend their lives making mature blood cells. In the process, HSCs interact with a variety of cells in the bone marrow microenvironment, including macrophages. These macrophages mediate a range of processes by releasing cytokines and chemokines and patrolling to remove stressed, dead or aged cells. In this way, macrophages contribute to the maintenance of tissue homeostasis.
Recently, the research team of Maike Hofmann and Robert Thimme from the University of Freiburg in Germany published an article titled "Attenuated effector T cells are linked to control of chronic HBV infection" in Nature Immunology, and found that in chronic Hepatitis B virus (HBV) infection, in addition to classic CD8+T cell exhaustion, effector CD8+T cell attenuation also occurs. HBV-specific CD8+T cell attenuation is manifested by cytotoxic characteristics and inhibition of effector differentiation programs. This process is regulated by antigen recognition and TGFβ signaling and is associated with viral control in chronic HBV infection.
