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Imagine if there was a technology that could directly modify our DNA to cure or prevent genetic diseases that are currently untreatable. This is no longer a plot in science fiction, but a reality with gene editing therapy. By precisely editing the genome, we can correct or eliminate problematic genes and provide long-lasting treatments for genetic diseases.
TCF1high progenitor CD8+ T cells can mediate the efficacy of immunotherapy, but researchers are currently unclear about the molecular mechanisms behind their generation and maintenance. Recently, in a research report published in the international journal Nature Immunology, entitled "Deficiency of metabolic regulator PKM2 activates the pentose phosphate pathway and generates TCF1+ progenitor CD8+ T cells to improve immunotherapy", scientists from Weill Cornell Medical College and other institutions found through a preclinical study that stimulating key metabolic pathways in T cells may enable them to more effectively resist and fight tumors when combined with immune checkpoint inhibitor therapy. This research finding may help researchers develop new potential strategies to enhance the potential of anti-cancer immunotherapy.
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.
Pleural mesothelioma is a rare and highly malignant tumor that originates from mesothelial cells on the surface of the pleura and is often closely related to asbestos exposure. Although immunotherapy such as immune checkpoint inhibitors can improve the survival of patients to a certain extent, the overall efficacy is limited. The median overall survival of patients is only about 18 months, and the prognosis is not ideal. Therefore, it is urgent to further explore and develop new strategies for the treatment of pleural mesothelioma.
Chimeric antigen receptor T (CAR-T) cell therapy is a revolutionary immunotherapy, but its durable response is still limited, partly due to the exhaustion of CAR-T cells. Recently, in a research report titled "IL-4 drives exhaustion of CD8+ CART cells" published in the international journal Nature Communications, scientists from Mayo Clinic and other institutions revealed a new reason why CAR-T cell therapy fails in some patients, and proposed a new strategy combining antibodies and gene editing technology, which is expected to improve the effect of this breakthrough therapy.
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.