The journal Nature Communications published a study on the 14th, in which a research team from the CMRI in Australia tested a gene therapy in the entire human liver with the aim of developing more effective treatments for life-threatening genetic diseases. Researchers say this is the first time gene therapy function has been directly evaluated in human liver. This human liver model allows scientists to more accurately estimate the effective dose of new therapies and identify potential toxic side effects. It will also be a powerful system for developing new viral vectors as the foundation for the next generation of advanced therapies. One of the biggest challenges faced by researchers in pushing gene therapy from the laboratory to clinical practice is the lack of effective preclinical models that can be used to develop and test new therapies. For example, before the therapy can be tested on patients, laboratory tests with biological relevance and clinical predictability should be conducted. The testing model must faithfully reproduce the physiological condition and complex tissue structure of humans, so that they can accurately predict the outcomes of patients receiving treatment. Last year, CMRI's research team collaborated with other teams to develop a new method for maintaining human liver survival in a laboratory environment, known as a normothermic liver perfusion system. Those livers that were not suitable for human transplantation and were previously discarded or stored on ice for research can now be preserved outside the body at human temperature, making cutting-edge biomedical research possible. The most common gene therapy is to replace or repair defective genes, and the most effective delivery system is a harmless viral system based on adeno-associated viruses (AAVs). The CMRI team has demonstrated the potential of using a normothermic liver perfusion system to test AAV based gene therapy before initiating clinical studies. The use of the entire human liver is a revolutionary advancement in the field of gene therapy, as it enables researchers to accurately test how new therapies will affect the liver. (Lai Xin She)