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Mouse Model Successfully Simulates Human Neurofibromatosis
Neurofibromatosis (NF1) is a genetic disease, and researchers at the University of Washington School of Medicine recently successfully modeled this disease in humans using a mouse model, and will therefore promote in-depth studies of tumor development, diagnosis, and treatment. After validating their animal models, the research team obtained two important findings: New blood vessels and cells of the immune system are critical to the initial formation of the tumor and may therefore become drug targets; brain images commonly used to determine if treatment is needed may not be possible Accurate diagnosis. The study will be published in the January 2005 issue of Annals of Neurology. NF1 is the most common neurological disease caused by a single gene mutation. This disease can lead to many complications, including brain tumors. In order to conduct animal clinical studies, the Gutmann-led research group has constructed a mouse model that, like humans with the disease, has an abnormal copy of the NF1 gene in each somatic cell. One of the supporting cells, astrocytes, has two abnormal copies of this gene. Recent research papers have shown that the formation of brain tumors in these mice has several similar clinical features with tumor development in NF1 children. First, these mice develop tumors along the optic nerve and the optic chiasm, which is responsible for sending visual information from the eye to the brain. This type of tumor, called the visual pathway glioma, is most common in children with NF1. Second, the time course of tumor development is similar to that of humans. The human visual pathway glioma is usually surrounded by blood vessels and glial cells. The researchers found that when the mutant mice were three weeks old, their number of small blood vessels in the optic nerve and optic nerve intersected was four times that of the control mice, and glial cells were also found in the nerve and nerve intersections before tumor formation in the mutant mice. This finding suggests that these molecular events are important for the development of the tumor. These findings may mean that compounds that inhibit the supply of growth-promoting factors provided by new blood vessels and glial cells can be used for targeted therapy of NF1 brain tumors. Later, the researchers also conducted clinical trials using this mouse model.