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Mouse brain tumors mimic those in human genetic disorder

St. Louis, Dec. 29, 2004 -- A recently developed mouse model of brain tumors common in the genetic disorder neurofibromatosis 1 (NF1) successfully mimics the human condition and provides unique insight into tumor development, diagnosis and treatment, according to researchers at Washington University School of Medicine in St. Louis.

After validating their animal model, the team made two important discoveries: New blood vessels and immune system cells may be essential to the initial formation of tumors and therefore may be promising drug targets; and brain images often used to determine the need for treatment may not actually be diagnostically informative.

"These mice develop brain tumors with many of the same features as those seen in children with NF1, and studying those tumors has helped us understand the cellular events involved in NF1 brain tumor development," says principal investigator David H. Gutmann, M.D., Ph.D., the Donald O. Schnuck Family Professor of Neurology.

The study appears online and will be published in the January 2005 issue of the journal Annals of Neurology.

NF1 is one of the most common neurological disorders caused by a single gene mutation. The disorder can lead to a variety of complications including brain cancer.

To supplement their clinical research, Gutmann's team developed a mouse model in which the animals, like humans with the disease, have one abnormal copy of the gene for NF1 in every cell in their body, while specific support cells in the brain called astrocytes have two abnormal copies of this same gene.

Their latest paper shows that brain tumor formation in these mice has several of the same distinguishing clinical characteristics as tumor development in children with NF1.

First, the mice developed tumors along the optic nerve and optic chiasm, which transmit visual information from the eye to the brain. This type of tumor, called an optic pathway glioma, is the most common
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Contact: Gila Z. Reckess
reckessg@wustl.edu
314-286-0109
Washington University School of Medicine
29-Dec-2004


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