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A gene that UVA Health researchers discovered is responsible for the deadliest type of brain tumor is also responsible for two forms of childhood cancer, the scientists have found.

The new discovery may open the door to the first targeted treatments for two types of rhabdomyosarcoma, a cancer of the soft tissue that primarily strikes young children.

The gene may also play an important role in other cancers that form in muscle, fat, nerves and other connective tissues in both children and adults, cold prevention medicine the research suggests.

“We accumulated multiple lines of evidence supporting [the gene] AVIL is powerful driver for both major types of rhabdomyosarcoma,” said researcher Hui Li, PhD, of the University of Virginia School of Medicine’s Department of Pathology and UVA Cancer Center. “The tumors are oncogene addicted to AVIL, which supports the rationale to design therapeutic interventions to target AVIL in this childhood cancer.”

Rhabdomyosarcoma Oncogene

Li and his team discovered in 2020 that the gene AVIL is the oncogene (cancer-causing gene) responsible for glioblastoma, the most lethal form of brain cancer. Less than 7% of patients with glioblastoma survive five years after diagnosis.

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