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Researchers at Memorial Sloan Kettering found that PGBD5, a gene known to drive pediatric brain cancers by cutting and rearranging DNA, also plays an essential role in normal brain development. The study shows that PGBD5 deliberately creates DNA breaks in developing neurons that are precisely repaired to support proper neuron formation and function. This makes PGBD5 a rare “double-edged” gene whose beneficial developmental role can, when misregulated, lead to childhood cancers and neurodevelopmental disorders.
This important finding will hopefully lead to future development of therapies that can block PGBD5’s cancer-causing activity while preserving its normal role in brain development, potentially leading to safer treatments for pediatric brain tumors.