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Pediatric brain tumors are more complex disorders than other cancers due to gaps in our understanding of their cellular origins, insufficient drug therapy, and complications arising from the blood brain barrier. Nevertheless, in the last decade, there has been significant improvement in the treatment of these tumors although the survival rate is still low. Thus, new innovative approaches to treat these malignant tumors are warranted. Among pediatric brain tumors, medulloblastoma is the most common and contributes significantly to the high mortality rate among children throughout the world. Defects in Sonic Hedgehog (Shh) signal transduction are major contributors to brain tumor development, especially medulloblastoma. In this book chapter, we review recent publications that identified new Shh downstream target genes that are implicated in medulloblastoma development. The products of these genes represent novel therapeutic targets that might lead to innovative approaches to treat brain tumors.

Transcription Factor Targets as Treatment for Medulloblastoma