Functionally-defined Therapeutic Targets in Diffuse Intrinsic Pontine Glioma

By Catherine S. Grasso, Yujie Tang, Nathalène Truffaux, N. Berlow, Lining Liu, M. Debily, M. Quist, L. Davis, Elaine C. Huang, Pamelyn J. Woo and 30 more. Nature Medicine, 2015

595 citations recorded by the source corpus.

Diffuse intrinsic pontine glioma (DIPG) is a fatal childhood cancer. We performed a chemical screen in patient-derived DIPG cultures along with RNA-seq analyses and integrated computational modeling to identify potentially effective therapeutic strategies. The multi–histone deacetylase inhibitor panobinostat demonstrated therapeutic efficacy both in vitro and in DIPG orthotopic xenograft models. Combination testing of panobinostat and the histone demethylase inhibitor GSK-J4 revealed that the two had synergistic effects. Together, these data suggest a promising therapeutic strategy for DIPG.

Subfields: Genetics, Molecular Biology

View Paper: https://www.semanticscholar.org/paper/95289eb0a524809db0a4e362c7aa8a2732109999

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