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Results: 269

Therapeutic reversal of pre-natal pontine ID1 signaling in DIPG

Diffuse intrinsic pontine glioma (DIPG) is a lethal pediatric brain tumor wand few children with this diagnosis survive up to two years. Even with the advent of precision-based medicine, experimental therapies have yet to show benefit beyond standard radiation, highlighting the dire need to identify and investigate novel genetic therapeutic ...

Targeting purine synthesis and one carbon metabolism in DIPG

Diffuse intrinsic pontine glioma (DIPG) is a rare, infiltrative, and incurable brainstem malignancy in children resulting in death within one year from diagnosis.  DIPG represents up to 85% of brainstem tumors and has the worst prognosis of all childhood cancers with a five-year overall survival of < 1% (1-5).  These ...

Targeted protein degradation for DIPG therapy

Childhood brain tumors are the second most common type of pediatric cancer, with overall survival and treatment related long-term side effects far worse compared to other children with solid tumors and haematological malignancies. The prognosis of brain tumors in children has improved over the past decades; however, one third of ...

Chromatin defects and therapeutic targets in pediatric brain cancers

DNA is wrapped around an octamer of histone proteins, known as a nucleosome, to form the basic repeating unit of chromatin. Genes encoding two chromatin regulators, ATRX and the histone H3.3, are frequently mutated in pediatric glioblastoma (pGBM) which is a lethal form of brain cancer. To translate this new ...

The role of QPRT and NAD pathways in DIPG treatment ...

The proposed study will provide insights into a novel strategy to inhibit energy metabolism as a potential way to tackle treatment-resistant high-grade gliomas (HGGs) and diffuse intrinsic pontine gliomas (DIPGs), the most commonly fatal brain tumors of childhood. This work incorporates our unique resource of treatment-naïve and treatment-resistant ...

Phase I/II study of oral Difluoromethylornithine (DFMO) and AMXT 1501 ...

Brain cancers are the deadliest of all childhood cancer and the leading cause of disease associated death in children. Diffuse intrinsic pontine glioma (DIPG), and diffuse midline glioma (DMG) remain the most aggressive childhood cancers and the only available treatments for this group of brain cancers are palliative. Our group ...