University of California San Francisco

University of California San Francisco

745 Parnassus Ave

San Francisco, CA 94143

United States

Chemical Genetics and CRISPRi/a Screen for Novel Therapeutic Targets in a Humanized Model for MYCN Associated High-Risk Pediatric Neuroblastoma

Background

Neuroblastoma is the most common extracranial solid tumor in children. Amplification of the MYCN oncogene is identified in about half of high-risk patients and is associated with rapid tumor progression and poor prognosis irrespective of tumor stage. MYCN protein overexpression is sufficient to drive pathogenesis of neuroblastoma in vitro and in vivo. Extensive studies have been done on targeting known regulators of MYCN to decrease pathological MYCN protein level.

Understanding the Role of Translational Control in Humanized Mouse Models for Medulloblastoma

Brain Tumors, Medulloblastoma

Background

Medulloblastoma (MB) is the most common malignant pediatric brain tumor. While 70-80% of patients survive more than 5 years, survivors have significant neurocognitive and neuro-endocrine disabilities. Several recent studies have identified 4 distinct molecular subgroups (WNT, SHH, Group 3 and Group 4). These subgroups differ in their mutational spectra, gene expression signatures and clinical features including outcome.

Impact of Whole Body Radiation Dose After Therapy with 131I-Metaiodobenzylguanidine (MIBG)

Neuroblastoma

Investigating the Role of Genetic and Epigenetic Variation in Risk of Childhood Acute Lymphoblastic Leukemia in Down Syndrome

Acute Myeloid Leukemia (AML)

Background

Acute lymphoblastic leukemia (ALL) is the most common cancer in childhood. Down syndrome (DS) is a genetic disorder caused by an extra chromosome 21, and DS children have a 20-fold increased risk of ALL. Furthermore, children with ALL and DS ("DS-ALL") have increased treatment-related deaths and disease recurrence than children without DS. Determining the cause of DS-ALL is, therefore, of paramount importance. The additional chromosome 21 must play a role in increased leukemia risk, but not every child with DS develops ALL.

Targeting eEF2 Kinase in Neuroblastoma

Neuroblastoma

Background

Identifying Drug Resistant Mutations in Ph-like Acute Lymphoblastic Leukemia

Acute Lymphoblastic Leukemia (ALL)

Investigating therapeutic strategies for JMML: a screen of kinase inhibitors to reveal potential therapeutic strategies and inform future efforts to address aberrant signal transduction.

Juvenile Myelomonocytic Leukemia (JMML)

A Study of Epigenetic Reprogramming with HDAC Inhibitors and DNMT Inhibitors to Restore Chemosensitivity in Relapsed Childhood T-Cell Acute Lymphoblastic Leukemia Cell Cultures

Leukemia

Evaluation of MIBG Transporters in Neuroblastoma

Neuroblastoma

Incidence and Outcomes of Clostridium Difficile in Pediatric Hematopoietic Stem Cell Transplant (HSCT) Recipients

Leukemia, Neuroblastoma

Alex's Lemonade Stand Foundation for Childhood Cancer