Fanconi anemia is a childhood disease associated with impaired bone marrow function, developmental retardation, premature aging and early onset of cancer limiting the lifespan of patients to 35 years. Fanconi anemia results from impairment of one or more steps in the preventive pathway that removes aberrant DNA structures, which are caused by environmental stresses like pollutants, toxins in diet, etc.
Project Goal
Principal Investigator Name:
Timsi Rao, PhD
Project Title:
Elucidating the Role of Human FAN1 Nuclease in DNA Crosslink Repair
Pre-clinical Evaluation of LSD1 Inhibitors for the Treatment of Pediatric Ewing sarcoma: Defining the Biomarkers of Sensitivity and Mechanisms of Resistance
This project originated at The Cleveland Clinic in Cleveland, OH. Effective July 2017, Dr. Mack has accepted a new position will be completing his Young Investigator project at Baylor.
Background
Principal Investigator Name:
Stephen Mack, PhD
Project Title:
Interrogating the Enhancer Landscapes of Ependymoma to Inform Novel Therapies
Retinoblastoma is a childhood ocular tumor initiated by the inactivation of the RB1 gene and subsequent loss of retinoblastoma protein (pRB). Although pRB loss is the key-initiating event, the molecular mechanisms controlling the transformation of a normal retinal cell into malignancy remain unclear.
Principal Investigator Name:
Sunhye Lee, PhD
Project Title:
Single-cell RNA-seq Profiling of Transcriptional Transition States During Human Retinoblastoma
Neuroblastoma is an embryonal tumor responsible for 15% of pediatric cancer deaths. Survival rate for high-risk neuroblastoma, which affects the majority of patients, is still dismal despite the use of aggressive therapy. Relapse occurs in over half of patients, with no current prospect for a cure. Therefore, understanding the cause of relapse is critical for enabling the development of effective treatments.
Principal Investigator Name:
David Debruyne, PhD
Project Title:
Molecular Basis of ALK Inhibition Resistance in High-risk Neuroblastoma
Wilms' tumor is the most common pediatric kidney cancer. While many patients are cured, children with high-risk disease continue to have poor outcomes. No alternative, targeted therapies have proven effective in Wilms' tumor. One-fifth of Wilms' tumors harbor mutations in the enzymes are responsible for producing microRNAs, small RNA molecules that regulate expression of target genes. These mutations drive tumor formation; leading to a worse prognosis.
Project Goal
Principal Investigator Name:
Kenneth Chen, MD
Project Title:
The Role of MiRNA Impairment in Wilms' Tumor Formation
Hux is 7 years old and a party animal who loves dancing. Since he was diagnosed with Phelan-MCDermid syndrome at birth, he gets yearly brain MRIs - and one revealed a tumor when Hux turned 3. After a year of treatment, Hux has remained cancer-free!
