Basic and Translational Research Faculty Mentors

Research Interests

• Methods for analysis of ultra-high dimensional data and massive multiple hypothesis tests
• Modeling and analysis of complex survival outcome
• Applications in biomedicine, especially multi-omics, image analyses, and clinical trials. 
• Incorporation of ML/AI in the above areas

Research Interests

• Pediatric Hematology and Oncology
• Clinical trial design, analysis, and monitoring
• Innovative trial designs for rare cancers and other diseases

Research Interests

• Mathematical/statistical modeling for complex data
• Pre-clinical and clinical trials design and analysis
• Statistical genetics/genomics
• Systems biology/genetical genomics

Research Interests

• Functional data analysis
• High-dimensional data analysis
• Imaging analysis
• Statistical learning and computing
• Nonparametric statistics
• Bioinformatics

Research Interests

• Causal inference
• Cancer survivorship
• Decision theory
• Clinical trial
• Infectious disease
• Oncology
• Semiparametric statistics
• Survival analysis
• Meta-analysis

Research Interests

• Statistical and machine learning methods for high-dimensional omics data
• Integrative analysis in multi-omics studies
• Longitudinal and time series models

Research Interests

• Neuroimaging statistics
• Longitudinal and dependent data analysis
• Survival data analysis
• Clinical trial design

Research Interests

• Developing novel statistical methods for incomplete survival/time-to-event data
• Cancer survivorship-late effect of new therapies
• Computational statistics and M-health
• Clinical trials

Research Interests

• Basic, translational, clinical and population science

Research Interests

• Dose finding methodologies as they apply to pediatric oncology
• Efficacy designs in rare diseases
• Clinical trial operations with specific focus on multi-institutional studies

Research Interests

• Bayesian adaptive clinical trials design
• Nonparametric Bayesian modelling
• Digital health
• Longitudinal data analysis

Research Interests

• Development of statistical methodology to identify biologically and clinically relevant genomic alterations 
• Creation of novel methods that integrate multiple pharmacologic and clinical endpoints with multiple forms of genomic data

Research Interests

• Design of clinical trials
• Sequential monitoring of clinical trials
• Survival analysis with long-term survivors
• Composite endpoints in studies of bone marrow transplantation

Research Interests

• Univariate and multivariate robust inference
• Robust regression
• Goodness-of-fit
• Longitudinal analysis
• Clinical trials design
• Survival analysis

Research Interests

• Enhancing contouring quality assurance in radiation oncology using AI and machine learning
• Developing interpretable AI models for improved clinical decision-making in radiation therapy
• Advancing functional statistical methods for medical research applications
• Applying measurement error models to improve accuracy in clinical data analysis

Research Interests

• Precision medicine; individualized treatment rules
• Longitudinal data analysis
• Spatialtemporal data analysis
• Clinical trials; observational studies

Research Interests

• Gene therapy and genome editing
• Inherited bone marrow failure syndromes, with particular focus on Diamond-Blackfan anemia
• Role of ribosomes in hematopoiesis
• Bone marrow transplantation for bone marrow failure syndromes

Research Interests

• Adoptive cell therapy for pediatric patients with solid tumors
• Overcoming obstacles to success for clinical implementation of CAR T-cell therapy for pediatric patients with solid and central nervous system (CNS) tumors

Research Interests

• Cancer immunotherapy
• Cell therapy
• Stem cell transplantation

Research Interests

• Generating and optimizing novel and safe CAR T cell-based therapies to target pediatric brain tumors
• Enhancing CAR T persistence and efficacy using different genetic manipulations
• Understanding tumor escape mechanism post CAR T-cell treatment
• Understanding CAR T-cell functional memory programs that control their long-term effector function
• Defining CAR T-cell and brain tumor immune microenvironment (TIME) interactions using immune competent brain tumor models

Research Interests

• Adoptive immunotherapy of hematological malignancies
• Functional characterization of genetically modified cells

Research Interests

• Fundamental mechanisms of T cell biology

Research Interests

• Investigating molecular and cellular mechanisms driving neural differentiation and maturation during brain development.
• Studying how neural cells utilize signaling pathways, transcriptomic regulation, and cytoskeletal dynamics.
• Exploring dual-function proteins that regulate RNA and the cytoskeleton, linked to neurodevelopmental and neurological disorders.
• Uncovering regulatory mechanisms in the nervous system to identify potential therapeutic targets.

Research Interests

• Spatial-temporal regulation of energy homeostasis at lipid droplet-organelle contact sites
• Dynamic organization of ER-Golgi secretory compartments
• Alteration of inter-organelle communication in neurological diseases

Research Interests

• Therapeutic development for rare pediatric neurological disorders
• Stem cell-based tools for studying human nervous system
• (Patho)physiological transcription regulation across human brain development

Research Interests

• Spatial order and assembly of chromosomes
• Cellular compartment formation through phase separation
• DNA nanoscopy and chromatin structure
• Biochemical reconstitution of nuclear functions

Research Interests

• Excitatory and inhibitory synaptogenesis
• Astrocyte-neuron communication
• Astrocyte-secreted molecules during synapse formation
• Role of astrocyte dysfunction in neurodevelopmental disorders

Research Interests

• Molecular mechanisms contributing to disease pathogenesis 
• Development of diagnostic tools and therapies 
• Role of somatic mtDNA mutations in the development of cancer and cancer sequelae
• Contribution of trafficking and autophagy defects in the pathogenesis of neuromuscular disorders 

Research Interests

• DNA damage response in the nervous system
• Molecular neurobiology
• Mouse models of human disease
• Brain tumors
• Neurodegeneration
• Tumor suppressors

Research Interests

• Genetics of pediatric neurological disease
• Molecular mechanisms in epileptic encephalopathies
• Genome structure and rearrangements

Research Interests

• Mechanisms of sonic hedgehog signal transduction 
• Mechanisms of cell-to-cell communication during development 
• Regulation of cytoneme-based signal transport

Research Interests

• Programmed cell death 
• Cellular homeostasis
• Apoptotic proteins (BCL2 family members)
• Modulation of cell death machinery as a therapeutic strategy for lymphoid and myeloid leukemia

Research Interests

• Genome-editing technologies
• Disease modeling in iPSCs
• Development of preclinical animal models using CRISPR-Cas9
• Development of novel genome editing strategies to treat β-hemoglobinopathies

Research Interests

• Neurogenetics
• Mechanisms of neurological diseases
• RNA and RNA-binding proteins
• Biomolecular condensates 

Research Interests

• Synthetic gene regulators (SynGRs) as precision-tailored therapeutics for personalized medicine
• Artificial transcription factors (ATFs) to regulate cell fate programs and enable regenerative medicine
• Specificity and Energy landscapes for SynGR/ATF design, genome annotation, and precision medicine
• Chemical-Genetic dissection of RNA polymerase II transcription cycle

Research Interests

• Covalent interactions between small molecules and proteins/RNA
• Automated synthesis and study of small organic molecules

Research Interests

• Regulation of PXR and CAR (nuclear receptors), and CYP3A (drug–metabolizing enzymes) in drug toxicity, drug resistance and human diseases
• Development of chemical probes and therapeutic leads for PXR, CAR and CYP3A
• Development of drug discovery technologies
• High throughput screening and high content screening

Research Interests

• Mechanism and regulation of protein machines
• Chemical Probe Discovery
• Cancer biology

Research Interests

• Design of chemical probes to study epigenetic regulations
• Chromatin structure and function
• Protein engineering
• Cancer biology

Research Interests

• Ligand discovery
• Variable-temperature protein crystallography
• Protein conformational ensembles
• Protein flexibility and hydration
• Protein structure and its role in protein function and malfunction
• Molecular docking

Research Interests

• Design, synthesis, and development of novel antibiotic and chemotherapeutic agents
• Modulation of Coenzyme A levels for the treatment of rare diseases and cancer
• Development of innovative therapeutic approaches to tackle drug-resistant infections

Research Interests

• Translational research in pediatric cancer
• Development of novel and selective epigenetic drug therapies
• Targeting the DNA damage response to optimize chemo- and  radiation therapy
• Multi-scale modeling of biological and chemical data

Research Interests

• Studying how gene transcription is regulated, including genome organization and responses to environmental cues.
• Investigating transcriptional changes in disease to understand pathology and identify potential treatments.
• Exploring DNA elements that regulate cell identity, tumor survival, and mutations, while modeling core regulatory circuits.
• Developing tools to map super-enhancers, their interactions, and the genes they regulate using sequencing data.

Research Interests

• Algorithm development for assessing the interplays between genetic alterations and epigenetic deregulations
• Functional consequences of complex genetic alterations in pediatric solid tumors
• Novel biological discovery from high throughput screening

Research Interests

• Applications of machine learning and AI in translational medicine
• Genomics technologies (e.g. single-cell / spatial transcriptomics)
• Drug target and drug combination discovery
• Neuroblastoma

Research Interests

• Mathematical modeling
• Early detection of cancers
• Clonal evolution
• Germline mosaicism

Research Interests

• Systems biology and immunology 
• Single-cell and spatial systems biology 
• Immuno-oncology 
• Translational research 

Research Interests

• Using large-scale, multi-dimensional genomic data to understand the initiation and progression of pediatric cancers
• Development of computational methods for analyzing large-scale genomic data and visualization tools for viewing integrative genomic data

Research Interests

• Designing and developing state-of-the-art software systems for:
  • indexing and integrating structured omics and biomedical big data
  • real-time querying and analysis
  • visual exploration of cancer clinical and omics datasets

Research Interests

• Investigating mechanisms driving diffuse high-grade gliomas (HGGs) in children, including Grade III anaplastic astrocytomas and Grade IV glioblastomas
• Studying unique features of pediatric HGGs, such as anatomical differences and distinct oncogenic mutations compared to adult gliomas
• Identifying subgroups of pediatric HGG based on clinical features and recurrent mutations linked to chromatin regulation and developmental signaling
• Developing improved disease models to study pediatric HGG subgroups and test targeted therapies

Research Interests

• Mechanisms through which neural circuits govern motor output
• Molecular diversity of neurons in the vertebrate motor system
• Interactions between supraspinal brain systems, spinal circuits and sensory inputs that control movement

Research Interests

• Defining mechanisms that regulate the growth of the vertebrate nervous system
• Understanding how growth-control mechanisms are coupled with patterning mechanisms to achieve the proper size, shape, and cytoarchitecture of the nervous system
• Determining the connection between growth-control mechanisms and human neurological tumors and malformations
• Identifying the genetic basis of brain size evolution

Research Interests

• Role of proteostasis and stress responses in skeletal muscle aging
• Systemic signaling via muscle-derived secreted factors (myokines) during aging and age-related diseases 

Research Interests

• Coordination of proliferation and differentiation during neurogenesis
• Retinal disease and stem cell based therapies
• Retinoblastoma and targeted chemotherapy
• Pediatric solid malignancies
• High throughput screening and drug discovery
• Genomics and epigenomics of pediatric solid tumors

Research Interests

• Brain development (mechanisms of neocortical growth and folding)
• Molecular mechanisms of Hedgehog signaling
• Function of primary cilia in medulloblastoma
  

Research Interests

• Membrane trafficking during synaptic vesicle recycling
• Sorting of synaptic vesicle proteins
• Emergence of bilaterian synaptic systems
• Defects in synaptic function related to epilepsy, autism, and Down syndrome

Research Interests

• Brain tumors
• Gene regulation
• Brain development
• Developmental therapeutics

Research Interests

• Molecular mechanisms contributing to normal and pathological neuronal death and differentiation
• Genes and signaling pathways involved in synaptic stability 
• Human diseases of the nervous system 

Research Interests

• Embryonal central nervous system tumors
• Disrupted neurodevelopment in pediatric brain tumors
• Genetic dependencies and preclinical model development
• Liquid biopsy genetic and methylation signatures 

Research Interests

• Proteins and RNAs in epigenetic programming of stem cell functions
• Epigenetic mechanisms influencing gene expression changes
• Epigenetic regulation of stem cell functions

Research Interests

• Mass spectrometry-based proteomics and metabolomics
• Systems biology of cancer
• Molecular pathogenesis of Alzheimer’s disease
• Ubiquitin and proteome turnover

Research Interests

• Single cell systems biology
• Spatial genomics and proteomics
• Disease pathogenesis from cells of origin
• Genomic technology development

Research Interests

• Mechanisms regulating diverse functions of neuromodulatory circuits
• Activity patterns of neuromodulatory neurons in vivo during arousal-related behaviors
• Molecular diversity of locus coeruleus neurons
• How changes in neuromodulatory circuits promote psychiatric and neurodegenerative disorders

Research Interests

• Molecular and cellular mechanisms regulating neuronal motility
• The interplay between neuronal differentiation and neuronal polarity signaling
• The centrosome as a focal point to coordinate cellular polarity
• Time-lapse microscopy as a tool to unlock the cell biology of neural development
• Cerebellar development

Research Interests

• Molecular mechanisms of the synaptic vesicle cycle
• Presynaptic origins of neurodegenerative disease
• Organelle trafficking and polarity in neurons
• Organelle quality control in neurons

Research Interests

• Learning and memory
• Synaptic transmission and plasticity
• Neuronal circuits and behavior
• Genetic models of schizophrenia, autism, and other neuropsychiatric diseases

Research Interests

• Childhood Cancer Survivor Study (CCSS), Principal Investigator
• Longitudinal Cardiotoxicity in Adult Survivors of Childhood Cancer (R01, NIH), Principal Investigator
• Encouraging Mammography and Preventive Opportunities for Women Exposed to Radiation, Co-Investigator
• Reducing Skin Cancer in Childhood Cancer Survivors, Co-Investigator
• Adult Neurobehavioral Late Effects of Pediatric Low Grade Brain Tumors, Co-Investigator

Research Interests

• Dissemination and implementation science research
• Cancer prevention and control: human papillomavirus (HPV) vaccination and cervical cancer screening
• Partnered- and engaged-approaches to research (community-engaged research)
• Cancer-related health disparities among underserved populations

Research Interests

• Gait and movement disorders of childhood 
• Peripheral neuropathy and nervous system malformations 
• Clinical Outcome Assessments and biomarkers
• Precision measurement and disability prevention

Research Interests

• Hypothalamic/pituitary consequences of childhood cancer and its therapy
• Genetic determinants of susceptibility to late effects of childhood cancer therapy
• Pubertal disorders and their molecular causes

Research Interests

• Late effects of childhood cancer therapy
• Hodgkin and non-Hodgkin lymphomas

Research Interests

• Patient-reported outcomes measurement and clinical application
• Psychometric analysis
• Quality of life and symptoms assessment in childhood cancers
• Social network/integration research in cancer survivors
• Financial toxicity in childhood cancer

Research Interests

• Disease burden after childhood cancer
• Health status and quality of life after childhood cancer
• Pediatric cancer survivorship care guideline development

Research Interests

• Late effects of childhood cancer therapy
• Cardiovascular outcomes of cancer therapy
• Transition of care from pediatric to adult medicine
• Adolescent and young adult oncology

Research Interests

• Clinical and population measurement of physical performance and disability
• Exercise, physical activity and dietary intervention to prevent and remediate frail health
• Accelerated aging in cancer survivors
• Analysis of epidemiologic data
• Cardiac rehabilitation

Research Interests

• Pharmacogenomics of cancer therapies
• Genetic epidemiology
• Multi-omics (epigenomics, transcriptomics, proteomics, and metabolomics) and system genetics (e.g., eQTLs)
• Functional genomics using human induced pluripotent stem cells
• Polygenic risk prediction
• Genetic contributions to health disparities

Research Interests

• Genetic epidemiology
• Cancer survivorship
• Statistical learning from large biological data

Research Interests

• Global pediatric health
• Hospital quality of care
• Critical care in pediatric oncology
• Clinical prediction scores

Research Interests

• Disease burden estimation, decision analysis, global epidemiology of childhood cancer
• Prioritization and the cost-effectiveness of childhood cancer treatment in low and middle income countries
• Pediatric leukemia, cancer survivorship