Office of Science-Industry Partnerships
Our Vision
By developing collaborative relationships with industry, we will translate the work of our research investigators and clinicians into innovative products, devices and medical technology that will improve the lives of children and their families.
Partnering Opportunities
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Therapeutics
- ADHD
Treatments for attention deficit hyperactivity disorder (ADHD) and related problems through personalized medicine and improved diagnosis – Mark Stein - Allergy and Asthma – Mast Cells
Investigating mast cell contributions to inflammation-related diseases and adverse drug events – Adrian Piliponsky - Asthma
Using primary cell lines from the upper and lower respiratory tracts in organotypic airway epithelial cell models of pediatric asthma – Jason Scott Debley - Bone Disorders and Malformations
Understanding bone biology through the discovery of osteogenic proteins derived from the study of craniosynostosis – Michael Cunningham - Brain Malformations
Using Next Generation Sequencing to trace the genetic basis for brain malformations linked to PI3K-AKT-MTOR dysfunction – Ghayda Mirzaa - Brain Tumors – Combination Therapies
Identifying novel targets for combination therapies that reduce toxicities of treating childhood medulloblastomas – Myron K. Evans II - Cancer, Inherited, and Infectious Disease – Cell Transduction
Identifying methods to enhance transduction of viral vectors or nanoparticles into hematopoietic and other stem cells – Bruce Torbett - Cancer – Advancing Immunotherapy
Advancing T cell immunotherapy with fusion proteins that improve antitumor function in suppressive tumor microenvironments – Shannon Oda - Cancer – Drugs for Sarcomas
Identifying pathways in the development of Ewing and other sarcomas that have potential targets for existing drugs – Elizabeth Lawlor - Cancer – Improved CAR T Targeting
Creating next-generation adoptive immunotherapy for rhabdomyosarcoma and other cancers – Rimas Orentas - Cancer – Macrophage Regulation
Using macrophage cytokine production and epigenetics to predict and control immune response – Heather Gustafson - Cancer – Mouse Model for CAR-T Therapy
Studying CAR T cells for solid tumors under conditions that reflect normal human immunity – Michael Jensen, Vandana Kalia and Surojit Sarkar - Cancer – Neurotoxicity
Understanding neurologic complications of CAR T-cell therapy in pediatric cancer – Juliane Gust - Cancer – Small Molecules for CAR T
Screening for compounds that promote expression of CAR T targets – Rimas Orentas - Cancer – T cell conditioning
Metabolic conditioning of T cells using IL-2 – Vandana Kalia and Surojit Sarkar - Cancer – Target Validation
Rapidly measuring effects of T-cell function modulators on anti-cancer activity with gene-edited mouse models – Vandana Kalia and Surojit Sarkar - Cell and Gene Therapies
Genome-editing B cells for in vivo delivery of antibodies and other therapeutic proteins – Richard James - Cerebellar Development
Discovering genes that affect the cerebellum and may contribute to autism and epilepsy – Kimberly Aldinger - Chronic Pain
Using technology to treat chronic pain in children and adolescents – Tonya Palermo - Chronic and Recurrent Pain
Clinical trial opportunities in pediatric analgesics - Gary Walco - Concussion
Using magnetic resonance spectroscopic imaging to assess GABA levels in pediatric concussion patients – Jeffrey Ojemann - Congenital Disorders – Genetic Testing
Developing minimally invasive genetic tests for vascular malformations and other non-neoplastic pediatric disorders – James Bennett - Congenital Disorders of Glycosylation
Discovering genes, biomarkers, and the natural history of rare, single-gene genetic diseases – Christina Lam - Congenital Heart Defects – Zebrafish Models
CRISPR editing in zebrafish congenital heart defect models – Lisa Maves - COVID-19 Resources – Neutralizing Nanobodies
Screening, optimizing, and producing stable, specific, recombinant nanobodies against SARS-CoV-2 – John Aitchison - COVID-19 and Other Viral Diseases
Generating vaccines from antigen discovery through human clinical trials for influenza, West Nile, Zika, chikungunya, yellow fever, and SARS-CoV-2 viruses – Rhea Coler - Craniofacial Malformations — Clinical Data
Developing improved diagnostics, medical interventions, and coordinated care for patients with craniofacial malformations – Kelly Evans, Carrie Heike, Daniela Luquetti - Cryptosporidium and Other Parasites
Identifying and testing small molecule inhibitors of N-myristoyltransferase – Bart Staker - Dravet – Advanced Models
Developing novel therapies for genetic refractory epilepsies of childhood using mouse and organoid models – Franck Kalume - Duchenne Muscular Dystrophy – Zebrafish Models
Zebrafish as a preclinical model for evaluating DMD drug therapies and biomarkers – Lisa Maves - Epilepsy – Drug Screening
Using mouse models to screen potential drugs that could treat the most common forms of epilepsy and seizures – Kathleen J. Millen - Epilepsy – Stem Cell Models
Stem cell-based models for studying epilepsies associated with mTOR mutations – Ghayda Mirzaa and Filomena Pirozzi - Fibrosis
Acomys: A natural mammalian model of regeneration – Mark Majesky - Group B Streptococcus
Understanding GBS infection induced preterm labor – Lakshmi Rajagopal - Heart Arrhythmia
New technologies for improving catheter ablation – Stephen Seslar - Heart Metabolism and Function
Bridging clinical cardiology and research on cellular energy changes – Aaron Olson - Heart Surgery – “Ductal Paint”
Developing a method to fluorescently mark tissue for more accurate cardiac surgery – Mark Majesky - Heart Transplant – Extracorporeal Photophoresis
Using extracorporeal photophoresis to prevent graft rejection – Yuk Law - Heart Transplant – Immunoreactivity and Tolerance
Using flow cytometry as an immunophenotyping technique to predict transplant immunoreactivity and tolerance – Yuk Law - Hemophilia – Immunomodulation Therapy
Immunomodulation therapy to prevent or modulate inhibitory antibody responses in hemophilia patients – Carol Miao - Hemophilia – Intraosseous Lentiviral Therapy
Intraosseous delivery of lentiviral vectors targeting factor VIII expression in platelets for hemophilia A treatment – Carol Miao - HIV and Other Infectious Diseases – B-cell Sequencing
Sequencing complete B-cell receptor genes to characterize effective immune responses – Noah Sather - HIV Microbiomes
Defining the links between bacterial microbiome, virome, innate and adaptive immune responses, and the roles they play in HIV susceptibility – Heather Jaspan and Melanie Gasper - HIV – Diagnostics
Developing simple, rapid methods for determining drug resistance and viral persistence – Lisa Frenkel - HIV – CAR-T Therapies
Using a validated anti-cancer treatment to eliminate or reduce HIV-infected cells – Thor Wagner - HIV – Transmission and Progression
Studying mucosal immunology for novel approaches to vaccines and immunotherapies – Donald Sodora - HIV – Viral Reservoirs
Understanding the persistence of HIV viral reservoirs – Lisa Frenkel - Hypoplastic Left Heart Syndrome
Translational medicine for hypoplastic left heart syndrome – Vishal Nigam - Infantile Hemangiomas and Other Vascular Malformations
Endothelial and circulating C19MC microRNAs as biomarkers of infantile hemangioma – Jonathan Perkins - Infectious Diseases – Immune Development
Detecting maternal cells in cord and infant blood to study their effects on disease susceptibility, immune modulation, and vaccine effectiveness – Whitney Harrington - Infectious and Neurodegenerative Diseases: Interactomes
Developing compact, high-affinity antibodies for research and clinical applications – John Aitchison - Inflammatory Bowel Disease – Dietary Modifications
Effect of dietary modifications on disease activity – David Suskind - Influenza – Vaccines
Increasing antibody production and virus cross-reactivity with small molecule integrin inhibitors – Mridu Acharya - Kawasaki Disease
Genetic analysis for diagnosis, monitoring and predicting response to therapy in Kawasaki Disease, vasculitis and other vasculopathies – Michael Portman - Lupus – B-Cell Integrins
Identifying targets to modify B cell activation during autoimmunity – Mridu Acharya - Lupus – B-Cell Targets
Using novel mouse models to study the signals and pathways of systemic autoimmune diseases – Shaun Jackson - Malaria – Dormant Liver Form
Eliminating malaria through attenuated whole-organism vaccines and new drug targets – Stefan Kappe - Malaria – Human Immune Response
Defining immune responses to advance development of anti malaria vaccines – Ken Stuart - Malaria – Host-Pathogen Interactions
Host-based Drug Discovery for Infectious Diseases – Alexis Kaushansky - Malaria – Molecular Mechanisms
Using in vitro models and clinical cohorts to understand vascular aspects of disease and investigate interventions to restore vascular function – Joe Smith - Malaria – Next-Generation Therapeutics
Generating highly selective inhibitors of Plasmodium N-myristoyltransferase – Alexis Kaushansky and Bart Staker - Malaria – Vaccines, Therapies, Drug Resistance
Improving prevention and therapy with better vaccines and predictions about drug resistance – Ashley Vaughan - Malaria and HIV – Monoclonal Antibodies
Optimizing antibody isolation, characterization, and production with a rapid, cost-efficient platform – Noah Sather - Mitochondrial Diseases – Identifying Therapies
Identifying pathways, targets, and new interventions for treating childhood mitochondrial disorders and related seizures – Simon Johnson - Motor Impairments and Paralysis
Implantable sensory/stimulation devices for treatment of motor impairments – Jeffrey Ojemann - MRSA Infections
Identification of kinase inhibitors that increase the Beta-lactam sensitivity of MRSA – Lakshmi Rajagopal - Neural Development and Neurodegeneration
Applying in vivo proteomics and genetic approaches to study pathways in nervous system development and neurodegenerative disease – Brock Grill - Nontuberculous Mycobacterial Diseases – Drug Discovery
Discovering targets and screening small molecules to treat mycobacterial infections – Tanya Parish - Obesity
Testing novel pathways for obesity drugs – Christian Roth - Opioid Sensitivity and Tolerance
Using a scalable C. elegans behavioral screening platform to identify druggable candidates for modulating μ-opioid receptor sensitivity and tolerance – Brock Grill - Optimizing CAR T Cells at the Level of Receptor Signaling
Determining responsiveness and safety using a multiplex quantitative protein-protein interaction platform – Stephen Smith - Peroxisomal Disorders
Applying a systems approach to the molecular mechanisms of rare diseases – John Aitchison - Retinal Disorders
Using high-throughput chromatin immunoprecipitation sequencing (ChIP-Seq) to understand retinal development – Timothy Cherry - Rheumatic Diseases
Research network to study chronic recurrent multifocal osteomyelitis (CRMO) and other childhood rheumatic diseases – Yongdong (Dan) Zhao - Sepsis
Understanding how mast cell proteases influence mediators generated during the septic response – Adrian Piliponsky - Stroke – Neurovascular Imaging
Using two-photon microscopy and other methods to study neurovascular structure and function, including response to injury, disease, and drugs – Andy Shih - Trypanosomal Diseases
Identifying novel drug targets for sleeping sickness, Chagas disease, and leishmaniasis – Ken Stuart - Tuberculosis
Designing vaccines and therapies with a novel mouse model – Kevin Urdahl - Tuberculosis – Drug Discovery
Identifying and optimizing candidate compounds using a high-throughput multiassay platform – Tanya Parish - Tuberculosis – Drug Regimens
Using validated computational models to identify new targets and score combination therapies for clinical efficacy against tuberculosis and other diseases – Shuyi Ma - Tuberculosis – Phosphosignaling pathways
Targeting phosphosignaling pathways and non-replicating bacteria – Christoph Grundner - Tuberculosis and Nontuberculous Mycobacterial Diseases
Translating vaccine and treatment candidates from the lab to clinical trials – Rhea Coler - Vascular and Lymphatic Malformations – Genotyping and Treatment
Applying cell-free DNA testing to precision diagnosis and personalized medicine for congenital disorders – Jonathan Perkins
- ADHD
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Diagnostics
- Autism – Characterizing Protein Interactions
Using animal models and high throughput immunoprecipitation assays to characterize disease subtypes and mechanisms of action of autism – Stephen Smith - Cancer – Macrophage Regulation
Using macrophage cytokine production and epigenetics to predict and control immune response – Heather Gustafson - Cancer – Neurotoxicity
Understanding neurologic complications of CAR T-cell therapy in pediatric cancer – Juliane Gust - Concussion
Using magnetic resonance spectroscopic imaging to assess GABA levels in pediatric concussion patients – Jeffrey Ojemann - Congenital Disorders – Genetic Testing
Developing minimally invasive genetic tests for vascular malformations and other non-neoplastic pediatric disorders – James Bennett - COVID-19 – Diagnostic for Immunity
A validated, high-throughput, cell-free test for SARS-CoV-2 neutralizing antibodies – Stephen Smith - COVID-19 Resources – Neutralizing Nanobodies
Screening, optimizing, and producing stable, specific, recombinant nanobodies against SARS-CoV-2 – John Aitchison - Healthcare Associated Infections
Using metagenomics Next Generation Sequencing (mNGS) to track outbreaks of healthcare associated infections – Danielle Zerr - Heart Transplant – Eplet Mismatches
Using donor-recipient Human Leukocyte Antigen (HLA) eplet mismatches to assess risks for pediatric heart transplant patients – Yuk Law - Heart Transplant – Immunoreactivity and Tolerance
Using flow cytometry as an immunophenotyping technique to predict transplant immunoreactivity and tolerance – Yuk Law - HIV – Diagnostics
Developing simple, rapid methods for determining drug resistance and viral persistence – Lisa Frenkel - HIV – Viral Reservoirs
Understanding the persistence of HIV viral reservoirs – Lisa Frenkel - Human Herpesvirus HHV-6 – Epidemiology
Understanding primary and reactivated HHV-6 to prevent acute graft vs. host disease – Danielle Zerr - Infantile Hemangiomas and Other Vascular Malformations
Endothelial and circulating C19MC microRNAs as biomarkers of infantile hemangioma – Jonathan Perkins - Juvenile Idiopathic Arthritis
Developing a mobile phone-based thermal imaging tool for assessing JIA in primary care and family practice – Yongdong (Dan) Zhao - Kawasaki Disease
Genetic analysis for diagnosis, monitoring and predicting response to therapy in Kawasaki Disease, Vasculitis and Other Vasculopathies – Michael Portman - Lupus – B-Cell Targets
Using novel mouse models to study the signals and pathways of systemic autoimmune diseases – Shaun Jackson - Retinal Disorders
Using high-throughput chromatin immunoprecipitation sequencing (ChIP-Seq) to understand retinal development – Timothy Cherry - Sepsis
Understanding how mast cell proteases influence mediators generated during the septic response – Adrian Piliponsky - Sleep Apnea
Developing improved diagnostics and treatments for pediatric sleep apnea patients – Maida Chen - Tuberculosis and Antimicrobial-Resistance
Adapting a single-cell growth assay for infectious disease agents – John Aitchison - Vascular and Lymphatic Malformations – Genotyping and Treatment
Applying cell-free DNA testing to precision diagnosis and personalized medicine for congenital disorders – Jonathan Perkins - Wilson Disease and Other Lysosomal Storage Diseases – Newborn Screening
A new method uses dried blood samples to detect lysosomal storage diseases, primary immunodeficiencies and Wilson disease – Sihoun Hahn
- Autism – Characterizing Protein Interactions
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Medical Devices
- Craniofacial Malformations – Clinical Data
Developing improved diagnostics, medical interventions, and coordinated care for patients with craniofacial malformations – Kelly Evans, Carrie Heike, Daniela Luquetti - Epilepsy – Medical Device Interventions
Using advanced models to develop and test medical interventions for treatment-resistant epilepsy – Franck Kalume - Heart Arrhythmia
New technologies for improving catheter ablation – Stephen Seslar - Hemophilia – Gene Therapy
Ultrasound-mediated gene delivery for hemophilia treatment – Carol Miao - Hypoplastic Left Heart Syndrome
Translational medicine for hypoplastic left heart syndrome – Vishal Nigam - Juvenile Idiopathic Arthritis
Developing a mobile phone-based thermal imaging tool for assessing JIA in primary care and family practice – Yongdong (Dan) Zhao - Malaria – Insect Dissection Tool
Streamlining insect handling for faster workflow, higher throughput, and more uniform samples – Alexis Kaushansky - Motor Impairments and Paralysis
Implantable sensory/stimulation devices for treatment of motor impairments – Jeffrey Ojemann - Sleep Apnea
Developing improved diagnostics and treatments for pediatric sleep apnea patients – Maida Chen
- Craniofacial Malformations – Clinical Data
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Vaccines
- COVID-19 and Other Viral Diseases
Generating vaccines from antigen discovery through human clinical trials for influenza, West Nile, Zika, chikungunya, yellow fever, and SARS-CoV-2 viruses – Rhea Coler - Group B Streptococcus
Understanding GBS infection induced preterm labor – Lakshmi Rajagopal - HIV and Other Infectious Diseases – B-cell Sequencing
Sequencing complete B-cell receptor genes to characterize effective immune responses – Noah Sather - HIV – Transmission and Progression
Studying mucosal immunology for novel approaches to vaccines and immunotherapies – Donald Sodora - Infectious Diseases – Immune Development
Detecting maternal cells in cord and infant blood to study their effects on disease susceptibility, immune modulation, and vaccine effectiveness – Whitney Harrington - Influenza – Vaccines
Increasing antibody production and virus cross-reactivity with small molecule integrin inhibitors – Mridu Acharya - Malaria – Dormant Liver Form
Eliminating malaria through attenuated whole-organism vaccines and new drug targets – Stefan Kappe - Malaria – Human Immune Response
Defining immune responses to advance development of anti malaria vaccines – Ken Stuart - Developing vaccines against malaria-causing parasites, including the dormant form of Plasmodium vivax – Noah Sather
- Malaria – Vaccines, Therapies, Drug Resistance
Improving prevention and therapy with better vaccines and predictions about drug resistance – Ashley Vaughan - Malaria and HIV – Monoclonal Antibodies
Optimizing antibody isolation, characterization, and production with a rapid, cost-efficient platform – Noah Sather - Tuberculosis
Designing vaccines and therapies with a novel mouse model – Kevin Urdahl - Tuberculosis and Nontuberculous Mycobacterial Diseases
Translating vaccine and treatment candidates from the lab to clinical trials – Rhea Coler
- COVID-19 and Other Viral Diseases
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Research Tools and Understanding Disease Pathways
- Addiction – Characterizing Pathways
Multi-level testing of the cortico-basal ganglia-thalamic circuit to identify distinct addiction causation – Susan Ferguson - Allergy and Asthma – Mast Cells
Investigating mast cell contributions to inflammation-related diseases and adverse drug events – Adrian Piliponsky - Anesthesia Risks
Understanding and reducing neurotoxicity after pediatric exposure to volatile anesthetics – Margaret Sedensky, Philip Morgan and Simon Johnson - Asthma
Using primary cell lines from the upper and lower respiratory tracts in organotypic airway epithelial cell models of pediatric asthma – Jason Scott Debley - Autism – Characterizing Protein Interactions
Using animal models and high throughput immunoprecipitation assays to characterize disease subtypes and mechanisms of action of autism – Stephen Smith - Bone Disorders and Malformations
Understanding bone biology through the discovery of osteogenic proteins derived from the study of craniosynostosis – Michael Cunningham - Brain Overgrowth Disorders
Using transgenic mouse models of pediatric intractable epilepsy and brain overgrowth to define new therapeutics and mechanisms – Kathleen Millen - Brain Malformations
Using Next Generation Sequencing to trace the genetic basis for brain malformations linked to PI3K-AKT-MTOR dysfunction – Ghayda Mirzaa - Brain Tumors – Combination Therapies
Identifying novel targets for combination therapies that reduce toxicities of treating childhood medulloblastomas – Myron K. Evans II - Brain Tumors - Neurtotrophin Receptors
Investigating neurotrophin receptors in neural development and as gene fusions that are potential drug targets – Siobhan S. Pattwell - Breathing Dysregulation
Developing and validating animal models to characterize the neuronal control of autonomic regulation – Nino Ramirez - Cancer, Inherited, and Infectious Disease – Cell Transduction
Identifying methods to enhance transduction of viral vectors or nanoparticles into hematopoietic and other stem cells – Bruce Torbett - Cancer – Improved CAR T Targeting
Creating next-generation adoptive immunotherapy for rhabdomyosarcoma and other cancers – Rimas Orentas - Cancer – Macrophage Regulation
Using macrophage cytokine production and epigenetics to predict and control immune response – Heather Gustafson - Cancer – Mouse Model for CAR-T Therapy
Studying CAR T cells for solid tumors under conditions that reflect normal human immunity – Michael Jensen, Vandana Kalia and Surojit Sarkar - Cancer – Neurotoxicity
Understanding neurologic complications of CAR T-cell therapy in pediatric cancer – Juliane Gust - Cancer – Small Molecules for CAR T
Screening for compounds that promote expression of CAR T targets – Rimas Orentas - Cancer – Target Validation
Rapidly measuring effects of T-cell function modulators on anti-cancer activity with gene-edited mouse models – Vandana Kalia and Surojit Sarkar - Cell and Gene Therapies
Genome-editing B cells for in vivo delivery of antibodies and other therapeutic proteins – Richard James - Cerebellar Development
Discovering genes that affect the cerebellum and may contribute to autism and epilepsy – Kimberly Aldinger - Congenital Disorders – Genetic Testing
Developing minimally invasive genetic tests for vascular malformations and other non-neoplastic pediatric disorders – James Bennett - Congenital Disorders of Glycosylation
Discovering genes, biomarkers, and the natural history of rare, single-gene genetic diseases – Christina Lam - Congenital Heart Defects – Zebrafish Models
CRISPR editing in zebrafish congenital heart defect models - Lisa Maves - Cryptosporidium and Other Parasites
Identifying and testing small molecule inhibitors of N-myristoyltransferase – Bart Staker - Dravet – Advanced Models
Developing novel therapies for genetic refractory epilepsies of childhood using mouse and organoid models – Franck Kalume - Duchenne Muscular Dystrophy – Zebrafish Models
Zebrafish as a preclinical model for evaluating DMD drug therapies and biomarkers – Lisa Maves - Epilepsy – Drug Screening
Using mouse models to screen potential drugs that could treat the most common forms of epilepsy and seizures – Kathleen J. Millen - Epilepsy – Stem Cell Models
Stem cell-based models for studying epilepsies associated with mTOR mutations – Ghayda Mirzaa and Filomena Pirozzi - Fibrosis
Acomys: A natural mammalian model of regeneration – Mark Majesky - Food Insecurity and Chronic Disease Management
Understanding the role that poor nutrition caused by food insecurity has on children living with type 1 diabetes or HIV – Jason Mendoza - Healthcare Associated Infections
Using metagenomics Next Generation Sequencing (mNGS) to track outbreaks of healthcare associated infections – Danielle Zerr - Heart Metabolism and Function
Bridging clinical cardiology and research on cellular energy changes – Aaron Olson - Heart Surgery – “Ductal Paint”
Developing a method to fluorescently mark tissue for more accurate cardiac surgery – Mark Majesky - Heart Transplant – Eplet Mismatches
Using donor-recipient Human Leukocyte Antigen (HLA) eplet mismatches to assess risks for pediatric heart transplant patients – Yuk Law - Heart Transplant – Extracorporeal Photophoresis
Using extracorporeal photophoresis to prevent graft rejection – Yuk Law - Heart Transplant – Immunoreactivity and Tolerance
Using flow cytometry as an immunophenotyping technique to predict transplant immunoreactivity and tolerance – Yuk Law - HIV and Other Infectious Diseases – B-cell Sequencing
Sequencing complete B-cell receptor genes to characterize effective immune responses – Noah Sather - HIV Microbiomes
Defining the links between bacterial microbiome, virome, innate and adaptive immune responses, and the roles they play in HIV susceptibility – Heather Jaspan and Melanie Gasper - HIV – Transmission and Progression
Studying mucosal immunology for novel approaches to vaccines and immunotherapies – Donald Sodora - HIV – Viral Reservoirs
Understanding the persistence of HIV viral reservoirs – Lisa Frenkel - Human Herpesvirus HHV-6 – Epidemiology
Understanding primary and reactivated HHV-6 to prevent acute graft vs. host disease – Danielle Zerr - Hypoplastic Left Heart Syndrome
Translational medicine for hypoplastic left heart syndrome – Vishal Nigam - Infectious Diseases – Immune Development
Detecting maternal cells in cord and infant blood to study their effects on disease susceptibility, immune modulation, and vaccine effectiveness – Whitney Harrington - Infectious and Neurodegenerative Diseases: Interactomes
Developing compact, high-affinity antibodies for research and clinical applications – John Aitchison - Influenza – Vaccines
Increasing antibody production and virus cross-reactivity with small molecule integrin inhibitors – Mridu Acharya - Juvenile Idiopathic Arthritis
Developing a mobile phone-based thermal imaging tool for assessing JIA in primary care and family practice – Yongdong (Dan) Zhao - Lupus – B-Cell Integrins
Identifying targets to modify B cell activation during autoimmunity – Mridu Acharya - Lupus – B-Cell Targets
Using novel mouse models to study the signals and pathways of systemic autoimmune diseases – Shaun Jackson - Malaria – Dormant Liver Form
Eliminating malaria through attenuated whole-organism vaccines and new drug targets – Stefan Kappe - Malaria – Host-Pathogen Interactions
Host-based drug discovery for infectious diseases – Alexis Kaushansky - Malaria – Human Immune Response
Defining immune responses to advance development of anti malaria vaccines – Ken Stuart - Malaria – Insect Dissection Tool
Streamlining insect handling for faster workflow, higher throughput, and more uniform samples – Alexis Kaushansky - Malaria – Molecular Mechanisms
Using in vitro models and clinical cohorts to understand vascular aspects of disease and investigate interventions to restore vascular function – Joe Smith - Developing vaccines against malaria-causing parasites, including the dormant form of Plasmodium vivax – Noah Sather
- Malaria – Vaccines, Therapies, Drug Resistance
Improving prevention and therapy with better vaccines and predictions about drug resistance – Ashley Vaughan - Malaria and HIV – Monoclonal Antibodies
Optimizing antibody isolation, characterization, and production with a rapid, cost-efficient platform – Noah Sather - Mitochondrial Disease
Using Ndufs4 mouse models to understand mTOR activation in Leigh Syndrome and other mitochondrial diseases – Margaret Sedensky and Philip Morgan - Mitochondrial Diseases – Identifying Therapies
Identifying pathways, targets, and new interventions for treating childhood mitochondrial disorders and related seizures – Simon Johnson - MRSA Infections
Identification of kinase inhibitors that increase the Beta-lactam sensitivity of MRSA – Lakshmi Rajagopal - Neural Development and Neurodegeneration
Applying in vivo proteomics and genetic approaches to study pathways in nervous system development and neurodegenerative disease – Brock Grill - Nontuberculous Mycobacterial Diseases – Drug Discovery
Discovering targets and screening small molecules to treat mycobacterial infections – Tanya Parish - Obesity
Testing novel pathways for obesity drugs – Christian Roth - Opioid Sensitivity and Tolerance
Using a scalable C. elegans behavioral screening platform to identify druggable candidates for modulating μ-opioid receptor sensitivity and tolerance – Brock Grill - Optimizing CAR T Cells at the Level of Receptor Signaling
Determining responsiveness and safety using a multiplex quantitative protein-protein interaction platform – Stephen Smith - Organ Development
Using genomics, genetics, and mouse models to identify disease mechanisms and novel therapeutic targets – David Beier - Peroxisomal Disorders
Applying a systems approach to the molecular mechanisms of rare diseases – John Aitchison - Retinal Disorders
Using high-throughput chromatin immunoprecipitation sequencing (ChIP-Seq) to understand retinal development – Timothy Cherry - Rheumatic Diseases
Research network to study chronic recurrent multifocal osteomyelitis (CRMO) and other childhood rheumatic diseases – Yongdong (Dan) Zhao - Sepsis
Understanding how mast cell proteases influence mediators generated during the septic response – Adrian Piliponsky - Sleep Apnea
Developing improved diagnostics and treatments for pediatric sleep apnea patients – Maida Chen - Stroke
Understanding the genetic causes and treatment options of pediatric stroke patients and developing improved prognostic biomarkers – Catherine Amlie-Lefond - Stroke – Neurovascular Imaging
Using two-photon microscopy and other methods to study neurovascular structure and function, including response to injury, disease, and drugs – Andy Shih - Trypanosomal Diseases
Identifying novel drug targets for sleeping sickness, Chagas disease, and leishmaniasis – Ken Stuart - Tuberculosis
Designing vaccines and therapies with a novel mouse model – Kevin Urdahl - Tuberculosis – Drug Discovery
Identifying and optimizing candidate compounds using a high-throughput multiassay platform – Tanya Parish - Tuberculosis – Drug Regimens
Using validated computational models to identify new targets and score combination therapies for clinical efficacy against tuberculosis and other diseases – Shuyi Ma - Tuberculosis – Phosphosignaling pathways
Targeting phosphosignaling pathways and non-replicating bacteria – Christoph Grundner - Tuberculosis and Antimicrobial-Resistance
Adapting a single-cell growth assay for infectious disease agents – John Aitchison - Vascular and Lymphatic Malformations – Genotyping and Treatment
Applying cell-free DNA testing to precision diagnosis and personalized medicine for congenital disorders – Jonathan Perkins - Viral Diseases – Improving RNA-Seq
Increasing fidelity of long-read RNA sequencing with novel template preparation chemistries – Bruce Torbett - Wilson Disease and Other Lysosomal Storage Diseases – Newborn Screening
A new method uses dried blood samples to detect lysosomal storage diseases, primary immunodeficiencies and Wilson disease – Sihoun Hahn
- Addiction – Characterizing Pathways
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Educational Products and Digital Health
- Activity and Fitness
Combining wearable fitness tracking with intervention programs aimed to increase daily physical activity of children – Jason Mendoza - Activity Trackers and Apps
Optimizing activity tracker and app use to promote physical activity, health and wellness – Michelle Garrison - ADHD
Treatments for attention deficit hyperactivity disorder (ADHD) and related problems through personalized medicine and improved diagnosis – Mark Stein - Adolescent-Focused Digital Health Interventions
Developing improved electronic health assessment tools to encourage self-reported health tracking and personalized care for adolescent patients – Cari McCarty and Laura Richardson - Chronic Pain
Using technology to treat chronic pain in children and adolescents – Tonya Palermo - Inflammatory Bowel Disease
Effect of dietary modifications on disease activity – David Suskind
- Activity and Fitness
-
Treatments for attention deficit hyperactivity disorder (ADHD) and related problems through personalized medicine and improved diagnosis – Mark Stein
-
Combining wearable fitness tracking with intervention programs aimed to increase daily physical activity of children – Jason Mendoza
-
Addiction – Characterizing Pathways
Multi-level testing of the cortico-basal ganglia-thalamic circuit to identify distinct addiction causation – Susan Ferguson
-
Adolescent-Focused Digital Health Interventions
Developing improved electronic health assessment tools to encourage self-reported health tracking and personalized care for adolescent patients – Cari McCarty and Laura Richardson
-
Allergy and Asthma – Mast Cells
Investigating mast cell contributions to inflammation-related diseases and adverse drug events – Adrian Piliponsky
-
Understanding and reducing neurotoxicity after pediatric exposure to volatile anesthetics – Margaret Sedensky, Philip Morgan and Simon Johnson
-
Using primary cell lines from the upper and lower respiratory tracts in organotypic airway epithelial cell models of pediatric asthma – Jason Scott Debley
-
Autism and Fragile X Syndrome — Signal Transduction and Therapeutics
Using animal models and high throughput immunoprecipitation assays to characterize disease subtypes and mechanisms of action of autism – Stephen Smith
-
Bone Disorders and Malformations
Understanding bone biology through the discovery of osteogenic proteins derived from the study of craniosynostosis – Michael Cunningham
-
Using Next Generation Sequencing to trace the genetic basis for brain malformations linked to PI3K-AKT-MTOR dysfunction – Ghayda Mirzaa
-
Brain Tumors – Combination Therapies
Identifying novel targets for combination therapies that reduce toxicities of treating childhood medulloblastomas - Myron K. Evans II, PhD
-
Brain Tumors – Neurotrophin Receptors
Investigating neurotrophin receptors in neural development and as gene fusions that are potential drug targets - Siobhan S. Pattwell, PhD
-
Developing and validating animal models to characterize the neuronal excitatory networks in control of rhythmic breathing activity – Nino Ramirez
-
COVID-19 and Other Viral Diseases
Generating vaccines from antigen discovery through human clinical trials for influenza, West Nile, Zika, chikungunya, yellow fever, and SARS-CoV-2 viruses - Rhea Coler
-
Cancer – Advancing Immunotherapy
Advancing T cell immunotherapy with fusion proteins that improve antitumor function in suppressive tumor microenvironments - Shannon Oda
-
Identifying pathways in the development of Ewing and other sarcomas that have potential targets for existing drugs - Elizabeth Lawlor
-
Cancer – Improved CAR T Targeting
“Improved CAR T Targeting For Pediatric Solid Tumors and Modulation of CAR-T Targets in Hematological Malignancy – Rimas Orentas
-
Cancer – Improving CAR T Cells
Determining responsiveness and safety using a multiplex quantitative protein-protein interaction platform – Stephen Smith
-
Cancer – Macrophage Regulation
Using macrophage cytokine production and epigenetics to predict and control immune response - Heather Gustafson
-
Cancer – Mouse Model for CAR-T Therapy
Studying CAR T cells for solid tumors under conditions that reflect normal human immunity – Michael Jensen, Vandana Kalia and Surojit Sarkar
-
Understanding neurologic complications of CAR T-cell therapy in pediatric cancer – Juliane Gust
-
Cancer – Small Molecules for CAR T
Screening for compounds that promote expression of CAR T targets – Rimas Orentas
-
Metabolic conditioning of T cells using IL-2 – Vandana Kalia and Surojit Sarkar
-
Rapidly measuring effects of T-cell function modulators on anti-cancer activity with gene-edited mouse models – Vandana Kalia and Surojit Sarkar
-
Cancer, Inherited, and Infectious Disease – Cell Transduction
Cancer, Inherited, and Infectious Disease – Cell Transduction - Bruce Torbett
-
Genome-editing B cells for in vivo delivery of antibodies and other therapeutic proteins – Richard James
-
Discovering genes that affect the cerebellum and may contribute to autism and epilepsy – Kimberly Aldinger
-
Using technology to treat chronic pain in children and adolescents – Tonya Palermo
-
Clinical trial opportunities in pediatric analgesics – Gary Walco
-
Concussion: Diagnosis, Monitoring, and Treatment
Using magnetic resonance spectroscopic imaging to assess GABA levels in pediatric concussion patients – Jeffrey Ojemann
-
Congenital Disorders of Glycosylation
Studying the natural history of rare, recently discovered genetic diseases – Christina Lam
-
Congenital Disorders – Genetic Testing
Using Next Generation Sequencing to identify the genetic profiles of tissue specific mosaic mutations – James Bennett
-
Congenital Heart Defects – Surgical Solutions
Translational medicine for hypoplastic left heart syndrome – Vishal Nigam
-
Congenital Heart Defects – Zebrafish Models
Zebrafish CRISPR editing and congenital heart defect models – Lisa Maves
-
Craniofacial Malformations – Clinical Data
Developing improved diagnostics, medical interventions, and coordinated care for patients with craniofacial malformations – Kelly Evans, Carrie Heike, Daniela Luquetti
-
Cryptosporidium and Other Parasites
Identifying and testing small molecule inhibitors of N-myristoyltransferase - Bart Staker
-
Using SCN1A knockout mice as a model and characterization of symptoms and co-morbidities – Franck Kalume
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Duchenne Muscular Dystrophy – Zebrafish Models
Zebrafish as a preclinical model for evaluating DMD drug therapies and biomarkers – Lisa Maves
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Using mouse models to screen potential drugs that could treat the most common forms of epilepsy and seizures – Kathleen J. Millen
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Epilepsy – Medical Device Interventions
Using advanced models to develop and test medical interventions for treatment-resistant epilepsy – Franck Kalume
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Stem cell-based models for studying epilepsies associated with mTOR mutations – Ghayda Mirzaa and Filomena Pirozzi
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Fibrosis – Models for Regenerative Medicine
Studying Acomys cahirinus rodents and ES cell lines to understand fibrosis versus tissue regeneration in wound healing – Kathleen J. Millen
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Fibrosis – Therapies for Controlling Fibrosis & Scar Tissue Formation
Acomys: A natural mammalian model of regeneration – Mark Majesky
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Food Insecurity and Chronic Disease Management
Understanding the role that poor nutrition caused by food insecurity has on children living with type 1 diabetes or HIV – Jason Mendoza
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Understanding GBS infection induced preterm labor – Lakshmi Rajagopal
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Defining the links between bacterial microbiome, virome, innate and adaptive immune responses, and the roles they play in HIV susceptibility – Heather Jaspan and Melanie Gasper
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HIV Transmission and Progression to AIDS
Studying mucosal immunology for novel approaches to vaccines and immunotherapies – Donald Sodora
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HIV and Other Infectious Diseases – B-cell Sequencing
Sequencing complete B-cell receptor genes to characterize effective immune responses – Noah Sather
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Using a validated anti-cancer treatment to eliminate or reduce HIV-infected cells – Thor Wagner
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Developing simple, rapid methods for determining drug resistance and viral persistence – Lisa Frenkel
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Understanding the persistence of HIV viral reservoirs – Lisa Frenkel
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Healthcare Associated Infections
Using metagenomics Next Generation Sequencing (mNGS) to track outbreaks of healthcare associated infections – Danielle Zerr
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New technologies for improving catheter ablation – Stephen Seslar
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Bridging clinical cardiology and research on cellular energy changes – Aaron Olson
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Heart Surgery – “Ductal Paint”
Developing a method to fluorescently mark tissue for more accurate cardiac surgery – Mark Majesky
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Heart Transplant – Eplet Mismatches
Using donor-recipient Human Leukocyte Antigen (HLA) eplet mismatches to assess risks for pediatric heart transplant patients – Yuk Law
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Heart Transplant – Extracorporeal Photophoresis
Using extracorporeal photophoresis to prevent graft rejection – Yuk Law
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Heart Transplant – Immunoreactivity and Tolerance
Using flow cytometry as an immunophenotyping technique to predict transplant immunoreactivity and tolerance – Yuk Law
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Ultrasound-mediated gene delivery for hemophilia treatment – Carol Miao
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Hemophilia – Immunomodulation Therapy
Immunomodulation therapy to prevent or modulate inhibitory antibody responses in hemophilia patients – Carol Miao
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Hemophilia – Intraosseous Lentiviral Therapy
Intraosseous delivery of lentiviral vectors targeting factor VIII expression in platelets for hemophilia A treatment – Carol Miao
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Human Herpesvirus HHV-6 – Epidemiology
Understanding primary and reactivated HHV-6 to prevent acute graft vs. host disease – Danielle Zerr
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Infantile Hemangiomas and Other Vascular Malformations
Endothelial and circulating C19MC microRNAs as biomarkers of infantile hemangioma – Jonathan Perkins
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Infectious Diseases – Immune Development
Detecting maternal cells in cord and infant blood to study their effects on disease susceptibility, immune modulation, and vaccine effectiveness – Whitney Harrington
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Infectious and Neurodegenerative Diseases: Interactomes
Developing compact, high-affinity antibodies for research and clinical applications – John Aitchison
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Inflammatory Bowel Disease – Dietary Modifications
Effect of dietary modifications on disease activity – David Suskind
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Increasing antibody production and virus cross-reactivity with small molecule integrin inhibitors – Mridu Acharya
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Developing a mobile phone-based thermal imaging tool for assessing JIA in primary care and family practice - Yongdong (Dan) Zhao
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Genetic analysis for diagnosis, monitoring and predicting response to therapy in Kawasaki disease, vasculitis and other vasculopathies – Michael Portman
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Identifying targets to modify B cell activation during autoimmunity – Mridu Acharya
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Using novel mouse models to study the signals and pathways of systemic autoimmune diseases – Shaun Jackson
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Identification of kinase inhibitors that increase the Beta-lactam sensitivity of MRSA – Lakshmi Rajagopal
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Malaria Vaccines, Therapies, and Drug Resistance
Improving prevention and therapy with better vaccines and predictions about drug resistance – Ashley Vaughan
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Malaria and HIV – Monoclonal Antibodies
Optimizing antibody isolation, characterization, and production with a rapid, cost-efficient platform – Noah Sather
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Eliminating malaria through attenuated whole-organism vaccines and new drug targets – Stefan Kappe
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Malaria – Host-pathogen interactions
Using in vitro, in vivo, and computational methods to find drugs and drug targets for bacterial, viral, and parasitic diseases – Alexis Kaushansky
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Malaria – Human Immune Response
Defining immune responses to advance development of anti malaria vaccines – Ken Stuart
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Malaria – Insect Dissection Tool
Streamlining insect handling for faster workflow, higher throughput, and more uniform samples - Alexis Kaushansky
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Malaria – Molecular Mechanisms
Using in vitro models and clinical cohorts to understand vascular aspects of disease and investigate interventions to restore vascular function – Joe Smith
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Malaria – Next-Generation Therapeutics
Generating highly selective inhibitors of Plasmodium N-myristoyltransferase – Alexis Kaushansky and Bart Staker
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Malaria – P. falciparum and P. vivax
Developing vaccines against malaria-causing parasites, including the dormant form of Plasmodium vivax – Noah Sather
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Medulloblastoma — Studies With Stem Cells
Using stem cell lines to gain insight into overproliferation leading to brain tumors and malformations – Kathleen J. Millen
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Using Ndufs4 mouse models to understand mTOR activation in Leigh Syndrome and other mitochondrial diseases – Margaret Sedensky and Philip Morgan
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Mitochondrial Diseases – Identifying Therapies
Identifying targets and screening compounds that inhibit the mTOR pathway using a mouse model and patient cell lines – Simon Johnson
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Motor Impairments and Paralysis
Implantable sensory/stimulation devices for treatment of motor impairments – Jeffrey Ojemann
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Nanobodies – Diagnostic and Therapeutic Resources
Using a unique pipeline to produce high-quality nanobodies with diversity in epitope recognition - John Aitchison
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Neural Development and Neurodegeneration
Applying in vivo proteomics and genetic approaches to study pathways in nervous system development and neurodegenerative disease – Brock Grill
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Nontuberculous Mycobacterial Diseases – Drug Discovery
Discovering targets and screening small molecules to treat mycobacterial infections –Tanya Parish
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Obesity – Chimeric Peptide Therapeutics
Testing novel pathways for obesity drugs – Christian Roth
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Opioid Sensitivity and Tolerance
Using a scalable C. elegans behavioral screening platform to identify druggable candidates for modulating μ-opioid receptor sensitivity and tolerance – Brock Grill
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Optimizing CAR T Cells at the Level of Receptor Signaling
Determining responsiveness and safety using a multiplex quantitative protein-protein interaction platform – Stephen Smith
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Using genomics, genetics, and mouse models to identify disease mechanisms and novel therapeutic targets – David Beier
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Applying a systems approach to the molecular mechanisms of rare diseases – John Aitchison
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Using high-throughput chromatin immunoprecipitation sequencing (ChIP-Seq) to understand retinal development – Timothy Cherry
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Research network to study chronic recurrent multifocal osteomyelitis (CRMO) and other childhood rheumatic diseases - Yongdong (Dan) Zhao
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Sepsis – Diagnostics and Therapeutics
Understanding how mast cell proteases influence mediators generated during the septic response – Adrian Piliponsky
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Developing improved diagnostics and treatments for pediatric sleep apnea patients – Maida Chen
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Stroke – Neurovascular Imaging
Using two-photon microscopy and other methods to study neurovascular structure and function, including response to injury, disease, and drugs – Andy Shih
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Stroke – Understanding Pediatric Stroke
Understanding the genetic causes and treatment options of pediatric stroke patients and developing improved prognostic biomarkers – Catherine Amlie-Lefond
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Identifying novel drug targets for sleeping sickness, Chagas disease, and leishmaniasis – Ken Stuart
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Designing vaccines and therapies with a novel mouse model – Kevin Urdahl
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Tuberculosis and Antimicrobial-Resistance
Adapting a single-cell growth assay for infectious disease agents – John Aitchison
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Tuberculosis and Nontuberculous Mycobacterial Diseases
Translating vaccine and treatment candidates from the lab to clinical trials – Rhea Coler
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Identifying and optimizing candidate compounds using a high-throughput multiassay platform – Tanya Parish
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Using validated computational models to identify new targets and score combination therapies for clinical efficacy against tuberculosis and other diseases - Shuyi Ma
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Tuberculosis – Phosphosignaling Pathways
Targeting phosphosignaling pathways and non-replicating bacteria – Christoph Grundner
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Vascular and Lymphatic Malformations – Genotyping and Treatment
Clinical trial opportunities in pediatric LM – Jonathan Perkins
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Viral Diseases – Improving RNA-Seq
Viral Diseases – Improving RNA-Seq - Bruce Torbett
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Wilson Disease and Other Lysosomal Storage Diseases – Newborn Screening
A new method uses dried blood samples to detect lysosomal storage diseases, primary immunodeficiencies and Wilson disease – Sihoun Hahn
Latest Partnerships
We’re partnering with companies to turn discoveries into advances that transform pediatric health and well-being. Our latest partnerships include:
- Developing cell therapies for acute myeloid leukemia with bluebird bio
- Partnering with Moderna to investigate mRNA therapies for hemophilia A
- Collaborating with Aptahem on aptamer-based treatments for sepsis
- Investigating a GBS vaccine for pregnant women, with Minervax
- Working with Nine to prevent stillbirth
- Studying innovative weight-loss agents with OXT Therapeutics
- Investigating the role of cadherin in disease
- Collaborating with UCB to identify mechanisms behind intractable epilepsy
- Working with Casebia Therapeutics to develop cell therapies for autoimmune disease
- Investigating mRNA therapies developed by Moderna Therapeutics for rare genetic disorders
- Creating a cost-effective, easy-to-use test for drug-resistant HIV, with help from InBios International
- Collaborating with Immunexpress to pursue a screening test for sepsis