Office of Science-Industry Partnerships
Partnering Opportunities
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- 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 – Neuroblastoma
Immune checkpoint inhibitors and CAR-T therapies for relapsed pediatric solid tumors – Navin Pinto - 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 – 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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- 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 – Neuroblastoma
Immune checkpoint inhibitors and CAR-T therapies for relapsed pediatric solid tumors – Navin Pinto - 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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- Activity Trackers and Apps
Optimizing activity tracker and app use to promote physical activity, health and wellness – Michelle Garrison - 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
- Activity Trackers and Apps
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- 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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- 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, Autoimmunity, and Infectious Disease – Macrophage Regulation
Designing technologies and drugs to direct macrophages to block or initiate inflammation – Heather Gustafson - 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 – Neuroblastoma
Immune checkpoint inhibitors and CAR-T therapies for relapsed pediatric solid tumors – Navin Pinto - 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 - COVID-19 – Diagnostic for Immunity
A validated, high-throughput, cell-free test for SARS-CoV-2 neutralizing antibodies – Stephen Smith - 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 – 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
-
- 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 - Sleep Science – Researching Child, Adolescent and Family Sleep
Developing and testing real world tools and strategies to help families improve sleep – Michelle Garrison
- Activity and Fitness
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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
-
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
-
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
-
Understanding Brain Overgrowth Diseases – Kathleen Millen
-
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
-
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
-
Immune checkpoint inhibitors and CAR-T therapies for relapsed pediatric solid tumors – Navin Pinto
-
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
-
Clinical trial opportunities in pediatric analgesics – Gary Walco
-
Using technology to treat chronic pain in children and adolescents – Tonya Palermo
-
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
-
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
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Craniofacial Malformations – Clinical Data
Developing improved diagnostics, medical interventions, and coordinated care for patients with craniofacial malformations – Kelly Evans, Carrie Heike, Daniela Luquetti
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Cryptosporidium and Other Parasites
Identifying and testing small molecule inhibitors of N-myristoyltransferase - Bart Staker
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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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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 – 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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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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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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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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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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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 and Neurodegenerative Diseases: Interactomes
Developing compact, high-affinity antibodies for research and clinical applications – John Aitchison
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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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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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Malaria and HIV – Monoclonal Antibodies
Optimizing antibody isolation, characterization, and production with a rapid, cost-efficient platform – Noah Sather
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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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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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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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Identification of kinase inhibitors that increase the Beta-lactam sensitivity of MRSA – Lakshmi Rajagopal
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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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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
Contact Us
Physical Address
818 Stewart St.
Seattle, WA 98101
Mailing Address
M/S 818-S
PO Box 5371
Seattle, WA 98145-5005
