Office of Science-Industry Partnerships

ADHD Treatments

Treatments for attention deficit hyperactivity disorder (ADHD) and related problems through personalized medicine and improved diagnosis – Mark Stein

Activity and Fitness

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

Anesthesia Risks

Understanding and reducing neurotoxicity after pediatric exposure to volatile anesthetics – Margaret Sedensky, Philip Morgan and Simon Johnson

Asthma – Ex Vivo Modeling

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

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, PhD

Brain Tumors – Neurotrophin Receptors

Investigating neurotrophin receptors in neural development and as gene fusions that are potential drug targets - Siobhan S. Pattwell, PhD

Breathing Dysregulation

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

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

“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

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

Cancer, Inherited, and Infectious Disease – Cell Transduction

Cancer, Inherited, and Infectious Disease – Cell Transduction - Bruce Torbett

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: 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

Dravet – Advanced Models

Using SCN1A knockout mice as a model and characterization of symptoms and co-morbidities – 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 – Medical Device Interventions

Using advanced models to develop and test medical interventions for treatment-resistant epilepsy – Franck Kalume

Epilepsy – Stem Cell Models

Stem cell-based models for studying epilepsies associated with mTOR mutations – Ghayda Mirzaa and Filomena Pirozzi

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

Fibrosis – Therapies for Controlling Fibrosis & Scar Tissue Formation

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

Group B Streptococcus

Understanding GBS infection induced preterm labor – Lakshmi Rajagopal

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 to AIDS

Studying mucosal immunology for novel approaches to vaccines and immunotherapies – Donald Sodora

HIV and Other Infectious Diseases – B-cell Sequencing

Sequencing complete B-cell receptor genes to characterize effective immune responses – Noah Sather

HIV – CAR-T Therapies

Using a validated anti-cancer treatment to eliminate or reduce HIV-infected cells – Thor Wagner

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

Healthcare Associated Infections

Using metagenomics Next Generation Sequencing (mNGS) to track outbreaks of healthcare associated infections – Danielle Zerr

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 – 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

Hemophilia – Gene Therapy

Ultrasound-mediated gene delivery for hemophilia treatment – Carol Miao

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

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

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

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 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

MRSA Infections

Identification of kinase inhibitors that increase the Beta-lactam sensitivity of MRSA – Lakshmi Rajagopal

Malaria Vaccines, Therapies, and 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

Malaria – Dormant Liver Form

Eliminating malaria through attenuated whole-organism vaccines and new drug targets – Stefan Kappe

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

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

Malaria – Next-Generation Therapeutics

Generating highly selective inhibitors of Plasmodium N-myristoyltransferase – Alexis Kaushansky and Bart Staker

Malaria – P. falciparum and P. vivax

Developing vaccines against malaria-causing parasites, including the dormant form of Plasmodium vivax – Noah Sather

Medulloblastoma — Studies With Stem Cells

Using stem cell lines to gain insight into overproliferation leading to brain tumors and malformations – Kathleen J. Millen

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 targets and screening compounds that inhibit the mTOR pathway using a mouse model and patient cell lines – Simon Johnson

Motor Impairments and Paralysis

Implantable sensory/stimulation devices for treatment of motor impairments – Jeffrey Ojemann

Nanobodies – Diagnostic and Therapeutic Resources

Using a unique pipeline to produce high-quality nanobodies with diversity in epitope recognition - John Aitchison

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 – Chimeric Peptide Therapeutics

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 – Diagnostics and Therapeutics

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 – Neurovascular Imaging

Using two-photon microscopy and other methods to study neurovascular structure and function, including response to injury, disease, and drugs – Andy Shih

Stroke – Understanding Pediatric Stroke

Understanding the genetic causes and treatment options of pediatric stroke patients and developing improved prognostic biomarkers – Catherine Amlie-Lefond

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 and Antimicrobial-Resistance

Adapting a single-cell growth assay for infectious disease agents – John Aitchison

Tuberculosis and Nontuberculous Mycobacterial Diseases

Translating vaccine and treatment candidates from the lab to clinical trials – Rhea Coler

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

Vascular and Lymphatic Malformations – Genotyping and Treatment

Clinical trial opportunities in pediatric LM – Jonathan Perkins

Viral Diseases – Improving RNA-Seq

Viral Diseases – Improving RNA-Seq - 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