LPCH Academic Grant Awards

SPARK 

Julie Baker, PhD
Professor, Department of Genetics   
(YR 2) A Non-invasive diagnostic for placenta Accreta 

Carolyn Bertozzi, PhD
Anne T. and Robert M. Bass Professor in the School of Humanities and Sciences; Professor, Department of Chemistry; and (by courtesy) of Radiology; and (by courtesy) of Chemical and Systems Biology
A PCR-based method for sample-sparing and cost-effective diagnosis of food allergy risk 

Helen Blau, PhD
The Donald E. and Delia B. Baxter Foundation Professor, Department of Microbiology & Immunology; Director, Baxter Laboratory for Stem Cell Biology
Transient telomere extension to treat liver failure in Dyskeratosis Congenita patients 

Tina Cowan, PhD
Professor of Pathology (Clinical), Stanford University Medical Center
A small-molecule activator of AMPK for treatment of mitochondrial disorders 

Shirit Einav, MD
Assistant Professor of Medicine (Infectious Diseases), and of Microbiology & Immunology
An assay for predicting the development of severe dengue prior to its onset

Jeffrey S. Glenn, MD, PhD
Professor, Department of Medicine (Gastroenterology & Hepatology); and of Microbiology & Immunology
Leveraging enterovirus dependency on the human host factor PIP5k1a for the development of a potent broad-spectrum antiviral 

Stuart Goodman, MD, PhD
Robert L. and Mary Ellenburg Professor of Surgery; Professor, Department of Orthopedic Surgery
Engineered mesenchymal stem cell therapy to promote immunological tolerance to pancreatic islets 

Mark A. Kay, MD, PhD
Dennis Farrey Family Professor in Pediatrics; Professor, Department of Genetics
Enhancement of adeno-associated virus-mediated homologous recombination (AAV-HR) through inhibition of the FANCM pathway 

Matthew Porteus, MD, PhD           
Associate Professor, Department of Pediatrics (Stem Cell Transplantation)
(YR 2) Genome edited airway stem cells as a durable cell-based therapy to treat cystic fibrosis

Robert M. Waymouth, PhD
Robert Eckles Swain Professor in Chemistry; Professor, Department of Chemistry; and (by courtesy) of Chemical Engineering
CARTS: new materials enable a new platform technology for gene delivery, vaccinations, and cancer immunotherapy 

Cardiovascular Institute

Christopher Gardner, PhD
Rehnborg Farquhar Professor, Stanford Prevention Research Center
Co-Investigators: Michael Snyder, PhD, Professor of Genetics; Francois Haddad, MD, Clinical Associate Professor of Medicine (Cardiology)
Addressing the obesity and Diabetes epidemic through understanding personalized energy expenditure

Doff McElhinney, MD
Professor, Department of Cardiothoracic Surgery (Pediatrics Cardiac Surgery) and of Pediatrics (Cardiology)
Psychosocial, Cognitive, and Quality of Life Outcomes in Children and Adults with Repaired Tetralogy of Fallot with Pulmonary Atresia and Major Aortopulmonary Collateral Arteries

James Priest, MD
Assistant Professor, Department of Pediatrics (Cardiology)
Co-Investigator: Mads Melbye, MD, DMSc, Visiting Professor of Medicine and Population Sciences
A Sensitized Genetic Association Study for Congenital Heart Disease

SPARK

Jeffrey Axelrod, MD, PhD
Professor, Department of Pathology
Novel Formulation and Repurposing of Gamma-Secretase Inhibitors to Restore Epithelial Health in the Cystic Fibrosis Airway

Rosa Bacchetta, MD
Associate Professor (Research), Department of Pediatrics (Stem Cell Transplantation)
(Project 1) Gene Corrected Autologous HSCs for the Treatment of IPEX Syndrome
(Project 2) Epigenetic Screening for Primary Immune Deficiencies and Immune Dysregulation

Julie Baker, PhD
Professor, Department of Genetics         
A Non-invasive Diagnostic for Placenta Accreta

Edward Bertaccini, MD
Professor, Department of Anesthesiology, Perioperative and Pain Medicine         
Refining a New Class of Anesthetic Agents for High Risk Populations including Infants and the Elderly

Helen Blau, PhD
The Donald E. and Delia B. Baxter Foundation Professor, Department of Microbiology & Immunology; Director, Baxter Laboratory for Stem Cell Biology
Design of Muscle Stem Cell Targeting Prodrug for Muscle Regeneration

Robson Capasso, MD
Associate Professor, School of Medicine, Divisions of Otolaryngology and Head and Neck Surgery
Haparin Binding Epidermal Growth Factor for Wound Healing

Jennifer Frankovich, MD
Clinical Associate Professor, Department of Pediatrics (Rheumatology)
Immunophenotypes in PANS: Biomarkers and Clues to Etiology

Michelle Monje-Deisseroth        
Assistant Professor, Department of Neurology & Neurological Sciences; (By courtesy) Departments of Neurosurgery, of Pediatrics, and of Pathology
GD2 CAR Immunotherapy in Diffuse Intrinsic Pontine Glioma

Matthew Porteus           
Associate Professor, Department of Pediatrics (Stem Cell Transplantation)
Genome Edited Airway Stem Cells as a Durable Cell-Based Therapy to Treat Cystic Fibrosis

Stanford Institute for Immunity, Transplantation and Infection (ITI)

Mark Kay, MD, PhD
The Dennis Farrey Family Professor in Pediatrics, Departments of Pediatrics and of Genetics                         
Sexually dimorphic viral-host interactions in pediatric liver diseases

Angelle LaBeaud, MD
Associate Professor, Department of Pediatrics (Infectious Diseases) and (by courtesy) of Health Research & Policy (Epidemiology)
Characterizing the Effects of Antenatal Parasitic Infection on Fetal Immune System Development

Hiro Nakauchi MD, PhD
Professor, Department of Genetics                         
Regenerative potential of human fetal versus adult HSC transplantation: B lymphocytes and macrophages heterogeneity

Stanford Cardiovascular Institute (CVI)  

Katharine S. Edwards, PhD      
Clinical Assistant Professor of Cardiovascular Medicine and (by courtesy) the of Psychiatry and Behavorial Sciences
Angiographic and Psychosocial Evaluation of Peripartum vs. Non:Peripartum Spontaneous Coronary Artery Dissection: A Collaborative Study

Tracey L. McLaughlin, MD 
Professor of Medicine (Endocrinology), The Stanford University Medical Center
Collaborators: Nazish Sayed, MD, PhD, Ian Chen, MD, PhD
Predicting Cardiovascular Benefits of Anti-diabetic Drugs

Ashby J. Morrison, PhD           
Assistant Professor of  Biology, School of Humanities and Sciences
Collaborators: Kristy Red-Horse, PhD and Will Greenleaf, PhD
Epigenetic Regulation of Cardiac Development

Virginia Winn, MD   
Associate Professor of Obstetrics and Gynecology (Reproductive Biology), The Stanford University Medical Center
Endothelial Dysfunction in Preeclampsia

Stanford Institute for Stem Cell Biology and Regenerative Medicine

Kyle Loh, PhD
Instructor, Institute for Stem Cell Biology and Regenerative Medicine
Expandable region-specific human radial glia: a new tool to study brain development and neurodevelopmental disorders in children

SPARK

PI: Paul Bollyky, MD, PhD

Preventive Therapy for Type 1 Diabetes Mellitus

Co-PIs: Heather Irina Cohn, Joyce Teng, MD, PhD

Topical Sirolimus for Plantar Treatment of Epidermolysis Bullosa Simplex

PI: Laura Downey, MD

Repurposing of Fibrinogen Concentrate as a Cost-Effective and Safe Hemostatic Agent in Infants Undergoing Cardiac Surgery on Cardiopulmonary Bypass

PI: Jennifer Frankovich, MD, MS

Pediatric Acute Onset Neuropsychiatric Syndromes (PANS) CyTOF

Co-PIs: Kevin Grimes, MD, Daria Mochly-Rosen, PhD

Metachromatic Leukodystrophy and Exeroderma Pigmentosa

PI: Peter S. Kim, PhD

Toward a Novel Inhibitor of Ebola Virus

Co-PIs: Aashish Manglik, MD, PhD, Brian Kobilka, MD

Development of a Novel Opioid Agonist with Reduced Side Effects

PI: Everett Meyer, MD, PhD

A Novel Monoclonal Antibody Therapy to Prevent Acute Graft-Versus-Host Disease Following Hematopoietic Cell Transplantation

Co-PIs: Kathleen M. Sakamoto, MD, PhD, Mark Smith

CREB Inhibitor to Treat Leukemia

PI: James R. Swartz, PhD

Developing a Broadly Neutralizing Influenza Vaccine

PI: Karl G. Sylvester, MD

Metabolic Assays for Newborn Disease and Feeding Tolerance

Stanford Cardiovascular Institute (CVI)  

Co-PIs: Ashby J. Morrison, PhD, Mary Kristy Red-Horse, PhD, William James Greenleaf, PhD

Epigenetic Regulation of Cardiac Development

Co-PIs: Tracey Lynn McLaughlin, MD, MS, Nazish Sayed, MD, PhD, Ying-Li Chen, MD, PhD

Predicting Cardiovascular Benefits of Anti-diabetic Drugs Using Induced Pluripotent Stem Cell-derived Endothelial Cells

Stanford Institute for Immunity, Transplantion and Infection (ITI)

Co-PIs:  Eliver Ghosn, PhD, Leonore A. Herzenberg, PhD, Hiromitsu Nakauchi, MD, PhD

Regenerative Potential of Human Fetal Versus Adult HSC Transplantation:  B lymphocytes and Macrophages Heterogeneity

Co-PIs:  Angelle Desiree LaBeaud, MD, Holden Maecker, PhD, Scott Boyd, MD, PhD

Characterizing the Effects of Antenatal Parasitic Infection on Fetal Immune System Development

Co-PIs:  Nicole K. Paulk, PhD, Mark A. Kay, MD, PhD

Sexually Dimorphic Viral-Host Interactions in Pediatric Liver Diseases

Stanford Institute for Stem Cell Biology and Regenerative Medicine

PI: Ravindra Majeti, MD, PhD

Investigation and Targeting of Pre-Leukemia in Familial Platelet Disorder Associated with Mutations in RUNX1

Co-PIs: Jennier A. Tremmel, MD, Katharine S. Edwards, PhD

Angiographic and Psychosocial Evaluation of Peripartum vs. Non-Peripartum Spontaneous Coronary Artery Dissection (SCAD): A Collaborative Study

Co-PIs: Virginia D. Winn, Valerie Baker, MD, Frauke von Versen-Höynck

Impact of assisted reproductive techniques (ART) on maternal and fetal vascular health and endothelial progenitor cell biology

SPARK

Co-PIs: Oyebimpe O. Adesina, Jason Gotlib, MD, Michael Jeng, MD, Carolyn Hoppe
Repurposing Alendronate to treat Osteonecrosis of the Femoral Head in Sickle Cell Disease
PI: Justin Annes, MD, PhD
Developing a Small Molecule Stimulator of Beta-Cell Regeneration

PI: Paul Bollyky, MD, PhD
Repositioning hymecromone for type 1 diabetes prevention

Co-PIs:  Kevin Grimes, MD, Daria Mochly-Rosen, PhD
Student Pediatric Therapeutic Projects

PI: Tobias Meyer, PhD
Developing third generation inhibitors of mTOR by targeting a novel GPCR-like receptor to treat an orphan mTORC1 Disease:  LAM

PI: Debra L. Safer, MD
Qsymia for Bulimia Nervosa and Binge Eating Disorder: A Double-Blinded Randomized Trial

Co-PIs: Kathleen M. Sakamoto, MD, PhD, Mark Smith
Developing peptides to target CREB for AML therapy

PI: David K. Stevenson, MD
Noninvasive, Non-Contact Sensor For Detecting Jaundice, Heart Rate, And Respiratory Rate

PI: Karl Sylvester, MD
Premature Newborns, Metabolic Abnormalities and Human Disease

Stanford Cancer Institute

PI:  Eric Alejandro Sweet-Cordero, MD
Personalized Genomics in Pediatric Oncology

Stanford Cardiovascular Institute (CVI)  

Co-PI:  Anitra Romfh, MD & Manish J. Butte, MD, PhD
T-Cell Deficiencies in Adult Congenital Heart Disease

Co-PI:  David Camarillo, PhD & Paul Wang
T-Cell Deficiencies in Adult Congenital Heart Disease

SPARK

PI:  Paul Bollyky
Repositioning Hymecromone for Type 1 Diabetes Prevention

Co-PIs:  Gilbert Chu, Matthew Kanan
Rapid Small Volume Detection of Blood Ammonia

Co-PIs:  Kevin Grimes, Daria Mochly-Rosen
Student-led Projects to Address Pediatric Indications

Co-PIs:  Alfred T. Lane, Jayakumar Rajadas, M. Peter Marinkovich
Microneedle Patch Delivery of Type VII Collagen to the Skin

PI:  Bruce Ling
Novel Diagnostics for Kawasaki Disease with Vasculitis Biomarkers

PI:  Clara Lo
N-Acetylcysteine for Chronic Immune Thrombocytopenia
Co-PIs:  Peter Marinkovich, Atul Butte

Topical Ion Channel Blockers: A Novel Mechanism to Treat Psoriasis
PI:  Anthony Ricci
Protecting the Inner Ear from Cisplatin Toxicity Using Small Molecule Activators of Aldehyde Dehydrogenase

Co-PIs:  Anthony J. Ricci, Chen Che-Hong
Developing Non-Ototoxic Aminoglycoside Antibiotics

Co-PIs:  Kathleen M. Sakamoto, Mark Smith
Targeting CREB for AML Therapy

PI:  Peter Santa Maria
Heparin Binding – Epidermal Growth Factor as a Treatment for Chronic Tympanic Membrane Perforation

PI:  David K. Stevenson
Chemoprevention of Neonatal Jaundice Using Zinc Protoporphyrin

PI:  Matt van de Rijn
Monoclonal Antibody Inhibiting R0R2 to Treat Sarcomas

Stanford Cancer Institute

PI:  Eric Alejandro Sweet-Cordero
Personalized Genomics in Pediatric Oncology

Stanford Cardiovascular Institute (CVI)

PI:  Susan Fernandes
Predicting 10-Year Mortality in Adults with Congenital Heart Disease

PI:  Scott Ceresnak
A Novel Signal Analysis Tool Ablation of Wolff-Parkinson-White Syndrome (WPW) in Children

Co-PIs:  Seda Tierney, David Rosenthal
Parental Acquisition of Echocardiographic Images in Pediatric Heart Transplant Recipients Using “Pocket” Ultrasound: A Tele-health Feasibility Study

PI:  Joshua Knowles
Exploring the Role of Maternal Insulin Resistance in Congenital Heart Defects

PI:  Matthew Porteus
Comparison of Isoform Expression Differences by SMRT Sequencing of Isogenic iPS Derived Cardiomyocytes Corrected for the Myotonic Dystrophy Expanded Triplet Report

Stanford Institute for Immunity, Transplantation and Infection (ITI)

PI:  Kenneth Weinberg
Optimizing Cytotoxic T Lymphocyte Therapy of EPB-PTLD by Clonal Tracking of Viral and Lymphocyte
Populations

PI:  Timothy Sweeney
Prospective Validation of an 11-Gene Set for Distinguishing Sterile SIRS from Sepsis in Pediatric ICU Patients at Admission

Stanford Institute for Stem Cell Biology and Regenerative Medicine

PI:  Marius Wernig
Developing a new therapy for childhood white matter diseases

PI:  Michelle Monje
The role of neuronal activity in NF1-associated optic pathway gliomagenesis

PI:  Maria-Grazia Roncarolo
Tr1-mediated killing of primary AML blasts

SPARK

PI:  Brian Feldman
A Regenerative Medicine Therapy for Treating Muscle Wasting

Co-PI:  Alfred T. Lane, Jayakumar Rajadas, M. Peter Marinkovich
Microneedle Patch Delivery of Type VII Collagen to the Skin

Co-PI:  David K. Stevenson, Jayakumar Rajadas, Ronald J. Wong
Chemoprevention of Neonatal Jaundice Using Zinc Protoporphyrin

PI:  Matt van de Rijn
ROR2 is a novel therapeutic target in pediatric and adult neoplasms

Stanford Cancer Intitute

PI:  Eric Alejandro Sweet-Cordero
Personalized Genomics in Pediatric Oncology

Stanford Cardiovascular Institute (CVI)

PI:  Gerry Berry
A clinical-grade next generation sequencing assay for targeting DNA mutations in inherited non-syndromic cardiomyopathies

PI:  Susan M. Fernandes
Risk Factors for Acquired Cardiovascular Disease in Adults with Congenital Heart Disease

Co-PIs:  Daria Mochly-Rosen, Daniel Bernstein, Tobias Meyer
Identifying the Master Integrator of Cardiac Cell-Fate Decision

Stanford Institute for Immunity, Transplantation and Infection (ITI)

PI:  Brian Feldman
Nano-Engineered Plasmonic Chip for Point-of-Care Diagnosis of Type 1 Diabetes

Stanford Institute for Neuro-Innovation & Translational Neurosciences (SINTN)

PI:  Michelle Monje
White matter development as a paradigm for postnatal plasticity

Stanford Institute for Stem Cell Biology and Regenerative Medicine

PI:  Michael Longaker
Identifying xenograft factors affecting human skeletal stem cell differentiation in mice

SPARK

Co-PIs:  Atul Butte, Bruce Ling
Development of Bedside Diagnostic/Prognostic Biosensor to Improve Preeclampsia Care

Co-PIs:  Alfred T. Lane, Jayakumar Rajadas, M. Peter Marinkovich
Microneedle Patch Delivery of Type VII Collagen to the Skin

Co-PIs:  Anthony Ricci, Alan Cheng
Novel aminoglycosides to prevent hearing loss

Co-PIs:  Matt van de Rijn, Roel Nusse
Monoclonal Antibody Inhibiting ROR2 to Treat Sarcomas

Stanford Cancer Institute

PI:  Eric Alejandro Sweet-Cordero
Genomics Initiative

Stanford Cardiovascular Institute (CVI)

PI:  Manish J. Butte
Measuring Electromechanical Asynchrony in iPSC-CM using Atomic Force Microscopy

PI:  Christopher H. Contag
Molecular Mechanisms of Fetal Bradycardia Due to Placental Infection

PI:  Pilar Ruiz-Lozano
Engineered Embryonic Epicardium Activates Cardiac Regeneration – A Preclinical Study in Pigs

Stanford Institute for Immunity, Transplantation and Infection (ITI)

Co-PIs:  Sheri Krams, Carlos Esquivel, Manish Butte
Examination of the functional interactions between NK cells and their ligands

Co-PIs:  Emmanuel Mignot, Elizabeth Mellins
H1N1 Peptide Presentation by DQB1*0602

Stanford Institute for Neuro-Innovation & Translational Neurosciences (SINTN)

PI:  Michelle Monje
White matter development as a paradigm for postnatal plasticity

Stanford Institute for Stem Cell Biology and Regenerative Medicine

PI:  Renee A Reijo Pera
Genomic health in children conceived naturally and via IVF

PI:  Marius Wernig
Engineering Autism‐associated large genetic lesions in human ES cells

SPARK

Project Title: B‐Type Natriuretic Peptide (BNP) Levels before and after Pulmonary Artery Banding and the Arterial‐ Atrial Switch Operation for Congenitally Corrected Transposition of the Great Vessels
Danton Char, MD, Instructor, Anesthesia Pediatric Cardiac Anesthesia

Project Title: Preventing Allergic Transfusion Reactions
Magali Fontaine, MD, PhD, Assistant Professor, Pathology Transfusion Medicine

Project Title: Mechanisms of Immune Evasion by Respiratory Syncytial Virus
David Hong, MD, Instructor, Pediatrics Immunology and Allergy

Project Title: Defining the Roles of IL‐4 and Alternatively Activated Macrophages in Urinary Tract Co‐ Infection
Michael Hsieh, MD, PhD, Associate Professor, Urology

Project Title: Impact of Fecal Microbial Diversity on Shedding of Oral Poliovirus Vaccine
Yvonne Maldonado, MD, Professor, Pediatrics Infectious Diseases

Project Title: A novel regulatory T cell population and control of autoimmunity
Elizabeth Mellins, MD, Professor, Pediatrics Human Gene Therapy

Project Title: Mechanistic Studies on the Role of CD4+ T cells in Food Allergy Immunotherapy
Kari Nadeau, MD, PhD, Associate Professor, Pediatrics Immunology and Allergy

Project Title :Double-blind, randomized,placebo-controlled cross-over trial of oxytocin treatment for social deficits in children with autism
Karen Parker, PhD, Assistant Professor, Psychiatry and Behavioral Sciences

Project Title: Complex Network Analysis of Neonatal Brain DTI: Prognostic Value for VLBW Preterm Infants
Jessica Rose, PhD, Associate Professor, Orthopaedic Surgery

Project Title: In Vivo Imaging of Chemotherapy‐Induced Intestinal Mucositis
Eric Sibley, MD, PhD, Associate Professor, Pediatrics Gastroenterolog

Project: The role of EWS as a regulator of IncRNAs involved in RNA splicing and editing
Alejandro Sweet-Cordero, MD, Assistant Professor, Pediatrics Hematology Oncology

Project Title: Can we change Vascular Health & Exercise Performance of Overweight Children in 12 weeks using Skype?
Seda Tierney, MD, Assistant Professor, Pediatrics Cardiology