Patient-Partnered Collaborations for Single-Cell Analysis of Rare Inflammatory Pediatric Disease RFA Grantees

CZI invited applications from collaborative teams bringing together patient-led rare disease organizations and research teams for four-year research projects aimed at advancing our understanding of the fundamental biology underlying rare diseases. The Patient-Partnered Collaborations for Single-Cell Analysis of Rare Inflammatory Pediatric Disease RFA aims to support the application of single-cell biology methods to rare inflammatory pediatric diseases to clarify cellular mechanisms of disease, understand disease heterogeneity, and improve biomarker identification or diagnostics. Patients are centered in this work. Each team is led by a Coordinating Primary Investigator (PI) and Patient Organization PI. Read about the grantees below.

Integrating Patients to Accelerate the Science Towards a World without NEC

to apply single-cell methodology to understand the cellular and molecular pathophysiology of neonatal necrotizing enterocolitis, a devastating disease affecting medically fragile infants

Coordinating PI: Misty Good

University of North Carolina at Chapel Hill

Patient Organization PI: Jennifer Canvasser

NEC Society

Scott Magness

University of North Carolina at Chapel Hill

Troy Markel

Indiana University School of Medicine

Camilia Martin

Weill Cornell Medicine

Amy Hair

Baylor College of Medicine

Understanding the Cellular Ecosystem of Primary Sclerosing Cholangitis

to understand the cellular ecosystem of the rare liver disease primary sclerosing cholangitis and identify potential cellular targets for effective therapies

Coordinating PI: Sonya MacParland

University Health Network

Patient Organization PI: Ruth-Anne Pai

PSC Partners Seeking a Cure

Gary Bader

University of Toronto

Amanda Ricuitto

Hospital for Sick Children

Creating an Inflammatory Childhood Interstitial Lung Disease Cell Atlas

to develop an open source atlas of childhood inflammatory interstitial lung diseases at single-cell resolution of children of diverse ancestries

Coordinating PI: John Christopher Kennedy

Boston Children’s Hospital/Harvard Medical School

Patient Organization PI: Heather Thompson

Children’s Interstitial and Diffuse Lung Disease (chILD) Foundation

Martha Potts Fishman

Boston Children’s Hospital/Harvard Medical School

James Hagood

University of North Carolina-Chapel Hill

Benjamin Raby

Boston Children’s Hospital/Harvard Medical School

Deciphering RUNX1-Familial Platelet Disorder at Single-Cell Resolution

to elucidate cellular processes responsible for inflammatory disease in RUNX1-Familial Platelet Disorder by leveraging single-cell multi-omic and spatial transcriptomic technologies

Coordinating PI: Alexander Bick

Vanderbilt University Medical Center

Patient Organization PI: Katrin Ericson

RUNX1 Research Program

Anupriya Agarwal

The Knight Cancer Institute, Oregon Health & Science University

Esther Obeng

St. Jude Children’s Research Hospital

Developing a Single-Cell Atlas to Accelerate Precision Medicine in Juvenile Dermatomyositis

to create a juvenile dermatomyositis single-cell, cross-tissue atlas to accelerate precision medicine via partnering with an international research network and diverse patient communities

Coordinating PI: Jessica Turnier

University of Michigan

Patient Organization PI: Andrew Heaton

Cure JM

Gabriela Fragiadakis

University of California, San Francisco

J. Michelle Kahlenberg

University of Michigan

Jessica Neely

University of California, San Francisco