Awarded Grants

 

The ODC offers over 50 grant opportunities in 30+ disease areas annually to researchers across the globe, as well as within the Penn and CHOP communities. Since 2011, the ODC has funded 400 awards totaling $40,269,515 to 120+ institutions in 25 countries worldwide

See below for a complete list of ODC-awarded grants since 2011.

 
 
 

Awarded Grants

Featured
Evaluation of a therapeutic potential of disulfiram for its repositioning against encephalopathies caused by GNAO1 mutations

Vladimir Katanaev

University of Geneva, Faculty of Medicine

$100,000

DEVELOPMENT OF PHARMACOLOGICAL CHAPERONE FOR FUNCTIONAL RESCUE OF GalphaO MUTATIONS INVOLVED IN ENCEPHALOPATHY AND MOVEMENT DISORDERS.

Patrick M. Giguère

University of Ottawa

$100,000

Pilot clinical trial to assess efficacy of Caffeine Citrate for controlling GNAO1-related dyskinetic crises

Serena Galosi

Sapienza University, Rome

$100,000

GNAO1-related epilepsy: genotype-phenotype correlations

Erika Axeen

University of Virginia

$40,000

Pre-clinical development of a CRISPR-mediated gene therapy for GNAO1 Encephalopathy using patient stem cells and a mouse model

Blair Leavitt

University of British Columbia

$100,000

Development of second generation AAV gene therapy for GNAO1 deficiency

Miguel Sena-Esteves

University of Massachusetts

$100,000

Regulation of CDKL5 Function by Type I Interferons"

Josephine Thinwa, PhD

UT Southwestern Medical Center, USA

$150,000

Development of a tau hyperphosphorylation assay in CDKL5 deficiency disorder iPSC-derived neurons

Jedd Hubbs, PhD

Boston Children's Hospital, USA

$149,050

Exploiting microRNAs to disclose novel pathophysiological mechanisms, biomarkers and therapeutic approaches for CDKL5 deficiency disorder

Maurizio Giustetto, PhD

University of Torino, Italy

$134,300

Preclinical Investigations Of Proteasome and Lysosome Inhibitors In CDKL5 Deficient Mice"

James Eubanks, PhD

University Health Network - Krembil Research Institute, Canada

$147,896

An Evaluation of Gene Modification Strategies for Bloom Syndrome

Caroline Kuo

University of California, Los Angeles

$50,000

The Somatic Mutational Landscape Of Blm-Deficient Tumors: Finding Clues for Future Therapeutic Opportunities

Richarda de Voer

Radboud University Medical Center (Stichting Radboud Fonds)

$150,000

Novel Approaches to Cancer Surveillance in Bloom Syndrome

Vivian Chang

University of California, Los Angeles

$150,000

Investigating clonal expansion of pathogenic T cells in idiopathic multicentric Castleman disease

David Fajgenbaum

University of Pennsylvania

$58,775

Targeting biomolecular condensates, as a novel approach to ameliorate SYNGAP1-related synaptopathy

Kristian Strømgaard

University of Copenhagen

$74,851

Mapping Molecular and Behavioral Impacts of SETBP1 Deficiency in Zebrafish

Alexander Little

McMaster University

$68,544

Assessing Excitation-Transcription Coupling in SCN2A-Related Disorder Mouse Models

Jennifer Kearney

Northwestern University

$62,492

Leveraging Human iPSC Derived Beta Cells to Investigate the Impact of Novel Therapeutics in Patients with Congenital Hyperinsulinism

Mansa Krishnamurthy

Cincinnati Children's Hospital Medical Center

$77,165

Safety, Accuracy, and Feasibility of Continuous Glucose Monitoring in Infants in the Inpatient NICU Setting with Hyperinsulinism and Hypoglycemia

Paul Thornton

Cook Children's Medical Center

$77,165

Tcf4 in gut development; investigating underlying causes of gastro-intestinal problems in patients with Pitt Hopkins syndrome

Simone Mesman

University of Amsterdam

$58,602

Transcriptomic Signatures in Neuroendocrine Cell Hyperplasia of Infancy (NEHI)

Sharon McGrath-Morrow

Children's Hospital of Philadelphia

$83,154

Bronchoalveolar Lavage Fluid-Derived Organoids as Translational Models for Neuroendocrine cell hyperplasia of infancy

James Hagood

University of North Carolina

$83,154

Quantifying Movement Patterns in Children with STXBP1 Related Disorders

Laura Prosser

Children's Hospital of Philadelphia

$69,708

A deep screen of RNA-based therapeutics for STXBP1 syndrome

Chaolin Zhang

The Trustees of Columbia University in the City of New York

$69,708

Exploring the profile of Extracellular Vesicles (EVs) in Lesch-Nyhan Syndrome (LNS)

Giulia Bernardini

Università degli Studi di Siena, Dipartimento di Biotecnologie, Chimica e Farmacia

$57,332

Deciphering the role of ephrin B2 mutation in the pathogenesis of central conducting lymphatic anomaly

Shoshana Greenberger

Sheba Medical Center and Tel Aviv University

$62,398

Serum biomarkers for diagnosis and sirolimus response in lymphangioleiomyomatosis

Elizabeth P Henske

Brigham and Women's Hospital, Harvard Medical School

$73,958

Deciphering the splicing order of COL6A1-3 pre-mRNAs to improve the prognosis and treatment of collagen VI-related muscular dystrophies

Karine Choquet

Université de Sherbrooke

$46,611

Novel insights into cellular interaction in collagen VI muscular dystrophy

Malte Tiburcy

University Medical Center Göttingen

$46,611

From mouse to human – Establishing novel biomarkers for APBD

Felix Nitschke

University of Texas Southwestern Medical Center

$56,857

 
 

For complete grant list click here