2019 – 

Title:  Drugs targeting DUX4 expression in FSHD

Principal Investigator: Dr. Fran Sverdrup, St. Louis University

The grant will sponsor a postdoctoral fellow supervised by Dr. Sverdrup.

Award Amount: $62,623

2018 – 

Dr. Peter L. Jones is the Mick Hitchcock, PhD, Endowed Chair in Medical Biochemistry and Associate Professor in Pharmacology at the University of Nevada, Reno School of Medicine.

BioPredict Inc. is a US biotechnology company focused on the development and application of novel drug discovery computational technologies.

The Carrino Foundation connected Dr. Peter Jones with BioPredict. The collaboration has identified new epigenetic regulators of DUX4 expression, which represent promising new drug targets for FSHD. This proposal aims to develop specific and effective small molecule inhibitors to these targets, in order to correct the underlying epigenetic defect in this disease.

Award Amount: $75,000

Title: Translational research and discovering prospective treatment option for FSHD patients. (Year 1 of 2)

Principal Investigator: Dr. Angela Lek, Yale University

Post Doc: Justin Cohen, PhD

Dr. Cohen is a talented researcher living with FSHD. In part, the grant will further develop his skills at the bench, while concurrently training him in other non-bench options that will allow him to transition to various roles beyond his post-doctoral roles.

Award Amount: $180,000 over 2 years

Title: Developing a novel FSHD therapy using N.A.P.A.L.M., a novel nucleic acid-peptide conjugate designed to destroy the DUX4 protein

Principal Investigator: Scott Q. Harper, PhD

Department of Pediatrics, The Ohio State University and Center for Gene Therapy, The Research Institute at Nationwide Children’s Hospital

Columbus, OH

Award Amount: $85,000

Title: Regulation and activity of the DUX4 transcription factor

Principal Investigators: Dr. Stephen Tapscott and Dr. Amy Campbell

Fred Hutchinson Cancer Research Center

Award Amount: $325,950 over 4 years*

*Co-Funded with Friends of FSH Research

Title: Developing a muscle-specific DUX4 inhibition system for FSHD therapy

Principal Investigator: Jocelyn O. Eidahl, Ph.D.

Department of Pediatrics, The Ohio State University and Center for Gene Therapy, The Research Institute at Nationwide Children’s Hospital

Columbus, OH

Award Amount: $71,613*

*Co-Funded with Friends of FSH Research

2017 – 

Title: Developing a muscle-specific DUX4 inhibition system for FSHD therapy

Principal Investigator: Jocelyn O. Eidahl, Ph.D.

Department of Pediatrics, The Ohio State University and Center for Gene Therapy, The Research Institute at Nationwide Children’s Hospital

Columbus, OH

Award Amount: $78,658*

*Co-Funded with Friends of FSH Research

2016 – 

Title: Developing a muscle-specific DUX4 inhibition system for FSHD therapy

Principal Investigator: Scott Q. Harper, PhD

Department of Pediatrics, The Ohio State University and Center for Gene Therapy, The Research Institute at Nationwide Children’s Hospital

Columbus, OH

Award Amount: $85,000

2015 – 

Title: Developing a muscle-specific DUX4 inhibition system for FSHD therapy

Principal Investigator: Scott Q. Harper, PhD

Department of Pediatrics, The Ohio State University and Center for Gene Therapy, The Research Institute at Nationwide Children’s Hospital

Columbus, OH

Award Amount: $75,625

2014 –

Title: An FSHD Model for therapeutic development, Renewal for Year 2

Principal Investigator: Peter L. Jones

Department of Cell and Developmental Biology, University of Massachusetts Medical School

Award Amount: $107,777

Title: Developing a muscle-specific DUX4 inhibition system for FSHD therapy

Principal Investigator: Scott Q. Harper, PhD

Department of Pediatrics, The Ohio State University and Center for Gene Therapy, The Research Institute at Nationwide Children’s Hospital

Columbus, OH

Award Amount: $49,473

2013 – 

Title: An FSHD Model for therapeutic development

Principal Investigator: Peter L. Jones

Department of Cell and Developmental Biology, University of Massachusetts Medical School

Award Amount: $25,000