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
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
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
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
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
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