Grant: Team Grant: E-Rare Joint Transnational Call
Ontario Institute for Cancer Research (Toronto, Ontario)
$90,254.00
Safety and efficacy of a possible epigenetic therapy for FSHD muscular dystrophy
FY 2020-21
Summary
Facioscapulohumeral muscular dystrophy (FSHD) is the most prevalent muscle disease that afflicts both children and adults regardless of their gender. FSHD is caused by aberrant gain of expression of the double homeobox 4 (DUX4) gene causing toxic effects in muscle cells. Despite the consensus on the pivotal role of DUX4 and several clinical trials, there is currently no cure or an effective therapeutic approach for FSHD patients. In our studies, we identified a novel regulator of DUX4 expression. Targeting this factor allows to block DUX4 expression and rescues the pathogenic behavior of muscle cells from FSHD patients. The treatment is safe to healthy muscle cells. Based on our results, we will use cellular and animal models of the disease to investigate a novel pharmacological approach that could represent a promising therapeutic option for FSHD patients.
Department
Program
Team Grant: E-Rare Joint Transnational Call
Location
Ontario, Canada
Type
Grant
Source
CIHR Funding Decisions Database
As published
Id
431263
Name
Al-Awar Rima S
Region
Ontario
Country
Canada
Orgtype
Institution Paid; Research Institution
Partnername
INSERM (Bordeaux, France); German Research Foundation (Germany); Muscular Dystrophy Canada (Toronto, Ontario); Regione Toscana (Florence, Italy); Ministero della Salute (Italy); The Scientific and Technological Research Council of Turkey
Instname2
Neurosciences, Mental Health and Addiction
Theme2
Biomedical
Prcname2
Special Cases
Projecttitle
Safety and efficacy of a possible epigenetic therapy for FSHD muscular dystrophy
Programtype2
Operating Grants
Orgnameinp2
Ontario Institute for Cancer Research (Toronto, Ontario)
Cihramount2
$90,254
Cihrequipment2
$0
Programname2
Team Grant: E-Rare Joint Transnational Call
Keyworddelim
Chromatin Therapy; Epigenetics; Fshd Muscular Dystrophy; Pre-Clinical Models; Wdr5
Abstract
Facioscapulohumeral muscular dystrophy (FSHD) is the most prevalent muscle disease that afflicts both children and adults regardless of their gender. FSHD is caused by aberrant gain of expression of the double homeobox 4 (DUX4) gene causing toxic effects in muscle cells. Despite the consensus on the pivotal role of DUX4 and several clinical trials, there is currently no cure or an effective therapeutic approach for FSHD patients. In our studies, we identified a novel regulator of DUX4 expression. Targeting this factor allows to block DUX4 expression and rescues the pathogenic behavior of muscle cells from FSHD patients. The treatment is safe to healthy muscle cells. Based on our results, we will use cellular and animal models of the disease to investigate a novel pharmacological approach that could represent a promising therapeutic option for FSHD patients.
Competitiondate
202006
Approvedterm2
3 yrs 0 mth
Pinamesdelim
Al-Awar, Rima S
Approvedterm
3 ans 0 mois
Cihrcontribution2
Institute of Genetics~ $90,254~ $0~
