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Grant: Project Grant

University of Ottawa

$956,250.00

Characterizing the implication and therapeutic potential of AMPK signaling in Myotonic Dystrophy Type 1 (DM1)

EF 2025-26

Résumé
Myotonic Dystrophy type 1 (DM1) is caused by a genetic mutation that affects the normal function of multiple organs including skeletal muscle. Our research led us to investigate AMPK signaling in DM1 skeletal muscle. We recently found that AMPK signaling is altered in DM1 muscle, and that the activation of AMPK with drugs and exercise is beneficial for the pathology. Furthermore, we also identified the RNA-binding protein HuR is affected by the altered AMPK signaling and that it plays a direct role in the regulation of genes affected in DM1. Here, we will build upon these initial findings and investigate the role of AMPK in DM1 and the influence of HuR in DM1 pathogenesis. The characterization of this pathway in DM1 appears crucial and timely as this study will pave the way to a better understanding of the DM1 pathology while leading to the development of novel therapeutic strategies for DM1.
Programme
Project Grant
Bénéficiaire
Emplacement
Ontario, Canada
Type de bénéficiaire
Academia
Type
Grant
Source
CIHR Funding Decisions Database

Tel que publié

Id
564919
Name
Jasmin Bernard J
Region
Ontario
Country
Canada
Orgtype
Institution Paid; Research Institution
Instname2
Musculoskeletal Health and Arthritis
Theme2
Biomedical
Prcname2
Cell Biology - Disease
Projecttitle
Characterizing the implication and therapeutic potential of AMPK signaling in Myotonic Dystrophy Type 1 (DM1)
Programtype2
Operating Grants
Orgnameinp2
University of Ottawa
Cihramount2
$956,250
Cihrequipment2
$0
Programname2
Project Grant
Keyworddelim
Alternative Splicing; Ampk Signalling; Excercise; Myotonic Dystrophy Type 1; Neuromuscular Disease; Pharmacological; Preclinical; Rna-Binding Proteins; Skeletal Muscle; Therapeutics
Abstract
Myotonic Dystrophy type 1 (DM1) is caused by a genetic mutation that affects the normal function of multiple organs including skeletal muscle. Our research led us to investigate AMPK signaling in DM1 skeletal muscle. We recently found that AMPK signaling is altered in DM1 muscle, and that the activation of AMPK with drugs and exercise is beneficial for the pathology. Furthermore, we also identified the RNA-binding protein HuR is affected by the altered AMPK signaling and that it plays a direct role in the regulation of genes affected in DM1. Here, we will build upon these initial findings and investigate the role of AMPK in DM1 and the influence of HuR in DM1 pathogenesis. The characterization of this pathway in DM1 appears crucial and timely as this study will pave the way to a better understanding of the DM1 pathology while leading to the development of novel therapeutic strategies for DM1.
Competitiondate
202603
Approvedterm2
5 yrs 0 mth
Pinamesdelim
Jasmin, Bernard J
Approvedterm
5 ans 0 mois
Cihrcontribution2
~ $956,250~ $0~