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Published on: April 10, 2019
Potential Involvement of Ferroptosis in Duchenne Muscular Dystrophy-Associated Cardiomyopathy
Nadezhda Fefelova1, Sri Harika Pamarthi1, Satvik Mareedu1
1Department of Cell Biology and Molecular Medicine, Rutgers New Jersey Medical School, 185 S Orange Ave. MSB C-506, Newark, NJ 07103, USA.
Insights
Ferroptosis, a cell death pathway, significantly contributes to Duchenne muscular dystrophy cardiomyopathy. Reducing sarcolipin expression may offer a therapeutic strategy by suppressing ferroptosis in DMD hearts.
Area of Science:
- Biomedical Science
- Cardiovascular Research
- Cellular Biology
Background:
- Cardiomyopathy (CM) is a major cause of mortality in Duchenne muscular dystrophy (DMD).
- Ferroptosis, a form of cell death involving lipid peroxidation, is implicated in cardiovascular diseases but its role in DMD-CM is unknown.
- DMD patients often experience severe cardiac complications, necessitating research into underlying mechanisms.
Purpose of the Study:
- To investigate the role of ferroptosis in the pathogenesis of Duchenne muscular dystrophy cardiomyopathy (DMD-CM).
- To explore the potential of targeting ferroptosis and sarcolipin (SLN) as therapeutic strategies for DMD-CM.
Main Methods:
- Utilized dystrophin and utrophin double-knockout (mdx:utr-/-) mice as a model for DMD-CM.
- Assessed iron deposition, lipid peroxidation, and ferroptosis susceptibility in cardiomyocytes.
- Examined ferroptosis biomarkers and the effect of heterozygous sarcolipin knockout (sln+/-) in mdx:utr-/- mice and human DMD heart samples.
Main Results:
- mdx:utr-/- mice hearts showed increased iron deposition and lipid peroxidation.
- mdx:utr-/- cardiomyocytes were more susceptible to ferroptosis.
- Reducing sarcolipin expression (sln+/-) significantly suppressed ferroptosis in mdx:utr-/- mice.
- Ferroptosis biomarker alterations in mdx:utr-/- mice mirrored those in human DMD hearts.
Conclusions:
- Ferroptosis is a key contributor to the pathology of Duchenne muscular dystrophy cardiomyopathy.
- Targeting ferroptosis and reducing sarcolipin expression show promise as potential therapeutic interventions for DMD-CM.
Abstract:
Background/Objectives: Cardiomyopathy (CM) is a leading cause of morbidity and mortality in Duchenne muscular dystrophy (DMD) patients. Ferroptosis, an iron-dependent form of cell death characterized by lipid peroxidation, is implicated in various cardiovascular diseases. However, the role of ferroptosis in DMD-CM remains unexplored. Methods: Here, we used dystrophin and utrophin double-knockout (mdx:utr-/-) mice as a model that exhibits cardiac pathological phenotypes similar to those seen in DMD patients to investigate the potential role of ferroptosis. Results: We observed an increased level of iron deposition and lipid peroxidation in the hearts of mdx:utr-/- mice. Live/Dead viability assays revealed that mdx:utr-/- cardiomyocytes exhibited greater susceptibility to ferroptosis than WT cardiomyocytes both at baseline and upon exposure to ferroptosis inducers. We also used mdx:utr-/- mice with a heterozygous sarcolipin (SLN) knockout background (sln+/-) to investigate the effect of SLN reduction on ferroptosis susceptibility in DMD-CM. Notably, ferroptosis was significantly suppressed in cardiomyocytes from mdx:utr-/-:sln+/- mice (p < 0.01). Western blot analysis confirmed the upregulation of transferrin receptor 1 (TfR1) and 15-lipoxygenase-1 (15LOX1), along with the downregulation of heme oxygenase-1 (HMOX-1) and ferroptosis suppressor protein 1 (FSP1) in mdx:utr-/- hearts, while glutathione peroxidase 4 (GPX4) levels remained unchanged. A similar pattern of alterations in ferroptosis-related biomarkers was observed in human heart samples from DMD patients compared to healthy controls. Conclusions: Our results provide direct evidence that ferroptosis contributes to the pathology of DMD-CM and suggest that reducing SLN expression and inhibiting ferroptosis may represent potential therapeutic strategies for this condition.
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