miR-33 inhibition as a novel therapeutic approach for treating muscular dystrophy
Open accessAbstract
Duchenne muscular dystrophy (DMD) is a devastating disorder caused by pathogenic variants in the dystrophin gene resulting in the absence of a functional dystrophin protein. While the primary cause of DMD is well-documented, the impact of disrupted secondary signaling pathways in dystrophic muscles and organs is still being unraveled. MicroRNAs are small, non-coding RNAs known to regulate key signaling pathways in growth, regeneration, and disease. In this issue of EMBO Molecular Medicine, Sowa and colleagues report the inhibition of a key microRNA (miR-33a/b) that when inhibited can improve muscle regeneration and mitigate dystrophic symptoms in a DMD (mdx) mouse model. miR-33a/b knockout mice showed enhanced muscle regeneration response to cardiotoxin injury and attenuated muscle fibrosis on the mdx background. miR-33a/b muscle expression blocked muscle satellite cell proliferation impaired muscle regeneration while increasing by targeting Cdk6, Fst, and Abca1 transcripts. Injection of anti-microRNA oligonucleotides (AMOs) targeting miR-33a/b improved muscle regeneration, histopathologies, and dystrophic regeneration signaling pathways. These studies demonstrate the impact of key secondary signaling pathways that may serve as new therapeutic avenues for the treatment of DMD symptoms.
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miR-33a/b is abundantly expressed in skeletal muscle tissues and is important for muscular regeneration.
“Recently, miR-33a/b was found to be abundantly expressed in skeletal muscle tissues and to be important for muscular regeneration.”
From Critical Role of microRNA-33a/b in Cardiovascular and Metabolic Disease: Molecular Mechanisms and Therapeutic Perspectives (2026) · cites this paper as [46]
The Abstract reports miR-33a expression in skeletal muscle and describes evidence that miR-33a/b affects muscle regeneration in mouse models. It does not establish that both miR-33a and miR-33b are abundantly expressed in skeletal muscle; the expression comparison given is for miR-33a alone. (LLM, conf=0.86)
Experimental — accuracy not yet measuredIn muscle stem cells (satellite cells), miR-33a/b suppress the expression of genes crucial for muscle regeneration and maturation, such as CDK6, FST, and ABCA1.
“In muscle stem cells, known as satellite cells, miR-33a/b suppress the expression of genes crucial for muscle regeneration and maturation, such as cyclin-dependent kinase 6 (CDK6), follistatin (FST), and ABCA1 45 , 46) .”
From Critical Role of microRNA-33a/b in Cardiovascular and Metabolic Disease: Molecular Mechanisms and Therapeutic Perspectives (2026) · cites this paper as [46]
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Cites
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Horie, T., Baba, O., Nishino, T., Yamashita, Y., Tsujisaka, Y., & Ono, K. (2026) · Journal of Atherosclerosis and Thrombosis · doi:10.5551/jat.rv22045 · Cited by 5 · see citing works
Beyond Junk DNA: Emerging Roles of Non-Coding RNAs in Cancer and Therapeutic Innovation
Qiu, L., Xu, Z., Xu, J., Lv, Z., & Li, W. (2026) · Biomolecules · doi:10.3390/biom16081110
- Authors
- Lopez, M. A., & Alexander, M. S.
- Affiliations
- Children's of Alabama (US)
- Civitan International (US)
- Corresponding author
- Alexander, Matthew S
- Venue
- EMBO Molecular Medicine
- Published
- 2025-07-23
- Volume
- 17
- Issue
- 8
- Pages
- 1893-1895
- ISSN
- 1757-4676 (ISSN-L)
- 1757-4684
- PMC
- PMC12339987
- OpenAlex
- W4412580587
- Cited by
- 2
- References
- 13
- Citations by year
- 2026: 1 · 2025: 1
- FWCI
- 0.60
- Type
- journal article
- Language
- en
- Open access
- gold
- doi.org
- Locations
- EMBO Molecular Medicine (publishedVersion, cc-by)
- PubMed Central (publishedVersion, cc-by)
- PubMed (publishedVersion)
- DOAJ (DOAJ: Directory of Open Access Journals) (submittedVersion, cc-by-sa)
- Funders
- HHS | NIH | NIH Office of the Director (U54OD030167)
- HHS | NIH | National Institute of Neurological Disorders and Stroke (R21NS140497, K08-NS120812)
- U.S. Department of Defense (MD240021)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › Muscle Physiology and Disorders
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Cancer Research › MicroRNA in disease regulation
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › Congenital heart defects research
- Keywords
- Duchenne muscular dystrophy, microRNA inhibition, muscle regeneration, muscle fibrosis, satellite cell proliferation, cardiotoxin injury, mdx mice, dystrophin, CDK6, FST, ABCA1, therapeutic target
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