Duchenne muscular dystrophy is caused by inadequate generation of functional dystrophin protein. Traditional clinical treatments can only slightly mitigate the progression of the disease, but not completely stem or reverse the decline in muscle function. Therapies aimed at dystrophin recovery are currently under development, among which are exon skipping and stop codon readthrough therapies. They are now used in clinics, while gene addition therapies are in phase III clinical trials. Gene editing therapies have also been approved for the first clinical trial recently. This review will discuss these emerging therapies, clinical trials, and directions for future developments.
- Open Access
- Review
Promising Treatments for Duchenne Muscular Dystrophy: Restoring Dystrophin Protein Expression Using Nucleic Acid Therapeutics
- Guo Hu,
- Chen Chen *
Author Information
Received: 10 Oct 2022 | Accepted: 10 Nov 2022 | Published: 11 Jan 2023
Abstract
Graphical Abstract
References
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How to Cite
Hu, G.; Chen, C. Promising Treatments for Duchenne Muscular Dystrophy: Restoring Dystrophin Protein Expression Using Nucleic Acid Therapeutics. International Journal of Drug Discovery and Pharmacology 2023, 2 (1), 4–10. https://doi.org/10.53941/ijddp.0201002.
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Guo Hu, Chen Chen
This work is licensed under a Creative Commons Attribution 4.0 International License.
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