A fleeting glimpse of functional benefit of the complete DMD gene in a large animal model of Duchenne muscular dystrophy
Author:
Publisher
Elsevier BV
Subject
Drug Discovery,Molecular Medicine
Reference10 articles.
1. Phenotypic features of dystrophin gene knockout pigs harboring a human artificial chromosome containing the entire dystrophin (DMD) gene;Watanabe;Mol. Ther. Nucleic Acids,2023
2. Is it time for genetic modifiers to predict prognosis in Duchenne muscular dystrophy?;Bello;Nat. Rev. Neurol.,2023
3. A cellular and molecular spatial atlas of dystrophic muscle;Stec;Proc. Natl. Acad. Sci. USA,2023
4. Single-cell and spatial transcriptomics identify a macrophage population associated with skeletal muscle fibrosis;Coulis;Sci. Adv.,2023
5. Assessment of systemic AAV-microdystrophin gene therapy in the GRMD model of Duchenne muscular dystrophy;Birch;Sci. Transl. Med.,2023
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