The Future of Exon Skipping for Duchenne Muscular Dystrophy
Author:
Affiliation:
1. Department of Human Genetics, Leiden University Medical Center, Leiden, the Netherlands.
Publisher
Mary Ann Liebert Inc
Subject
Genetics,Molecular Biology,Molecular Medicine
Link
https://www.liebertpub.com/doi/pdf/10.1089/hum.2023.026
Reference37 articles.
1. The complete sequence of dystrophin predicts a rod-shaped cytoskeletal protein
2. Duchenne muscular dystrophy
3. Characterization of Dystrophin in Muscle-Biopsy Specimens from Patients with Duchenne's or Becker's Muscular Dystrophy
4. Next steps for the optimization of exon therapy for Duchenne muscular dystrophy
5. Antisense-induced exon skipping and synthesis of dystrophin in the mdx mouse
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1. Large scale serum proteomics identifies proteins associated with performance decline and clinical milestones in Duchenne muscular dystrophy;2024-08-07
2. SPLICER: A Highly Efficient Base Editing Toolbox That EnablesIn VivoTherapeutic Exon Skipping;2024-04-02
3. Nucleic Acid Therapeutics: Successes, Milestones, and Upcoming Innovation;Nucleic Acid Therapeutics;2024-04-01
4. Dystrophin and Associated Proteins;Reference Module in Neuroscience and Biobehavioral Psychology;2024
5. Applying Lessons Learned from Developing Exon Skipping for Duchenne to Developing Individualized Exon Skipping Therapy for Patients with Neurodegenerative Diseases;Synlett;2023-11-15
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