Correction of genetic disease by making sense from nonsense
Author:
Publisher
American Society for Clinical Investigation
Subject
General Medicine
Reference24 articles.
1. Suppression of a nonsense mutation in mammalian cellsin vivoby the aminoglycoside anthiotics G–418 and paromomycin
2. Aminoglycoside antibiotics restore CFTR function by overcoming premature stop mutations
3. Suppression of a CFTR premature stop mutation in a bronchial epithelial cell line
4. Aminoglycoside antibiotics restore dystrophin function to skeletal muscles of mdx mice
5. The Molecular Basis of Muscular Dystrophy in the mdx Mouse: a Point Mutation
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1. Gene therapy for Duchenne muscular dystrophy: an update on the latest clinical developments;Expert Review of Neurotherapeutics;2023-08-21
2. Balancing Nonsense Mutation Readthrough and Toxicity of Designer Aminoglycosides for Treatment of Genetic Diseases;ACS Medicinal Chemistry Letters;2023-04-27
3. Chemo‐enzymatic Synthesis of Pseudo‐trisaccharide Aminoglycoside Antibiotics with Enhanced Nonsense Read‐through Inducer Activity;ChemMedChem;2022-11-08
4. Therapeutic Strategies for Dystrophin Replacement in Duchenne Muscular Dystrophy;Frontiers in Medicine;2022-03-28
5. Chemo-Enzymatic Synthesis of Pseudo-Trisaccharidic Aminoglycoside Antibiotics with Enhanced Nonsense Read-Through Inducer Activity;SSRN Electronic Journal;2022
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