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65Rescue of nonsense mutations by amlexanox in human cells, Orphanet Journal of Rare Diseases Das ??berlesen von Nonsense-Mutationen, medizinische genetik Recoding Therapies for Genetic Diseases, Recoding: Expansion of Decoding Rules Enriches Gene Expression Therapy Strategies for Usher Syndrome Type 1C in the Retina, Retinal Degenerative Diseases An Overview of Recent Therapeutics Advances for Duchenne Muscular Dystrophy, Duchenne Muscular Dystrophy Genome-Edited Cell Lines for High-Throughput Screening, Reporter Gene Assays CFTR Modulator Therapies in Cystic Fibrosis, Treatment of Cystic Fibrosis and Other Rare Lung Diseases Pharmaceutical Products and Non-pharmaceutical Interventions as Potential Treatments for Patients with Muscular Dystrophy, Muscular Dystrophy Nonsense-Mediated mRNA Decay and Cystic Fibrosis, Cystic Fibrosis