Empirical and Computational Comparison of Alternative Therapeutic Exon Skip Repairs for Duchenne Muscular Dystrophy
Author:
Funder
National Institute of General Medical Sciences
Publisher
American Chemical Society (ACS)
Subject
Biochemistry
Link
https://pubs.acs.org/doi/pdf/10.1021/acs.biochem.9b00062
Reference70 articles.
1. Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and neuromuscular, rehabilitation, endocrine, and gastrointestinal and nutritional management
2. Duchenne Muscular Dystrophy: From Diagnosis to Therapy
3. Developing a Natural History Progression Model for Duchenne Muscular Dystrophy Using the Six-Minute Walk Test
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1. Structural Perturbations of Exon-Skipping Edits within the Dystrophin D20:24 Region;Biochemistry;2021-03-03
2. Developing DMD therapeutics: a review of the effectiveness of small molecules, stop-codon readthrough, dystrophin gene replacement, and exon-skipping therapies;Expert Opinion on Investigational Drugs;2021-01-06
3. Advances in Genetic Characterization and Genotype–Phenotype Correlation of Duchenne and Becker Muscular Dystrophy in the Personalized Medicine Era;Journal of Personalized Medicine;2020-09-03
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