Morpholino Oligomer-Induced Dystrophin Isoforms to Map the Functional Domains in the Dystrophin Protein
Li D, Adams A, Johnsen R, Fletcher S, Wilton S. Morpholino Oligomer-Induced Dystrophin Isoforms to Map the Functional Domains in the Dystrophin Protein. Molecular Therapy - Nucleic Acids 2020, 22: 263-272. PMID: 33230432, PMCID: PMC7516190, DOI: 10.1016/j.omtn.2020.08.019.Peer-Reviewed Original ResearchGenotype-phenotype correlationDuchenne muscular dystrophyDystrophin isoformsDystrophin expressionPre-messenger RNA splicingLack of genotype-phenotype correlationIn-frame deletionMuscular dystrophyExon-skipping strategyPeptide-conjugated phosphorodiamidate morpholino oligomersReading frameBecker muscular dystrophyRNA splicingPhosphorodiamidate morpholino oligomersSevere dystrophic pathologyExon deletionsFunctional proteinsSarcolemma stabilityExonDystrophic pathologyFunctional domainsDystrophin proteinAllelic disordersIntraperitoneal injectionMutations
This site is protected by hCaptcha and its Privacy Policy and Terms of Service apply