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Reengineering a transmembrane protein to treat muscular dystrophy using exon skipping.
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Reengineering a transmembrane protein to treat muscular dystrophy using exon skipping.
Reengineering a transmembrane protein to treat muscular dystrophy using exon skipping. J Clin Invest. 2015 Nov 02; 125(11):4186-95.
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PubMed
subject areas
Animals
Codon, Nonsense
Diaphragm
Drosophila melanogaster
Drosophila Proteins
Dystrophin-Associated Protein Complex
Exons
Fibrosis
Genetic Therapy
HEK293 Cells
Humans
Mice
Mice, Transgenic
Muscle, Skeletal
Muscular Dystrophies, Limb-Girdle
Muscular Dystrophy, Animal
Mutation
Myocardium
Oligonucleotides, Antisense
Protein Engineering
Protein Interaction Mapping
Protein Structure, Tertiary
Recombinant Fusion Proteins
RNA, Messenger
Sarcoglycans
Sarcolemma
Sequence Deletion
authors with profiles
David Barefield
Alexis Demonbreun
Eugene Wyatt