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Amoasii, Leonela; Hildyard, John C W; Li, Hui; Sanchez-Ortiz, Efrain; Mireault, Alex; Caballero, Daniel; Harron, Rachel; Stathopoulou, Thaleia-Rengina; Massey, Claire; Shelton, John M; Bassel-Duby, Rhonda; Piercy, Richard J; Olson, Eric N
Science, 10/2018, Letnik: 362, Številka: 6410Journal Article
Mutations in the gene encoding dystrophin, a protein that maintains muscle integrity and function, cause Duchenne muscular dystrophy (DMD). The deltaE50-MD dog model of DMD harbors a mutation corresponding to a mutational "hotspot" in the human gene. We used adeno-associated viruses to deliver CRISPR gene editing components to four dogs and examined dystrophin protein expression 6 weeks after intramuscular delivery ( = 2) or 8 weeks after systemic delivery ( = 2). After systemic delivery in skeletal muscle, dystrophin was restored to levels ranging from 3 to 90% of normal, depending on muscle type. In cardiac muscle, dystrophin levels in the dog receiving the highest dose reached 92% of normal. The treated dogs also showed improved muscle histology. These large-animal data support the concept that, with further development, gene editing approaches may prove clinically useful for the treatment of DMD.
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JCR | SNIP | JCR | SNIP | JCR | SNIP | JCR | SNIP |
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in: SICRIS
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