Assessment of rAAVrh.74.MHCK7.micro-dystrophin Gene Therapy Using Magnetic Resonance Imaging in Children With Duchenne Muscular Dystrophy
This case-control study uses magnetic resonance imaging and spectroscopy to evaluate the association between treatment with recombinant adeno-associated virus serotype rh74 (rAAVrh74) and muscle quality in children with Duchenne muscular dystrophy.
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| Veröffentlicht in: | JAMA network open 2021-01, Vol.4 (1), p.e2031851-e2031851 |
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| creator | Willcocks, Rebecca J Forbes, Sean C Walter, Glenn A Sweeney, Lee Rodino-Klapac, Louise R Mendell, Jerry R Vandenborne, Krista |
| description | This case-control study uses magnetic resonance imaging and spectroscopy to evaluate the association between treatment with recombinant adeno-associated virus serotype rh74 (rAAVrh74) and muscle quality in children with Duchenne muscular dystrophy. |
| doi_str_mv | 10.1001/jamanetworkopen.2020.31851 |
| format | Article |
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| subjects | Case-Control Studies Child Child, Preschool Dystrophin - genetics Gene therapy Genetic Therapy - methods Genetics and Genomics Humans Magnetic Resonance Imaging - methods Muscle, Skeletal - diagnostic imaging Muscular Dystrophy, Duchenne - diagnostic imaging Muscular Dystrophy, Duchenne - therapy Online Only Research Letter Treatment Outcome |
| title | Assessment of rAAVrh.74.MHCK7.micro-dystrophin Gene Therapy Using Magnetic Resonance Imaging in Children With Duchenne Muscular Dystrophy |
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