Expression of SRP-9001 dystrophin and stabilization of motor function up to 2 years post-treatment with delandistrogene moxeparvovec gene therapy in individuals with Duchenne muscular dystrophy.

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作者： Jerry R,Mendell ^[1] ; Perry B,Shieh ^[2] ; Craig M,McDonald ^[3] ; Zarife,Sahenk ^[4] ; Kelly J,Lehman ^[5] ; Linda P,Lowes ^[1] ; Natalie F,Reash ^[2] ; Megan A,Iammarino ^[3] ; Lindsay N,Alfano ^[1] ; Brenna,Sabo ^[1] ; Jeremy D,Woods ^[2] ; Christy L,Skura ^[1] ; Howard C,Mao ^[1] ; Loretta A,Staudt ^[1] ; Danielle A,Griffin ^[1] ; Sarah,Lewis ^[4] ; Shufang,Wang ^[4] ; Rachael A,Potter ^[4] ; Teji,Singh ^[4] ; Louise R,Rodino-Klapac ^[6]

作者单位： Center for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus, OH, United States. ^[1] Department of Pediatrics, The Ohio State University, Columbus, OH, United States. ^[2] Department of Neurology, The Ohio State University, Columbus, OH, United States. ^[3] UCLA Medical Center, Los Angeles, CA, United States. ^[4] Departments of Physical Medicine and Rehabilitation and Pediatrics, Lawrence J. Ellison Ambulatory Care Center, UC Davis Health, Sacramento, CA, United States. ^[5] Sarepta Therapeutics Inc, Cambridge, MA, United States. ^[6]

关键词 AAVrh74 Duchenne muscular dystrophy SRP-9001 dystrophin gene therapy

DOI 10.3389/fcell.2023.1167762

PMID 37497476

发布时间 2023-07-28

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Frontiers in cell and developmental biology

2023年11卷

1167762页

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Expression of SRP-9001 dystrophin and stabilization of motor function up to 2 years post-treatment with delandistrogene moxeparvovec gene therapy in individuals with Duchenne muscular dystrophy.

Frontiers in cell and developmental biology

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Frontiers in cell and developmental biology

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Expression of SRP-9001 dystrophin and stabilization of motor function up to 2 years post-treatment with delandistrogene moxeparvovec gene therapy in individuals with Duchenne muscular dystrophy.

Frontiers in cell and developmental biology

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Frontiers in cell and developmental biology

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