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Center for Gene Therapy
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The ZZ domain of dystrophin in DMD: making sense of missense mutations.
Human mutation Feb, 2014 | Pubmed ID: 24302611
Dp412e: a novel human embryonic dystrophin isoform induced by BMP4 in early differentiated cells.
Skeletal muscle , 2015 | Pubmed ID: 26568816
Personalized gene and cell therapy for Duchenne Muscular Dystrophy.
Neuromuscular disorders : NMD 10, 2018 | Pubmed ID: 30224293
Lack of toxicity in non-human primates receiving clinically relevant doses of an AAV9.U7snRNA vector designed to induce DMD exon 2 skipping.
Human gene therapy Jan, 2021 | Pubmed ID: 33406986
The Research Institute at Nationwide Children’s Hospital
The Ohio State University
Camila F. Almeida1,
Emma C. Frair1,
Nianyuan Huang1,
Reid Neinast2,
Kim L. McBride2,3,4,6,
Robert B. Weiss5,
Kevin M. Flanigan1,6,
Nicolas Wein1,6
1Center for Gene Therapy, The Research Institute at Nationwide Children’s Hospital,
2Center for Cardiovascular Research, The Research Institute at Nationwide Children’s Hospital,
3The Heart Center, Nationwide Children’s Hospital,
4Division of Genetic and Genomic Medicine, Nationwide Children’s Hospital,
5Department of Human Genetics, The University of Utah School of Medicine,
6Department of Pediatrics, The Ohio State University
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