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Center for Gene Therapy
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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
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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