Emma C. Frair

Center for Gene Therapy

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PUBLICATIONS

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

INSTITUTIONS

The Research Institute at Nationwide Children’s Hospital

JOVE ARTICLES

Camila F. Almeida¹,

Emma C. Frair¹,

Nianyuan Huang¹,

Reid Neinast²,

Kim L. McBride^2,3,4,6,

Robert B. Weiss⁵,

Kevin M. Flanigan^1,6,

Nicolas Wein^1,6

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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