Nicolas Wein

Center for Gene Therapy

The Research Institute at Nationwide Children’s Hospital

BIOGRAPHY

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PUBLICATIONS

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

INSTITUTIONS

The Research Institute at Nationwide Children’s Hospital

The Ohio State University

JOVE ARTICLES

Direct Reprogramming of Human Fibroblasts into Myoblasts to Investigate Therapies for Neuromuscular Disorders

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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Nicolas Wein

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The Research Institute at Nationwide Children’s Hospital

Direct Reprogramming of Human Fibroblasts into Myoblasts to Investigate Therapies for Neuromuscular Disorders

Center for Gene Therapy