Nicolas Wein

Assistant Professor

Nationwide Children's Hospital

United States

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Articles (15)

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2023

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2023

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2023

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2023

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2022

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2022

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2022

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2022

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2022

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2021

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2021

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2021

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2021

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2021

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2020

Collaborators (6)

Kathrin Meyer

Assistant Professor

Ohio State University

UNITED STATES

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Kamal Gadalla

Lecturer

University of Nottingham

UNITED KINGDOM

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Steven Moore

Professor

University of Iowa Healthcare

UNITED STATES

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Carlos J Miranda

VP, Head of Preclinical

Biological E. Limited

UNITED KINGDOM

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Jeffrey Statland

Professor

University of Kansas Medical Center

UNITED STATES

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Kevin M. Flanigan

UNITED STATES

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Nicolas Wein

Research Interests

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Articles (15)

Novel MECP2 gene therapy is effective in a multicenter study using two mouse models of Rett syndrome and is safe in non-human primates

Persistence of exon 2 skipping and dystrophin expression at 18 months after U7snRNA-mediated therapy in the Dup2 mouse model.

Neuron-Schwann cell interactions in peripheral nervous system homeostasis, disease, and preclinical treatment.

Promising AAV.U7snRNAs vectors targeting <i>DMPK</i> improve DM1 hallmarks in patient-derived cell lines.

Intron mutations and early transcription termination in Duchenne and Becker muscular dystrophy.

Emerging Perspectives on Gene Therapy Delivery for Neurodegenerative and Neuromuscular Disorders

Systemic PPMO-mediated dystrophin expression in the Dup2 mouse model of Duchenne muscular dystrophy.

Systemic delivery of an AAV9 exon-skipping vector significantly improves or prevents features of Duchenne muscular dystrophy in the Dup2 mouse.

Mechanisms of IRF2BPL-related disorders and identification of a potential therapeutic strategy.

U7 snRNA, a Small RNA with a Big Impact in Gene Therapy.

Absence of Significant Off-Target Splicing Variation with a U7snRNA Vector Targeting DMD Exon 2 Duplications

Pre-clinical dose-escalation studies establish a therapeutic range for U7snRNA-mediated DMD exon 2 skipping

Lack of Toxicity in Nonhuman Primates Receiving Clinically Relevant Doses of an AAV9.U7snRNA Vector Designed to Induce <i>DMD</i> Exon 2 Skipping.

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

Designed U7 snRNAs inhibit <i>DUX4</i> expression and improve FSHD-associated outcomes in <i>DUX4</i> overexpressing cells and FSHD patient myotubes.

Collaborators (6)

Kathrin Meyer

Kamal Gadalla

Steven Moore

Carlos J Miranda

Jeffrey Statland

Kevin M. Flanigan

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