Nicolas Wein
Biochemistry, Genetics and Molecular Biology · The Ohio State University
Publications
97
Citations
1,239
Est. group size
~3
Recurring co-author estimate
Active years
20
Publishing since 2007
Nicolas Wein's research focuses on developing gene therapy approaches for Duchenne muscular dystrophy (DMD) and related neuromuscular and genetic disorders, particularly using antisense oligonucleotide and AAV.U7snRNA-mediated exon-skipping techniques to restore dystrophin protein expression. His work spans preclinical studies in cell and animal models (including mice and dogs) as well as translational and mechanistic studies of related conditions like Rett syndrome. Prospective students would work on molecular therapeutics aimed at correcting genetic mutations underlying inherited muscle diseases.
Publication output rose from a low base in 2017 to a steady rate of about 6 papers per year between 2020 and 2024, though counts appear lower in 2025-2026.
Generated by claude-sonnet-5 from public bibliographic data · Jul 20, 2026
- Bridging science and hope: the evolving story of gene therapy for neuromuscular diseases
Frontiers in Cell and Developmental Biology · 2026
- Design parameter optimization of a membrane reactor for methanol synthesis using a sophisticated CFD model
Energy Advances · 2025
- Exploring Therapies for Duchenne Muscular Dystrophy Using Transdifferentiated Patient Fibroblasts
Methods in molecular biology · 2025
- 422P U7snRNA-mediated exon 17 skipping restores dystrophin expression in cells and in a novel mouse model of Duchenne muscular dystrophy
Neuromuscular Disorders · 2024
- 699P Exon skipping for the second Calponin Homology Domain of dystrophin using AAV.U7snRNA - In vitro & Intramuscular studies using a novel murine model of Duchenne muscular dystrophy
Neuromuscular Disorders · 2024
- 701P AAV.U7.ex44 mediates efficient exon skipping, protein restoration & phenotype rescue – pre-clinical intramuscular and dose escalation study for a mutational hotspot of the Duchenne muscular dystrophy (DMD)
Neuromuscular Disorders · 2024
- Novel MECP2 gene therapy is effective in a multicenter study using two mouse models of Rett syndrome and is safe in non-human primates
Molecular Therapy · 2023
- Intron mutations and early transcription termination in Duchenne and Becker muscular dystrophy
Human Mutation · 2022
- Mechanisms of IRF2BPL-related disorders and identification of a potential therapeutic strategy
Cell Reports · 2022
- Performance Requirements of Membrane Reactors for the Application in Renewable Methanol Synthesis: A Techno‐Economic Assessment
Advanced Sustainable Systems · 2022
- Pre-clinical dose-escalation studies establish a therapeutic range for U7snRNA-mediated DMD exon 2 skipping
Molecular Therapy — Methods & Clinical Development · 2021
- X-linked muscular dystrophy in a Labrador Retriever strain: phenotypic and molecular characterisation
Skeletal Muscle · 2020
- DMD – ANIMAL MODELS & PRECLINICAL TREATMENT
Neuromuscular Disorders · 2020
- X-linked muscular dystrophy in a Labrador Retriever strain: phenotypic and molecular characterization.
Research Square · 2020
- X-linked muscular dystrophy in a Labrador Retriever strain: phenotypic and molecular characterization.
Research Square · 2020
- Neuromuscular Disorders×15
- Molecular Therapy×3
- Molecular Therapy — Methods & Clinical Development×3
- Human Gene Therapy×2
- Molecular Therapy — Nucleic Acids×2
- Stephanie N. Oprescu
Biochemistry, Genetics and Molecular Biology · Purdue University West Lafayette
- K. Flanigan
Biochemistry, Genetics and Molecular Biology · The Ohio State University
- E. Frair
Biochemistry, Genetics and Molecular Biology · The Ohio State University
- Liubov V. Gushchina
Biochemistry, Genetics and Molecular Biology · The Ohio State University
- Scott Q. Harper
Biochemistry, Genetics and Molecular Biology · The Ohio State University
This profile was generated automatically from public scholarly data (OpenAlex). Group size and activity levels are estimates derived from co-authorship patterns.
Last updated Jul 19, 2026.
Claim or correct this profile