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Nicolas Wein

Observed names: N Wein, N. Wein, Wein, Nicolas
Institutions: Nationwide Children's Hospital, Inserm, Centre Hospitalier Universitaire de Nantes, Translational Research in Gene Therapy, Université de Nantes, Abigail Wexner Research Institute, The Ohio State University, Nantes Université
Observed institutions: Nationwide Children's Hospital, Inserm, Aix-Marseille Université, University of Utah, Centre Hospitalier Universitaire de Nantes, Sorbonne Université, Institut de Myologie, Pitié-Salpêtrière Hospital, Génétique Médicale & Génomique Fonctionelle, Hôpital de la Timone, Centre de Recherche en Myologie, Translational Research in Gene Therapy, Université de Nantes, Abigail Wexner Research Institute, The Ohio State University, Nantes Université
ORCID: 0000-0002-5892-3639
Observed ORCIDs: 0000-0002-5892-3639, 0000-0002-8211-3644
OpenAlex ID: A5038457477
Translation from a DMD exon 5 IRES results in a functional dystrophin isoform that attenuates dystrophinopathy in humans and mice
2014 · Nicolas Wein, Adeline Vulin, et al. · Nature Medicine · 120
Genetics and Emerging Treatments for Duchenne and Becker Muscular Dystrophy
2015 · Nicolas Wein, Lindsay Alfano, et al. · Pediatric Clinics of North America · 99
Efficient bypass of mutations in dysferlin deficient patient cells by antisense-induced exon skipping
2009 · Nicolas Wein, Aurélie Avril, et al. · Human Mutation · 98
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A Naturally Occurring Human Minidysferlin Protein Repairs Sarcolemmal Lesions in a Mouse Model of Dysferlinopathy
2010 · Martin Krahn, Nicolas Wein, et al. · Science Translational Medicine · 94
Neuron-Schwann cell interactions in peripheral nervous system homeostasis, disease, and preclinical treatment
2023 · Julia Teixeira Oliveira, Christopher Yanick, et al. · Frontiers in Cellular Neuroscience · 56
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Personalized gene and cell therapy for Duchenne Muscular Dystrophy
2018 · Florian Barthélémy, Nicolas Wein · Neuromuscular Disorders · 53
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Clinical phenotypes as predictors of the outcome of skipping around DMD exon 45
2015 · Andrew R. Findlay, Nicolas Wein, et al. · Annals of Neurology · 51
Intron mutations and early transcription termination in Duchenne and Becker muscular dystrophy
2022 · Megan A. Waldrop, Steven A. Moore, et al. · Human Mutation · 46
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UMD-DYSF, a novel locus specific database for the compilation and interactive analysis of mutations in the dysferlin gene
2011 · Gaelle Blandin, Christophe Beroud, et al. · Human Mutation · 46
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Exon 32 Skipping of Dysferlin Rescues Membrane Repair in Patients’ Cells
2015 · Florian Barthélémy, Cédric Blouin, et al. · Journal of Neuromuscular Diseases · 45
Translational Research and Therapeutic Perspectives in Dysferlinopathies
2011 · Florian Barthélémy, Nicolas Wein, et al. · Molecular Medicine · 43
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Lack of Toxicity in Nonhuman Primates Receiving Clinically Relevant Doses of an AAV9.U7snRNA Vector Designed to Induce DMD Exon 2 Skipping
2021 · Liubov V. Gushchina, Emma C. Frair, et al. · Human Gene Therapy · 41
Dp412e: a novel human embryonic dystrophin isoform induced by BMP4 in early differentiated cells
2015 · Emmanuelle Massouridès, Jérôme Polentes, et al. · Skeletal Muscle · 37
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Efficient Skipping of Single Exon Duplications in DMD Patient-Derived Cell Lines Using an Antisense Oligonucleotide Approach
2017 · Nicolas Wein, Adeline Vulin, et al. · Journal of Neuromuscular Diseases · 36
The first exon duplication mouse model of Duchenne muscular dystrophy: A tool for therapeutic development
2015 · Adeline Vulin, Nicolas Wein, et al. · Neuromuscular Disorders · 32
The ZZ Domain of Dystrophin in DMD: Making Sense of Missense Mutations
2013 · Adeline Vulin, Nicolas Wein, et al. · Human Mutation · 30
Pre-clinical dose-escalation studies establish a therapeutic range for U7snRNA-mediated DMD exon 2 skipping
2021 · Tabatha R. Simmons, Tatyana A. Vetter, et al. · Molecular Therapy — Methods & Clinical Development · 29
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Novel MECP2 gene therapy is effective in a multicenter study using two mouse models of Rett syndrome and is safe in non-human primates
2023 · Samantha Powers, Shibi Likhite, et al. · Molecular Therapy · 28
Designed U7 snRNAs inhibit DUX4 expression and improve FSHD-associated outcomes in DUX4 overexpressing cells and FSHD patient myotubes
2020 · Afrooz Rashnonejad, Gholamhossein Amini-Chermahini, et al. · Molecular Therapy — Nucleic Acids · 26
Identification of Different Genomic Deletions and One Duplication in the Dysferlin Gene Using Multiplex Ligation-Dependent Probe Amplification and Genomic Quantitative PCR
2009 · Martin Krahn, Ana Borges, et al. · Genetic Testing and Molecular Biomarkers · 26
Systemic delivery of an AAV9 exon-skipping vector significantly improves or prevents features of Duchenne muscular dystrophy in the Dup2 mouse
2022 · Nicolas Wein, Tatyana A. Vetter, et al. · Molecular Therapy — Methods & Clinical Development · 25
Emerging Perspectives on Gene Therapy Delivery for Neurodegenerative and Neuromuscular Disorders
2022 · Cintia Gomez Limia, Megan Baird, et al. · Journal of Personalized Medicine · 23
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X-linked muscular dystrophy in a Labrador Retriever strain: phenotypic and molecular characterisation
2020 · Barthélémy Inès, Inès Barthelemy, et al. · Skeletal Muscle · 21
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Mechanisms of IRF2BPL-related disorders and identification of a potential therapeutic strategy
2022 · Shrestha Sinha Ray, Debdeep Dutta, et al. · Cell Reports · 17
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U7 snRNA, a Small RNA with a Big Impact in Gene Therapy
2021 · Daniel Lesman, Yacidzohara Rodriguez, et al. · Human Gene Therapy · 17
Works count: 105
Citation count: 1,274
H-index: 22
I10-index: 32
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